Sharing Knowledge. Sharing Hope. 2015 Annual Report CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program®/Be The Match® and Medical College of Wisconsin 2015 Annual Report January – December Milwaukee Campus Medical College of Wisconsin 9200 W Wisconsin Ave, Suite C5500 Milwaukee, WI 53226 (414) 805-0700 Minneapolis Campus National Marrow Donor Program/ Be The Match 500 N 5th St Minneapolis, MN 55401 (612) 627-5800 cibmtr.org CIBMTR 2015 Annual Report TABLE OF CONTENTS TABLE OF CONTENTS 1.0 WHO WE ARE ........................................................................................................................................ 1 1.1 Mission .............................................................................................................................................. 1 1.2 Value to the HCT Community ............................................................................................................ 1 1.3 Organizational Structure ................................................................................................................... 1 1.3.1 Scientific Working Committees .................................................................................................. 6 2.0 WHAT WE DO ........................................................................................................................................ 8 2.1 Clinical Outcomes Research Program ............................................................................................. 11 2.1.1 Scientific Working Committees ................................................................................................ 11 2.1.2 Cellular Therapies and Non-Transplant Therapy Research Initiatives ..................................... 15 2.2 Immunobiology Research Program ................................................................................................. 16 2.3 Clinical Trials Support Program ....................................................................................................... 18 2.3.1 Blood and Marrow Transplant Clinical Trials Network ............................................................ 18 2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation ............................ 20 2.4 Health Services Research Program.................................................................................................. 21 2.5 Bioinformatics Research Program ................................................................................................... 23 2.6 Statistical Methodology Research Program .................................................................................... 24 2.7 Stem Cell Therapeutic Outcomes Database (SCTOD) ..................................................................... 25 2.8 Corporate Program.......................................................................................................................... 27 3.0 HOW WE SHARE KNOWLEDGE ........................................................................................................... 28 3.1 Information Request Service ........................................................................................................... 33 3.2 Internet Presence ............................................................................................................................ 34 3.2.1 CIBMTR Public Website ............................................................................................................ 34 3.2.2 CIBMTR Collaborative Site ........................................................................................................ 35 3.2.3 CIBMTR Portal Site.................................................................................................................... 36 3.2.4 Be The Match Public Website................................................................................................... 37 3.2.5 Be The Match Clinical Website ................................................................................................. 37 3.2.6 HRSA Blood Cell Transplant Website........................................................................................ 37 3.2.7 Other Applications and Data Exchange Standards................................................................... 38 3.3 BMT Tandem Meetings ................................................................................................................... 39 3.4 Data Management Training ............................................................................................................ 40 4.0 HOW WE COLLECT AND MANAGE DATA ............................................................................................ 41 4.1 Research Data Life Cycle ................................................................................................................. 41 4.2 Collecting and Storing Data ............................................................................................................. 42 4.2.1 FormsNet .................................................................................................................................. 42 4.2.2 Research Database ................................................................................................................... 42 4.3 Ensuring Data Quality ...................................................................................................................... 42 i CIBMTR 2015 Annual Report TABLE OF CONTENTS 4.3.1 Continuous Process Improvement ........................................................................................... 42 4.3.2 Verification and Validation ....................................................................................................... 43 4.3.3 On-Site Data Audit Program ..................................................................................................... 44 4.4 Protecting Patients and Data .......................................................................................................... 45 4.4.1 Human Subjects / HIPAA Compliance ...................................................................................... 45 4.4.2 Information Security and Data Privacy..................................................................................... 45 5.0 WHAT WE WILL DO NEXT.................................................................................................................... 46 2015 KEY ACCOMPLISHMENTS ................................................................................................................. 49 APPENDIX A: TRANSPLANT CENTERS ........................................................................................................ 53 Appendix A1: US Centers....................................................................................................................... 54 Appendix A2: International Centers ...................................................................................................... 65 APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP ..................... 74 Appendix B1: Organizational Structure – Milwaukee Campus ............................................................. 75 Appendix B2: Organizational Structure – Minneapolis Campus ........................................................... 76 Appendix B3: Coordinating Center Leadership ..................................................................................... 78 APPENDIX C: COMMITTEE MEMBERSHIP ................................................................................................. 87 Appendix C1: Advisory Committee Membership .................................................................................. 87 Appendix C2: Executive Committee Membership ................................................................................ 90 Appendix C3: Consumer Advocacy Committee Membership ............................................................... 92 Appendix C4: Nominating Committee Membership............................................................................. 93 Appendix C5: Scientific Working Committee Leadership ..................................................................... 94 Appendix C6: Immunobiology Steering Committee Membership ........................................................ 98 Appendix C7: Clinical Trials Advisory Committee Membership ............................................................ 99 APPENDIX D: PUBLICATIONS ................................................................................................................... 100 Appendix D1: Scientific Working Committee Publications ................................................................. 100 Appendix D2: BMT CTN Publications .................................................................................................. 112 Appendix D3: Health Services Research Program Publications .......................................................... 115 Appendix D4: Bioinformatics Research Program Publications ........................................................... 116 Appendix D5: Statistical Methodology Research Program Publications ............................................ 119 Appendix D6: Coordinating Center Publications................................................................................. 120 APPENDIX E: PRESENTATIONS ................................................................................................................ 124 APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS ..................................................... 132 APPENDIX G: CLINICAL TRIALS ................................................................................................................ 135 Appendix G1: BMT CTN Clinical Trials Open for Enrollment ............................................................... 135 Appendix G2: RCI BMT Clinical Trials .................................................................................................. 137 APPENDIX H: FORMS SUBMISSION PROCESS ......................................................................................... 140 APPENDIX I: WEBSITES ............................................................................................................................ 141 APPENDIX J: GLOSSARY ........................................................................................................................... 142 ii CIBMTR 2015 Annual Report TABLE OF CONTENTS FIGURES AND TABLES Figure 1.1. Scientific Organizational Structure ........................................................................................... 2 Figure 1.2. Functional Organizational Structure with Scientific Oversight................................................. 3 Table 1.3. Committee Structure ................................................................................................................. 4 Figure and Table 2.1. Distribution of Patients in the CIBMTR Research Database by Graft Source .......... 8 Figure and Table 2.2. Distribution of Patients in the CIBMTR Research Database by Disease .................. 9 Figure 2.3. Continued Growth in the Number of Patients Registered with the CIBMTR ......................... 10 Table 2.4. 2015 Working Committee Studies ........................................................................................... 12 Figure 2.5. 2015 Publications by Program ................................................................................................ 13 Figure 2.6. Working Committee Study Proposal Review Process............................................................. 14 Figure 3.1. How to Access CIBMTR Knowledge ........................................................................................ 28 Table 3.2. How to Access CIBMTR Information ........................................................................................ 29 Table 3.3 How to Access CIBMTR Data ..................................................................................................... 29 Table 3.4 How to Access CIBMTR Tools .................................................................................................... 30 Table 3.5 How to Access CIBMTR Biospecimens ...................................................................................... 30 Table 3.6. Standard Reports Published by the CIBMTR ............................................................................ 31 Table 3.7. Data Requests Addressed by the CIBMTR in 2015................................................................... 33 Figure 4.1. Research Data Life Cycle ......................................................................................................... 41 Figure 4.2. Audit Process .......................................................................................................................... 44 Figure 5.1. Plans for 2016 ......................................................................................................................... 46 Table 5.2. Plans to Enhance Data.............................................................................................................. 47 Table 5.3. Plans to Expand Knowledge Sharing ........................................................................................ 47 Table 5.4. Plans to Increase Impact .......................................................................................................... 48 Figure A.1. Location of Transplant Centers that Submit Data to the CIBMTR ......................................... 53 iii CIBMTR 2015 Annual Report 1.0 WHO WE ARE 1.0 WHO WE ARE The CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and the Medical College of Wisconsin (MCW). 1.1 MISSION The CIBMTR collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide to increase survival and enrich quality of life for patients. The CIBMTR facilitates critical observational and interventional research through scientific and statistical expertise, a large network of transplant centers, and a unique and extensive clinical outcomes database. CIBMTR Programs Clinical Outcomes Research Immunobiology Research Clinical Trials Support • Blood and Marrow Transplant Clinical Trials Network (BMT CTN) • Resource for Clinical Investigations in Blood and Marrow Transplantation (RCI BMT) Health Services Research Bioinformatics Research Statistical Methodology Research 1.2 VALUE TO THE HCT COMMUNITY The CIBMTR has been collecting HCT outcomes data worldwide for >40 years, resulting in a Research Database with information on >425,000 patients. These data are available to investigators with interest in HCT and treatments for cancer and other lifethreatening diseases. The CIBMTR has become a respected leader in HCT research by providing a unique resource of information and expertise to the medical and scientific communities. 1.3 ORGANIZATIONAL STRUCTURE The CIBMTR (Figures 1.1 and 1.2) represents a large network of approximately 400 participating transplant centers (Appendix A) that submit transplant-related data for patients. Centers submit data at two levels: a Transplant Essential Data (TED) level, which captures basic data, and a Comprehensive Report Form (CRF) level, which captures more detail. The CIBMTR Coordinating Center, staffed by almost 200 employees (Appendix B), provides data acquisition, management, and statistical support for analyses of these data. The Chief Scientific Director is responsible for all administrative and scientific operations. The Associate and Senior Scientific Directors assist in overseeing operational aspects of the Coordinating Center. CIBMTR committees (Table 1.3) provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of the scientific and medical communities. Page | 1 CIBMTR 2015 Annual Report 1.0 WHO WE ARE Figure 1.1. Scientific Organizational Structure Transplant Centers CIBMTR Assembly National Marrow Donor Program/Be The Match Medical College of Wisconsin Joint Affiliation Committee Executive Director J Chell, MD Executive Committee Advisory role Advisory Committee Senior Scientific Director for Research Operations M Eapen, MBBS, MS Chief Statistical Director MJ Zhang, PhD Chief Scientific Director M Horowitz, MD, MS Senior Scientific Director for SCTOD JD Rizzo, MD, MS Scientific Working Committees Steering Committees Statistical Methodology Research Program MJ Zhang, PhD Clinical Outcomes Research Program M Eapen, MBBS, MS Associate Scientific Director for CIBMTR Minneapolis D Confer, MD Senior Research Advisor D Weisdorf, MD Advisory role Health Services Research Program L Burns, MD Consumer Advocacy Committee Immunobiology Research Program S Lee, MD, MPH Senior Scientific Director for Data Operations B Shaw, MD, PhD Clinical Trials Support Program D Confer, MD L Burns, MD M Pasquini, MD, MS B Shaw, MD, PhD Blood and Marrow Transplant Clinical Trials Network (BMT CTN) M Pasquini, MD, MS Bioinformatics Research Program M. Maiers, MS Resource for Clinical Investigations in Blood and Marrow Transplantation (RCI BMT) D Confer, MD L Burns, MD B Shaw, MD, PhD Page | 2 CIBMTR 2015 Annual Report 1.0 WHO WE ARE Figure 1.2. Functional Organizational Structure with Scientific Oversight Research & Administration M Horowitz & D Confer Faculty CIBMTR Administration Finance, Grants & Contracts M Horowitz & D Confer P Steinert & R King MCW & NMDP/Be The Match PhDs MDs Statistics & Observational Research Human Research Protection Program W Perez & S Spellman MKE & MPLS R King MPLS (M Eapen) (**B Lindberg) Immunobiology Research Statistics & Clinical Outcomes S Spellman MPLS W Perez & S Spellman MKE & MPLS (S Lee) (MJ Zhang) (M Eapen) Health Services Research* E Denzen MPLS (L Burns) Clinical Studies Support Quality Assurance BMT CTN Information Technology R Drexler MPLS TBD MKE A Foley MPLS E Bergman & M Prestegaard MKE & MPLS (D Confer) (L Burns) (B Shaw) (M Horowitz) (M Pasquini) RCI BMT R Drexler MPLS (D Confer) (L Burns) (B Shaw) Business Office Advancement Auditing & Monitoring Data Operations Bioinfomatics C Gonzalez & P Vespalec MKE S Fisher & T Houseman MKE & MPLS D Christianson MPLS J Brunner & M Matlack MKE & MPLS M Maiers MPLS (M Horowitz) (B Shaw) (B Shaw) (M Eapen) (D Rizzo) (B Shaw) Survey Research Group Project Management & Analysis Applications Data Solutions D Mattila MPLS T Moerke & K Gee MKE & MPLS T Hongyu & E Chan MKE & MPLS R Renner MPLS Corporate Meetings S Fisher MKE T Houseman MPLS (M Eapen) (M Horowitz) Data Management Special Projects Recipient Data Management Donor Data Management J Brunner & M Matlack MKE & MPLS S Meiers & K Gardner MKE & MPLS A Hauck MPLS (B Shaw) (B Shaw) (D Confer) (D Confer) Database Administrator & Data Architect B Liu & TBD MKE & MPLS Technical Services T Moerke MKE Communications S Fisher MKE (M Horowitz) Data Entry & Imaging Data Support Training B Levesque MPLS M Matlack MPLS M Matlack MPLS (B Shaw) (B Shaw) (B Shaw) Key: MKE = Milwaukee, MPS = Minneapolis, ( ) indicates scientific oversight, *staffed through NMDP/Be The Match Patient and Health Professional Services, ** institutional official oversight Page | 3 CIBMTR 2015 Annual Report 1.0 WHO WE ARE Table 1.3. Committee Structure Committee Function Joint Affiliation Board • Reviews and approves the CIBMTR budget and research plan • Amends the terms of the affiliation agreement, as necessary • Reviews and approves data access and confidentiality policies • Annually Assembly • Includes representatives from each transplant center that submits CRF-level data • Elects members of the Advisory, Nominating, and Clinical Trials Advisory Committees • Annually during the BMT Tandem Meetings Advisory Committee • Oversees CIBMTR policies and scientific agenda • Partners with the Working Committees to prioritize scientific studies Executive Committee • Provides scientific and policy advice to the Chief Scientific Director and Coordinating Center • Reviews audit results and makes recommendations for improvement Consumer Advocacy Committee • Provides patient and donor perspectives during the development of the CIBMTR research agenda • Communicates CIBMTR research results and data to the nonmedical community Nominating Committee • Prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees • Makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments (subcommittee of Advisory Committee) (subcommittee of Advisory Committee) Meetings Roster • In person annually at the BMT Tandem Meetings • By teleconference quarterly and as needed Appendix C1 • Four times annually by teleconference Appendix C2 • In person annually at the BMT Tandem Meetings • By teleconference periodically Appendix C3 • At least once annually each Appendix C4 Fall by teleconference Page | 4 CIBMTR 2015 Annual Report 1.0 WHO WE ARE Committee Function Meetings Roster Scientific Working Committees (Section 1.3.1) • Design and conduct relevant studies using CIBMTR data, statistical resources, networks, and / or centers • Set priorities for clinical outcomes studies • Assess and revise CIBMTR data collection forms, as needed • Plan and conduct workshops at CIBMTR meetings • In person annually at the BMT Tandem Meetings • Leadership - by teleconference every 4-8 weeks Immunobiology Steering Committee / NMDP/Be The Match Histocompatibility Advisory Group • Reviews and approves the use of donor-recipient specimens from the Research Repository in CIBMTR studies • In person twice annually, in Appendix C6 summer and at the BMT Tandem Meetings Clinical Trials Advisory Committee • Assists in the review, approval, and oversight of proposals and protocols for Phase I and Phase II clinical trials submitted to the RCI BMT • In person annually at the BMT Tandem Meetings • By teleconference as needed Leadership Appendix C5 Appendix C7 Page | 5 CIBMTR 2015 Annual Report 1.0 WHO WE ARE To ensure broad input into the research process and efficient use of resources, the CIBMTR facilitates 15 Scientific Working Committees focused on specific research areas. pharmacogenetics, stem cell biology, and other areas related to HCT provide essential expertise in their respective research areas. The Working Committee structure encourages a collaborative but rigorous methodological approach to all CIBMTR activities. Scientific Working Committees Working Committee Leadership 1.3.1 Scientific Working Committees Acute Leukemia Chairs (usually 3-4) Autoimmune Diseases and Cellular Therapies MD Scientific Director Chronic Leukemia PhD Statistical Director Donor Health and Safety MS-level Statistician Graft Sources and Manipulation Graft-versus-Host Disease Health Services and International Studies Immunobiology Infection and Immune Reconstitution Late Effects and Quality of Life Lymphoma Pediatric Cancer Plasma Cell Disorders and Adult Solid Tumors Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders Regimen-Related Toxicity and Supportive Care Total Working Committee membership exceeds 2,300 researchers. Membership is open to any researcher willing to take an active role in developing and conducting studies that use CIBMTR data and / or resources. While most of these individuals are HCT clinicians, statisticians and basic scientists also participate. PhD-level statistical faculty and Master’s-level statisticians from the CIBMTR Coordinating Center provide their unique expertise in data analysis. Basic scientists investigating human leukocyte antigen (HLA), immunogenetics, Working Committee leadership is listed in Appendix C5. 1.3.1.1 Working Committee Chairs Working Committee Chairs are appointed by the Advisory Committee to non-renewable fiveyear terms. Appointments are made each fall, with terms commencing on March 1 of the following year. Terms are staggered to facilitate succession and maintain continuity. Individuals may serve as Chair more than once but not consecutively for the same committee. The Nominating Committee polls the CIBMTR membership for potential Chair candidates for open Chair positions. Current Chairs participate in the nomination process for replacement positions and give special consideration to promising junior investigators, thus promoting ongoing leadership for the work of the CIBMTR. Working Committee Chairs provide subject matter expertise in autologous and allogeneic transplantation as well as understanding of CIBMTR organization and procedures. They must be members of CIBMTR centers that submit CRFs and that are compliant with Continuous Process Improvement (CPI) standards for data submission, unless an exception is granted by the Advisory Committee. Chairs are occasionally selected from outside these guidelines for their specific Page | 6 CIBMTR 2015 Annual Report 1.0 WHO WE ARE scientific expertise, for example, a scientist who directs a histocompatibility laboratory, apheresis center, or donor registry, who is committed to the CIBMTR and to the field of HCT. Chairs monitor and facilitate the progress of studies in their Working Committee’s portfolio. They communicate with Principal Investigators to address barriers and / or delays in study completion and participate in weekly CIBMTR Coordinating Center study critiques when studies in their portfolios are being discussed. In addition to chairing annual Working Committee meetings, Chairs meet by teleconference every four to six weeks with their committee’s Scientific Director and biostatisticians to review the progress of study proposals and ongoing studies. Chairs lead the annual Working Committee meeting and, using input from that meeting, prioritize studies and set the research agenda for the following year. Page | 7 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.0 WHAT WE DO The CIBMTR collects data for approximately 21,000 new transplant patients annually as well as a continually increasing volume of follow-up data on previously reported recipients and donors. Submission of outcomes data is mandatory for allogeneic transplants in the US and those outside the US that use a US donor; all other submissions are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and about 80% of US autologous transplants are reported. The CIBMTR Research Database contains information on >425,000 patients. The distribution of patients in the database is displayed by graft type in Figure and Table 2.1 and by disease in Figure and Table 2.2.Figure 2.3 shows the continued growth in the number of patients registered with the CIBMTR. Publications There were 76 CIBMTR publications in 2015. As of December 31, an additional 17 manuscripts were submitted and are under review. A complete list of publications is provided in Appendix D. Presentations In 2015, CIBMTR study investigators presented 65 abstracts (41 oral and 24 poster) at national and international conferences, a 50% increase from last year. Presentations included 25 (17 oral and 8 poster) at the American Society for Hematology (ASH) Annual Meeting and 17 (13 oral and 4 poster) at the BMT Tandem Meetings. A complete list of presentations is provided in Appendix E. Figure and Table 2.1. Distribution of Patients in the CIBMTR Research Database by Graft Source Graft Source Bone Marrow Peripheral Blood Cord Blood TOTAL Allogeneic Autologous TED CRF TED CRF 49,509 58,935 9,885 5,887 73,780 33,848 148,822 34,858 5,408 8,721 136 8 128,697 101,504 158,843 40,753 Page | 8 CIBMTR 2015 Annual Report 2.0 WHAT WE DO Figure and Table 2.2. Distribution of Patients in the CIBMTR Research Database by Disease Disease Acute myelogenous leukemia Acute lymphoblastic leukemia Chronic myelogenous leukemia MDS / myeloproliferative syndromes Lymphoma Plasma cell disorders Other malignant diseases1 Severe aplastic anemia Inherited erythrocyte disorders2 Immune deficiency disorders TOTAL Allogeneic Autologous TED CRF TED CRF 40,448 28,231 5,721 2,379 21,096 16,616 1,089 469 13,914 15,163 414 282 12,805 10,635 182 86 15,793 8,009 64,110 13,514 4,141 2,169 57,615 11,594 6,039 3,794 28,917 12,247 5,981 6,753 14 8 6,085 7,133 781 174 2,395 3,001 0 0 128,697 101,504 158,843 40,753 1. Includes other leukemia (allogeneic, n=8,279; autologous, n=910), solid tumors (allogeneic, n=1,554; autologous, n=40,254) 2. Includes inherited erythrocyte disorders (allogeneic, n=8,602; autologous, n=9), inherited platelet disorder (allogeneic, n=186; autologous, n=4), inborn errors of metabolism (allogeneic, n=2,499; autologous, n=0), histiocytic disorders (allogeneic, n=1,413; autologous, n=16), autoimmune disease (allogeneic, n=125; autologous, n=555), other disorders (allogeneic, n=393; autologous, n=371) Page | 9 CIBMTR 2015 Annual Report 2.0 WHAT WE DO Figure 2.3. Continued Growth in the Number of Patients Registered with the CIBMTR Page | 10 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.1 CLINICAL OUTCOMES RESEARCH PROGRAM Clinical outcomes research using the CIBMTR Research Database is a core activity of the organization. These studies address a wide range of issues, focusing on questions that are difficult or impossible to address in singlecenter studies or randomized trials because diseases treated with HCT are uncommon, single centers treat few patients with a given disorder, and not all important questions are amenable to a randomized research design. 2.1.1 Scientific Working Committees Program Activities The 15 Scientific Working Committees oversee most of the CIBMTR’s clinical outcomes research. There are currently 176 studies in progress (Table 2.4). These studies and those recently published are detailed in the 2015 Report on Working Committee Studies. For the 2015 BMT Tandem Meetings, the Working Committees reviewed 157 new study proposals, 73 of which were presented and 35 of which were approved. The prioritization and selection process ensures that the most important issues can be addressed in a timely manner. Publications In 2015, Working Committee study investigators published 38 manuscripts in peer-reviewed journals, about 50% of the total number of CIBMTR publications this year (Figure 2.5). As of December 31, an additional 15 manuscripts were submitted for publication and are under review. A complete list of Working Committee publications is provided in Appendix D1. Key Working Committee Publications this Year Chen Y-B et al. Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous HCT. Biology of Blood and Marrow Transplantation. 2015 Jun 1; 21(6):1046-1053. Epub 2015 Feb 13. PMC4426014. Sorror ML et al. Prospective validation of the predictive power of the HCT comorbidity index: a CIBMTR® study. Biology of Blood and Marrow Transplantation. 2015 Aug 1; 21(8):14791487. Epub 2015 Apr 7. PMC4512746. Orchard PJ et al. HCT for infantile osteopetrosis. Blood. 2015 Jul 9; 126(2):270-276. Epub 2015 May 26. PMC4497967. Ciurea SO et al. Haploidentical transplant with post-transplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia. Blood. Epub 2015 Jun 30. Kollman C et al. The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy. Blood. Epub 2015 Nov 2. Presentations In 2015, Working Committee study investigators presented 38 abstracts (26 oral and 12 poster), including 19 (11 oral and 8 poster) at the ASH Annual Meeting and 10 (9 oral and 1 poster) at the BMT Tandem Meetings. A complete list of CIBMTR presentations is provided in Appendix E. Page | 11 CIBMTR 2015 Annual Report 2.0 WHAT WE DO Table 2.4. 2015 Working Committee Studies Working Committee Studies in Progress Publications Presentations Acute Leukemia 13 3 4 Autoimmune Diseases and Cellular Therapies 6 0 0 Chronic Leukemia 12 0 3 Donor Health and Safety 11 2 6 Graft Sources and Manipulation 5 3 0 Graft-versus-Host Disease 10 2 2 Health Services and International Studies 12 2 4 Immunobiology 43 6 5 Infection and Immune Reconstitution 9 1 0 Late Effects and Quality of Life 11 2 2 Lymphoma 6 6 7 Pediatric Cancer 4 2 0 Plasma Cell Disorders and Adult Solid Tumors 12 2 2 Primary Immune Deficiencies, Inborn Errors of Metabolism, and Other Non-Malignant Marrow Disorders 10 3 0 Regimen-Related Toxicity and Supportive Care 12 6 3 TOTAL 176 38* 38 *Two studies are joint studies between two committees. Page | 12 CIBMTR 2015 Annual Report 2.0 WHAT WE DO Funding Successful Working Committee Study Proposals are Support for the Working Committees is primarily provided by the National Institutes of Health (NIH) grant # U24CA076518 from the National Cancer Institute (NCI); National Heart, Lung, and Blood Institute (NHLBI); and National Institute for Allergy and Infectious Disease (NIAID). Feasible. Utilize data available in the CIBMTR Research Database. Unique. Fill a gap not addressed by current studies or publications. How to Get Involved Important. Impact the field by improving Working Committees are collaborative in nature, and all interested individuals are encouraged to participate. Please feel free to attend annual in-person meetings of the Working Committees at the BMT Tandem Meetings in February. Additionally, anyone willing to follow the study development and management process (Appendix F) is eligible to propose a study to the Working Committees (Figure 2.6). transplant procedures or results. See the CIBMTR How to Propose a Study webpage for additional guidelines and advice as well as the Study Proposal Outline. Figure 2.5. 2015 Publications by Program Coordinating Center (14) Bioinformatics (9) Clinical Outcomes Working Committees (38) BMT CTN (8) Other (9) Statistical Methodology (5) Health Services (4) Page | 13 CIBMTR 2015 Annual Report 2.0 WHAT WE DO Figure 2.6. Working Committee Study Proposal Review Process Submission Initial Review • By November 15, study investigator submits proposal to the CIBMTR Coordinating Center for consideration at the next BMT Tandem Meetings. • Working Committee Leaderships reviews for feasibility with CIBMTR data, potential conflict with active studies, scientific merit, and ability to complete the study in a timely fashion. Researchers with similar concepts may be advised to combine their proposals. • If Working Committee Leadership clears the proposal to move forward, the MSlevel Statistician contacts the study investigator and prepares a table of Preliminary characteristics of patient data based on the population defined in the proposal. Assessment Presentation Voting Final Approval Notification • Study investigator presents the proposal at the Working Committee meeting at the February BMT Tandem meetings. • Working Committee members vote for each proposal, assigning a scientific impact score to each. • Working Committee Leadership utilizes member feedback in determining which proposals to pursue. Advisory Committee approves the CIBMTR research agenda. • Working Committee Leadership contacts study investigator to notify of study approval / rejection by the end of April. Page | 14 CIBMTR 2015 Annual Report 2.1.2 Cellular Therapies and Non-Transplant Therapy Research Initiatives With the consensus and support of its Advisory Committee, the CIBMTR is committed to collecting data on cellular therapies and nontransplanted patient populations. The CIBMTR amended its registration of cases to accommodate data collection for cellular and other therapies that may not involve transplantation. Additionally, the CIBMTR is working collaboratively with the Primary Immune Deficiency Disease Consortium to add transplant outcomes data to data on nontransplant therapy collected by the Consortium. Cellular Therapies In 2015, the CIBMTR initiated a Cellular Therapies Task Force charged with determining appropriate content and structure for cellular therapies data collection. The CIBMTR also hosted a Cellular Therapies Forum in October 2015 to discuss how to further develop and provide recommendations for a cellular therapy registry. Participants included physicians, scientists, manufacturers, and government representatives. Cellular Therapies Forum Discussion Topics Cell therapy for treatment of viral infection CAR-T cells for malignancies Manufacturing models Regulatory framework and long-term outcomes reporting NCI Cancer Immunotherapy Trials Network 2.0 WHAT WE DO Myelodysplastic Syndrome The CIBMTR collects data on patients receiving non-HCT therapy for myelodysplastic syndrome (MDS) to compare with an ongoing study of HCT outcomes. By combining its resources with those of the BMT CTN (Section 2.3.1), the CIBMTR is able to do this in a costeffective manner. Through November 2015, 34 centers have been activated and 141 patients enrolled on this non-HCT therapy study. The HCT outcomes study was launched in 2010 because many elderly patients with MDS were denied access to HCT therapy in the US due to lack of Medicare insurance coverage by the Centers for Medicare and Medicaid Services (CMS). To help secure Medicare coverage for these patients, the CIBMTR, NMDP/Be The Match, American Society for Blood and Marrow Transplantation (ASBMT), and other organizations partnered with CMS to develop a Coverage with Evidence Development (CED) study, using data in the CIBMTR Research Database that are collected to fulfill SCTOD requirements. The CED approach allows CMS to provide coverage for procedures and to advocate for clinical studies that inform policy decisions. The CMS-approved study now has >2,300 patients enrolled. Assessment of HCT in Medicare Beneficiaries with MDS 127 centers 1,294 patients ≥65 years old 815 patients 55-64 years old 213 patients <54 years old Proposed CIBMTR Cellular Therapy Registry Page | 15 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.2 IMMUNOBIOLOGY RESEARCH PROGRAM The CIBMTR maintains a Research Repository of paired tissue samples from donors and recipients, both unrelated and related. The Immunobiology Research group manages the Research Repository inventory and immunogenetic testing programs that add critical HLA and killer-cell immunoglobulin-like receptors (KIR) data for use in CIBMTR clinical outcomes studies. The CIBMTR leverages the NMDP/Be The Match’s investment in the Unrelated Donor Research Repository with the NIH’s investment in the CIBMTR Research Database. Linking outcomes data to immunologic data available in the Research Repository supports studies that include genetic and immunobiologic data and clinical phenotype data. The Related Donor Research Repository, supported by the Health Resources and Services Administration (HRSA), is a unique opportunity to enhance immunobiologic research. Related donor and recipient samples are better matched than unrelated recipients for HLA, a measure of immunological compatibility, thus reducing the confounding effects of HLA disparity in clinical research. The combination of the Unrelated Donor and Related Donor Research Repositories facilitates an organized approach to studying transplant biology across the full spectrum of allogeneic HCT. Program Activities In 2015, 175 centers (128 transplant centers, 32 donor centers, and 15 cord blood banks) provided samples to the Research Repository. The Immunobiology Research group enhanced the Research Repository inventory and Immunogenetic Database this year by completing high resolution HLA and presence / absence KIR typing on 226 related and 2,772 unrelated HCT donor / cord and recipient Research Repository 2,043,345 aliquots 18,901 cell lines 59,375 samples from unrelated donors and 5,128 from related donors 57,018 samples from unrelated recipients and 5,377 from related recipients 10,566 samples from unrelated cord blood units Samples from complete pairs: 33,919 from complete unrelated adult donor-recipient pairs 4,545 from complete related donorrecipient pairs 3,579 from unrelated cord-recipient pairs pairs, bringing the total to >17,500 unrelated donor / cord and recipient pairs that have been retrospectively high resolution typed for HLA-A, -B, -C, -DRB1 and -DQB1; >70% include -DPB1, and >10,000 include KIR. The Immunobiology Research group consolidated all collections (unrelated, related, and clinical trial) under one inventory management system in 2015 and incorporated their information into the Integrated Data Warehouse to facilitate linkage of samples to clinical data. Additionally, the group distributed 8,770 research samples in support of Working Committee studies this year. Page | 16 CIBMTR 2015 Annual Report Publications In 2015, Immunobiology Working Committee study investigators published 6 manuscripts, 4 of which utilized research samples and immunobiology clinical outcomes data. The Immunobiology Research group also supports other investigators’ publications by providing research samples. 9 manuscripts published this year by investigators from other Working Committees and the BMT CTN utilized samples from the Research Repository. A complete list of Working Committee publications is provided in Appendix D1. Presentations In 2015, 5 Immunobiology Working Committee study investigators presented at national and international conferences, including 2 oral abstracts at the European Group for Blood and Marrow Transplantation (EBMT) Annual Meeting and 2 posters at the ASH Annual Meeting. A complete list of CIBMTR presentations is provided in Appendix E. Funding Support for the Immunobiology Research Program is primarily provided by the Office of Naval Research grant # N00014-14-1-0848, NIH grant # U24CA076518, and HRSA contract # HHSH250201200016C. The Immunobiology Research Program offers limited research funds supporting immunobiology research studies. The grants are intended to subsidize lab tests, sample collection, or costs associated with the use of research samples. These grants are available to approved CIBMTR studies that support organizational research priorities. For additional information, visit the CIBMTR Grants for Immunobiology Research webpage. 2.0 WHAT WE DO Key Immunobiology Publications this Year Gadalla SM et al. Association between donor leukocyte telomere length and survival after unrelated allogeneic HCT for severe aplastic anemia. Journal of the American Medical Association. 2015 Feb 10; 313(6):594-602. PMC4388056. Verneris MR et al. HLA mismatch is associated with worse outcomes after unrelated donor reduced-intensity conditioning HCT: an analysis from the CIBMTR. Biology of Blood and Marrow Transplantation. 2015 Oct 1; 21(10):17831789. Epub 2015 Jun 6. PMC4568127. Petersdorf EW et al. High HLA-DP expression and graft-versus-host disease. New England Journal of Medicine. 2015 Aug 13; 373(7):599-609. PMC4560117. How to Get Involved All interested parties may attend the annual in-person meeting of the Immunobiology Working Committee at the BMT Tandem Meetings in February. Additionally, the Immunobiology Working Committee encourages highly translational, hypothesisdriven proposals through the Working Committee Study Proposal Review Process (Figure 2.6). Page | 17 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.3 CLINICAL TRIALS SUPPORT PROGRAM Using data from observational studies to support decisions regarding design of prospective clinical trials and / or amendments of such trials, the CIBMTR Research Database provides an important resource to the Clinical Trials Support Program. This program consists of the BMT CTN and RCI BMT, which conduct multicenter clinical trials. Data from the Research Database are used to design, monitor, and analyze these trials. CIBMTR Coordinating Center Support of Clinical Trials Trial Planning. Determine patient populations available for trials as well as appropriate inclusion criteria and outcome estimates, and identify transplant centers likely to accrue. Data Collection. Share data to reduce reporting for transplant centers, and share data collection forms and database structures developed by transplant experts. Statistical Consultation. Provide expert statistical review of protocols. Real-Time Accrual Assessment. Review data to evaluate center and patient characteristics of enrolled and non-enrolled patients to address accrual barriers. Trial Interpretation. Evaluate results of clinical trials, including through the provision of matched controls. Long-Term Follow-Up Data. Capture follow-up data for long-term or secondary analyses, resulting in considerable costsavings. 2.3.1 Blood and Marrow Transplant Clinical Trials Network The BMT CTN, sponsored by NHLBI and NCI, is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation, a contract research organization based in Rockville, MD. Program Activities The BMT CTN has launched 37 trials (3 this year), and completed accrual for 28 of these trials (2 this year). The Network has accrued >8,500 patients to its trials from >100 centers, including >1,100 this year. Among trials currently open for enrollment, the BMT CTN achieved an overall accrual rate in 2015 that is approximately 125% of projections. The Network has established a Research Sample Repository that currently includes >350,000 biospecimens. Additionally, the BMT CTN has conducted 48 ancillary and correlative studies. More detail regarding Network activities and protocols is provided in the annual progress report on the BMT CTN website. A list of Network trials open for enrollment is provided in Appendix G1. Publications In 2015, BMT CTN study investigators published 8 manuscripts, 2 of which were primary results papers. These bring the total number of Network publications to 57, including 16 primary results papers. A complete list of 2015 BMT CTN publications is provided in Appendix D2. Page | 18 CIBMTR 2015 Annual Report Key BMT CTN Publications this Year MacMillan ML et al. A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality. Biology of Blood and Marrow Transplantation. 2015 Apr 1; 21(4):761-767. Epub 2015 Jan 10. PMC4359643. Khera N et al. Comparison of characteristics and outcomes of trial participants and nonparticipants: example of BMT CTN 0201 trial. Biology of Blood and Marrow Transplantation. 2015 Oct 1; 21(10):18151822. Epub 2015 Jun 11. PMC4568172. Anderlini P et al. Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose deescalation study. Lancet Haematology. 2015 Sep 1; 2(9):367-375. Epub 2015 Sep 8. Devine SM et al. Phase II study of allogeneic transplantation for older patients with acute myeloid leukemia in first complete remission using a reducedintensity conditioning regimen: results from CALGB 100103 / BMT CTN 0502. Journal of Clinical Oncology. Epub 2015 Nov 2. 2.0 WHAT WE DO Presentations BMT CTN study investigators presented 11 abstracts (8 oral and 3 poster) at national and international conferences in 2015, two of which were highlighted at the ASH Annual Meeting, one as a Late Breaking Abstract and the other as a 2016 Highlight of ASH. These bring the total number of Network presentations to 59. A complete list of 2015 CIBMTR presentations is provided in Appendix E. Funding Support for the BMT CTN Data and Coordinating Center is provided by the NIH grant # U10HL069294 from the NHLBI and NCI. How to Get Involved The Network is committed to widespread participation in its trials. If you would like to serve as an Affiliate Center, visit the BMT CTN website for more information. Additionally, you may act as a Center Principal Investigator or champion a trial to increase patient accrual at your Center, serve on a Protocol Team or an Endpoint Review Committee, or act as a Medical Monitor. You may also propose an ancillary study, which uses data, biospecimens, and / or analyses outside the specific objectives of a primary BMT CTN study. Page | 19 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.3.2 Resource for Clinical Investigations in Blood and Marrow Transplantation The RCI BMT provides researchers in the field of HCT with infrastructure and expertise in HCT clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting with groups such as the BMT CTN or the national cancer cooperative groups. Program Activities The RCI BMT has launched 15 trials, including 3 this year. In 2015, the RCI BMT accrued just under 3,000 patients, bringing the total number of accrued patients to approximately 28,000, of which >21,000 were enrolled in a cohort study examining long-term outcomes of unrelated donors. The RCI BMT is currently managing 2 Food and Drug Administration (FDA) investigational new drug (IND) protocols for NMDP/Be The Match Operations. Peripheral Blood Stem Cell (PBSC) Procurement accrued >2,100 patients this year, and Cord Blood Access accrued >500. Additionally, the RCI BMT supported 5 studies involving unrelated donor data or sample collection for investigators. In 2015, the RCI BMT launched two new interconnected software systems to enhance data collection and study management. These systems will increase efficiency, allowing the RCI BMT to effectively support a wide array of clinical trials and research studies. A complete list of RCI BMT trials is provided in Appendix G2. Presentations In 2015, RCI BMT study investigators presented 4 oral abstracts at national and international conferences. A complete list of CIBMTR presentations is provided in Appendix E. Survey Research Group The Survey Research Group is a team within the RCI BMT created to assist HCT researchers in developing and conducting research involving questionnaires, direct subject interviews, and patient reported outcomes. The group is responsible for collecting high quality, scientifically valid data from donors, patients, and their families. The Survey Research Group utilizes standardized and semi-structured telephone interviews as well as self-administered questionnaires. In 2015, the Survey Research Group supported 8 active studies and participated in the development of 1 upcoming study. Funding Support for the RCI BMT is primarily provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID; NMDP/Be The Match; and corporate and private sponsors of specific studies. The RCI BMT team can work with study investigators to seek funding from a variety of sources, including government agencies, foundations, pharmaceutical companies, and private corporations. How to Get Involved Study investigators may solicit clinical trials services from the RCI BMT, including assistance with funding proposals; protocol development and approvals; management of study conduct; data auditing, management, and analysis; and financial administration. Study investigators may also contract for specific services as needed, such as support with surveys, site selection and management, sample management, and more. For additional information, visit the RCI BMT webpage. Page | 20 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.4 HEALTH SERVICES RESEARCH PROGRAM Health services research is the multidisciplinary field of scientific investigation that studies how social factors, financial systems, organizational structures and processes, technology, and behavior affect treatment outcomes, quality, and cost. The CIBMTR conducts research through the Health Services Research Program in collaboration with NMDP/Be The Match’s Patient and Health Professional Services. Research activities complement those of the CIBMTR’s Health Services and International Studies Working Committee. Health Services Focus Areas Research Health Policy System Capacity Initiative Program Evaluation Clinical Trial Support Training, Education, and Consultation Program Activities Research. The Health Services Research Program currently has 9 studies in progress. In 2015, the program completed analysis for 3 research studies. Health Policy. In collaboration with NMDP/Be The Match’s Payer Policy department, the Health Services Research Program conducted a study, Financial barriers to HCT: the transplant center perspective, to examine the impact of the Patient Protection and Affordable Care Act on access to HCT and treatment decisionmaking. Select Health Services Research Studies in Progress Individualized care plans for HCT survivors Cost-effectiveness of HCT vs. alternative therapy for acute myeloid leukemia (AML) in patients aged 60-70 years Analysis of reimbursement for HCT in older patients HCT multidisciplinary care teams: burnout, moral distress, and career satisfaction Payer-partnered approach to community-based referral for HCT Easy-to-read informed consent forms for HCT clinical trials (BMT CTN 1205) System Capacity Initiative. The goal of the System Capacity Initiative is to determine how the US healthcare system can accommodate the growing number of patients in need of HCT. In 2015, the Health Services Research Program continued to support initiativerelated efforts, including the study on HCT workforce burnout. Program Evaluation. The Health Services Research Program conducted >30 evaluations for the HRSA Office of Patient Advocacy / Single Point of Contact contract and NMDP/Be The Match programs in 2015, including a needs assessment for limited English proficiency patients. Page | 21 CIBMTR 2015 Annual Report Clinical Trial Support. A collaboration between the Health Services Research Program and BMT CTN, Easy-to-read informed consent forms for HCT clinical trials evaluates the effectiveness of a novel consent form and describes barriers to implementation. The program also provided technical writing support and developed 3 easy-to-read consent / assent forms and 6 patient information sheets for BMT CTN protocols. Training, Education, and Consultation. In 2015, the Health Services Research Program established the HCT Health Economics Interest Group, which held its first meeting at the 2015 BMT Tandem Meetings. More detail regarding program activities is provided in the Health Services Research Annual Report. Publications In 2015, Health Services Research study investigators published 4 manuscripts in peerreviewed journals. A complete list of program publications is provided in Appendix D3. Presentations Through the HRSA Office of Patient Advocacy / Single Point of Contact contract, the Health Services Research Program is charged with disseminating findings to all stakeholder groups. In 2015, program investigators presented 3 oral abstracts at the ASH Annual Meeting. A complete list of research-focused CIBMTR presentations is provided in Appendix E. How to Get Involved For more information about the Health Services Research Program and / or the HCT Health Economics Interest Group, contact Ellen Denzen, MS, Senior Manager, at edenzen@nmdp.org or 612.884.8562. 2.0 WHAT WE DO Health Services Research Publications this Year Besse KL et al. Estimating demand and unmet need for allogeneic HCT in the US using geographic information systems. Journal of Oncology Practice. 11(2):e120e130. Epub 2015 Mar 1. PMC4371120. Majhail NS et al. National survey of HCT center personnel, infrastructure and models of care delivery. Biology of Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1308-1314. Epub 2015 Mar 31. PMC4466059. Clauser SB et al. Patient centeredness and engagement in quality-of-care oncology research. Journal of Oncology Practice. 2015 May 1; 11(3):176-179. Epub 2015 Apr 7. Preussler JM et al. Patient housing barriers to HCT: results from a mixed-methods study of transplant center social workers. Supportive Care in Cancer. Epub 2015 Aug 15. Funding Support for the Health Services Research Program is provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID. Individual studies are funded via a variety of mechanisms. Individualized care plans for HCT survivors is supported by the Patient Centered Outcomes Research Institute award # CD-1212-4062; Easy-to-read informed consent forms for HCT clinical trials is supported by the NHLBI grant # U10HL069294-12S1, and A payerpartnered approach to community-based referral for HCT is supported by the grant # 11762021 from the National Comprehensive Care Network / Pfizer. Page | 22 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.5 BIOINFORMATICS RESEARCH PROGRAM The Bioinformatics Research Program provides expertise in, and conducts research on, translational and operational bioinformatics. Program Activities Current Bioinformatics Research Goals Develop pipelines to analyze Next Generation Sequencing typing data, including full-gene HLA, KIR, and genomewide sequencing, to refine our understanding of genetic matching Investigate the role of genetic ancestry in transplantation, including the best way to match individuals with multiple races in their family tree Develop data standards and tools for making immunogenetic data portable for research and clinical use Investigate HLA data from other countries to better understand global frequencies and improve matching Develop methods for HLA association studies Publications In 2015, Bioinformatics study investigators published 9 manuscripts in peer-reviewed journals. A complete list of program publications is provided in Appendix D4. Key Bioinformatics Publications this Year Slater N et al. Power laws for heavy-tailed distributions: modeling allele and haplotype diversity for the NMDP. PLoS Computational Biology. 11(4):e1004204. Epub 2015 Apr 22. PMC4406525. Besse K et al. On modeling HLA-identical sibling match probability for alloHCT: estimating the need for an unrelated donor source. Biology of Blood and Marrow Transplantation. Epub 2015 Sep 5. Buck K et al. 7/8 and 9/10 or better high resolution match rate for the Be The Match® Unrelated Donor Registry. Biology of Blood and Marrow Transplantation. Epub 2015 Dec 24. Funding Support for the Bioinformatics Research Program is primarily provided by the grant # N00014-14-1-0848 from the Office of Naval Research as well as several grants from the NIH. How to Get Involved For more information about the Bioinformatics Research Program, visit the NMDP/Be The Match Clinical Bioinformatics webpage or contact Martin Maiers, Director, at mmaiers@nmdp.org or 612.627.5892. Presentations Bioinformatics study investigators presented 11 abstracts (5 oral and 6 poster) at national and international conferences in 2015. A complete list of CIBMTR presentations is provided in Appendix E. Page | 23 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.6 STATISTICAL METHODOLOGY RESEARCH PROGRAM The CIBMTR has enjoyed a positive, collaborative association with the Division of Biostatistics in the MCW Institute for Health and Society since 1980, an association that is a distinctive asset and crucial to the success of CIBMTR research. This long-standing relationship has many benefits. Biostatisticians ensure the statistical integrity of CIBMTR scientific activities, contribute to results in articles on HCT-related statistical issues for clinical audiences, and support Working Committee study investigators in developing scientific study protocols using CIBMTR data. CIBMTR biostatisticians have pioneered novel methodologic approaches to analyzing HCT data. Program Activities HCT is a complex process with multiple competing risks and dramatic changes in the risks of specific events over time. The CIBMTR has developed and evaluated the statistical models used in HCT research and helped guide the research community in appropriate application and interpretation of these sophisticated models. Statistical Methodology Research Goals Develop new statistical models Compare new statistical models with existing solutions using the CIBMTR Research Database Publications In 2015, PhD-level biostatisticians in the Statistical Methodology Research Program published 4 peer-reviewed manuscripts and 1 editorial. A complete list of program publications is provided in Appendix D5. Key Statistical Methodology Publications this Year Wang T et al. A re-formulation of generalized linear mixed models to fit family data in genetic association studies. Frontiers in Genetics. 6(120):1-10. Epub 2015 Mar 31. PMC4379931. Eriksson F et al. The proportional odds cumulative incidence model for competing risks. Biometrics. 2015 Sep 1; 71(3)687-695. Epub 2015 May 26. PMC4608382. Brazauskas R, Logan BR. Observational studies: matching or regression? Biology of Blood and Marrow Transplantation. Epub 2015 Dec 19. Funding Support for the Statistical Methodology Research Program is primarily provided by the NIH grant # U24CA076518 from the NCI, NHLBI, and NIAID and the HRSA contract # HHSH250201200016C. How to Get Involved During the BMT Tandem Meetings in February, PhD-level biostatisticians plan and present educational sessions related to statistical design and analysis, and they provide 1:1 statistical consultation to researchers writing proposals or developing protocols for CIBMTR studies. Any interested individual may participate in these sessions. Additionally, the MCW Division of Biostatistics presents a lecture series and a seminar series throughout the year in Milwaukee. Page | 24 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.7 STEM CELL THERAPEUTIC OUTCOMES DATABASE (SCTOD) The CIBMTR administers the SCTOD for the HRSA-sponsored C.W. Bill Young Cell Transplantation Program, established by the Stem Cell Therapeutic and Research Act of 2005. Continued support for the SCTOD is provided through the Stem Cell Therapeutic and Research Reauthorization Act of 2010. C.W. Bill Young Cell Transplantation Program Goals Fulfilled by the SCTOD Collect, analyze, and report outcomes data for all allogeneic transplants and other therapeutic uses of blood stem cells Publicize information about HCT to patients, families, health care professionals, and the public Define better processes for identifying unrelated matched marrow donors, PBSC donors, and cord blood units through one electronic system Increase availability of unrelated adult volunteer donors and cord blood units Expand research to improve patient outcomes Program Activities Annually, the CIBMTR publishes HCT volumes and performance data by transplant center and provides public access to this information via the HRSA Blood Cell Transplant website. Center-Specific Volumes and Survival Analysis. As part of the contract to operate the SCTOD, the CIBMTR provides the annual volume of transplants performed at each center and performs a center-specific survival analysis comparing the one-year survival rates among US centers. The report assesses transplants from both related and unrelated SCTOD Contract Requirements Collect HCT outcomes data for: • All allogeneic HCTs performed in the US using related or unrelated donors • All allogeneic HCTs worldwide that use grafts procured through the C.W. Bill Young Cell Transplantation Program • Clinical applications of hematopoietic stem cells other than hematopoietic cell recovery Use the data collected for the SCTOD to evaluate the performance of transplant centers Provide specific SCTOD data to the public Collect a basic set of data for analyses of program use, center-specific outcomes, donor registry, cord blood inventory size, and patient access to HCT Establish a Related Donor-Recipient Research Sample Repository (Section 2.2) donors. The most recent report was finalized in September 2015 and contains information on all first allogeneic transplants performed in US centers from January 1, 2011, through December 31, 2013. Center Outcomes Forums. The CIBMTR has conducted 4 Center Outcomes Forums to engage relevant stakeholders in the centerspecific outcomes reporting process. The most recent meeting was held in June 2014 and generated recommendations to improve risk adjustment, enhance future collection of relevant data elements, and develop tools to enhance centers’ quality improvement efforts. Page | 25 CIBMTR 2015 Annual Report Quality of Life Assessments. In 2011, the CIBMTR launched a pilot program at 5 adult and 3 pediatric transplant centers. The principal goal was to test center-based recruitment of patients to a quality-of-life data collection program, followed by communication between the CIBMTR and the patient to collect longitudinal quality-of-life information. This pilot program is unique in that the CIBMTR collects data directly from patients after HCT. Accrual closed in 2013 with 390 patients enrolled. Data analysis is in progress. Cellular Therapies for Regenerative Medicine (CTRM). The CTRM data repository tracks novel uses of blood stem cells. The SCTOD contract mandates data collection on uses of cells found in bone marrow, peripheral blood, and umbilical cord blood for alternative therapeutic applications, including regenerative medicine. The CTRM data repository captures uses of cells for the treatment of diseases without the intention of replacing the recipient’s hematopoietic function. These therapies include, but are not limited to, treatment of malignancies as well as infectious, cardiovascular, rheumatologic, neurologic, musculoskeletal, and endocrinologic diseases with the intent to improve organ function. As of November 30, the CTRM data repository includes data for 983 patients. The CIBMTR anticipates the expansion of the cellular therapy field with the use of not only hematopoietic derived cells but also cells from other tissues; therefore, it is expanding the capability and flexibility of data collection in this rapidly changing area. In October, the CIBMTR held a Cellular Therapy Forum (Section 2.1.2) to discuss how to further develop its cellular therapy registry. 2.0 WHAT WE DO Study Summaries for Patients. In conjunction with Be the Match’s Patient and Health Professional Services and the Consumer Advocacy Committee, the CIBMTR publishes lay summaries of CIBMTR publications for patients and their loved ones. In 2015, the CIBMTR published 12 patient-friendly research summaries. Funding Support for the SCTOD is provided by the HRSA contract # HHSH250201200016C. How to Get Involved All US centers performing allogeneic HCTs provide data to the CIBMTR for the SCTOD. These data are used to generate reports, which are distributed to transplant center medical directors and posted on the HRSA Blood Cell Transplant website. Publicly Available Reports developed by the CIBMTR for the SCTOD Transplant Outcomes • US Patient Survival Report • US Transplant Data by Center Report • US Transplant Data by Disease Report Transplant Activity Report These reports are available on the HRSA Blood Cell Transplant website Page | 26 CIBMTR 2015 Annual Report 2.0 WHAT WE DO 2.8 CORPORATE PROGRAM The CIBMTR Corporate Program provides opportunities for industry collaborators to access CIBMTR data and statistical support to address questions specific to their business needs through Corporate Membership as well as Corporate Studies and Projects. Corporate Membership. The CIBMTR Corporate Membership program provides a variety of resource materials to corporations needing access to the most current and comprehensive data on HCT. These materials are useful for Marketing Managers, Medical Directors, Research Directors, Product Managers, Case Managers, and Transplant Coordinators. There are four Corporate Membership levels available, each described on the CIBMTR Corporate Membership Program webpage. Corporate Membership Benefits CIBMTR Report on Survival Statistics for BMT Center Volumes Dataset Worldwide CIBMTR Directory of BMT Physicians Corporate Studies and Projects. Corporate partners may contract with the CIBMTR to conduct a study, support a project involving more complex analyses, or license a specified data set. Organizations interested in funding a study, such as one comparing HCT with one or more non-HCT therapies, or using historical controls, can negotiate with the CIBMTR for data and / or expert statistical analyses. Program Activities The CIBMTR engaged in 8 studies with corporate partners in 2015. Currently 14 organizations participate in the CIBMTR Corporate Membership program, including 9 that joined this year. How to Get Involved If you would like to learn more about the CIBMTR Corporate Program, visit the CIBMTR Corporate Membership webpage or contact Sherry Fisher, Director of Advancement, at slfisher@mcw.edu or 414.805.0687. If you are a Corporate Member requesting analyses, please complete the Corporate Member Information Request Form. Reduced registration rates at CIBMTR meetings and educational forums, including the BMT Tandem Meetings Access to CIBMTR data and resources Page | 27 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.0 HOW WE SHARE KNOWLEDGE The CIBMTR is committed to sharing the data we collect as well as the information and knowledge produced from our data and our extensive collaborations with investigators in the HCT field. The CIBMTR shares its knowledge in different ways. To determine the best way to access specific types of CIBMTR knowledge, review Figure 3.1 and Tables 3.2-3.5. Figure 3.1. How to Access CIBMTR Knowledge Details provided in Tables 3.2-3.5. Page | 28 CIBMTR 2015 Annual Report Table 3.2. How to Access CIBMTR Information* Information GENERAL In addition to reviewing this report, access the Summary of Accomplishments on the Administrative and Progress Reports webpage. Read editions of the quarterly newsletter on the Newsletters webpage, and email cibmtr-news@mcw. edu to be added to the electronic distribution list. ACTIVITIES Review Section 2 or visit the What We Do webpage. Visit the Studies webpage, SCTOD webpage, or Corporate Support webpage to learn more about CIBMTR research programs, the SCTOD, or Corporate Program, respectively. PUBLICATIONS Review the CIBMTR’s >1,000 publications on the Publication List webpage. For lay summaries of selected CIBMTR publications, written specifically for patients and the lay public, visit the Study Summaries for Patients webpage. MEETINGS Visit the BMT Tandem Meetings webpage to view agendas, register, reserve housing, and submit abstracts. TRAINING Review Section 3.4 or visit the Training and Reference webpage to access the Center Reference Guide, Forms Instruction Manual, FormsNet and AGNIS trainings, and online courses. OTHER Email contactus@cibmtr.org. 3.0 HOW WE SHARE KNOWLEDGE Table 3.3 How to Access CIBMTR Data* Data TYPES Review the baseline and follow-up data available for recipients and donors on the Types of Data Available for Research or Request webpage. STANDARD REPORTS Access the Summary Slides, BMT Survival Statistics Report, Center Transplant Activity Report, Patient Transplant Outcomes Reports, and Center-Specific Survival Reports via Table 3.6 or on the Slides and Reports webpage. ELECTRONIC RETURN OF CENTER Utilize the Data Back to Centers application on the Portal site to download TED-level variables that have been validated and processed in the CIBMTR Research Database. RESEARCH STUDY Propose a study as explained on the How to Propose a Study webpage, or participate in one of the existing studies listed on the Working Committee Study Lists webpage. CORPORATE ANALYSIS Complete the Corporate Member Information Request Form. CUSTOM ANALYSIS Complete the Custom Information Request Form. OTHER Email inforequest@mcw.edu. *If you are unable to access items using the electronic links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). Page | 29 CIBMTR 2015 Annual Report Table 3.4 How to Access CIBMTR Tools* Tools DISEASE RISK INDEX ASSIGNMENT TOOL Access this tool on the DRI Assignment Tool webpage to categorize patients undergoing allogeneic HCT for hematologic malignancy by disease risk. PATIENT ONE-YEAR SURVIVAL CALCULATOR FOR ALLOGENEIC TRANSPLANTS Transplant Center Medical Directors may access this tool on the Portal site to predict one year survival for individual allogeneic HCT recipients. 3.0 HOW WE SHARE KNOWLEDGE Table 3.5 How to Access CIBMTR Biospecimens* Biospecimens SAMPLES TYPES AND INVENTORY Determine the >2 million samples available in the Research Repository via the Sample Types and Inventory Summary webpage. REQUESTING SAMPLES For studies that include recipient clinical outcome data, propose a study as explained on the How to Propose a Study webpage. For studies that do not include clinical outcome data, review the How to Request Samples from the Research Sample Repository webpage. OTHER Email research-repos@nmdp.org. *If you are unable to access items using the electronic links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). Page | 30 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Table 3.6. Standard Reports Published by the CIBMTR Month(s) Released Report Title Description Format Accessibility* CIBMTR Summary Slides Charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT; developed in conjunction with the BMT Tandem Meetings February PPT Published on the CIBMTR Summary Slides webpage CIBMTR Progress Report Information on the CIBMTR's goals and achievements as well as operational details on how the CIBMTR is funded, supported, promoted, and maintained February PDF Published on the CIBMTR Administrative and Progress Reports webpage CIBMTR Report of Survival Statistics for BMT Highly detailed report on survival statistics that describes use and outcome of autologous and allogeneic HCT in the >500 centers that have participated in the CIBMTR September Word CIBMTR Summary High level summary of CIBMTR fiscal year of Accomplishments accomplishments and high impact publications September PDF Published on the CIBMTR Administrative and Progress Reports webpage US Centers Annual Transplant Activity Report Dataset containing center-specific pretransplant patient-, disease-, and transplantrelated characteristics data for nearly all allogeneic and a majority of autologous HCTs performed in the US annually since 2008 September PDF Published on the HRSA Blood Cell Transplant website US Patient CenterSpecific Survival Report Comparison of observed to expected oneyear survival rates among centers in the C.W. Bill Young Cell Transplantation Program network; evaluates outcomes for transplants using both related and unrelated donors December Web Published on the Be The Match Transplant Center Directory webpage; available as a Word document upon request to inforequest@mcw.edu Via Corporate Membership Program (Section 2.8) or by request from physicians for making treatment decisions or clinical investigators planning clinical studies to inforequest@mcw.edu Page | 31 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Month(s) Released Report Title Description Format Accessibility* US Patient Transplant Outcomes Disseminated in 3 different reports: • US Patient Survival Report: 100-day, 1year, and 3-year survival rate estimates for US HCT recipients by disease and donor type • US Transplant Data by Center Report: Number of bone marrow and cord blood transplants performed at a specific transplant center • US Transplant Data by Disease Report: Number of bone marrow and cord blood transplants for a specific disease December Web Published on the HRSA Blood Cell Transplant website US Allogeneic Transplant Activity Report Report containing patient, disease, donor HLA match, donor age, and gender match information for allogeneic transplant activity in the US since 2010 January April July October PDF Via Corporate Membership Program (Section 2.8) CIBMTR Newsletter Articles regarding Working Committees, the SCTOD, data management and collection, and noteworthy events in the HCT community February May August November Web Published on the CIBMTR Newsletters webpage and distributed via email; contact cibmtr-news@mcw.edu to be added to the distribution list Study Summaries for Patients Summaries of CIBMTR research publications written for patients and others in the lay public Ongoing PDF Published on the CIBMTR Study Summaries webpage *If you are unable to access items using the electronic links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). Page | 32 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.1 INFORMATION REQUEST SERVICE The CIBMTR Information Request Service provides timely access to data on transplantation to patients, physicians, hospitals, pharmaceutical companies, insurance companies, and others involved in healthcare. Requests range from simple queries of patient, disease, and transplant frequencies to those with greater complexity involving specific data combinations and / or statistical analysis of outcomes. Potential Reasons for Information Requests Self-education and decision making Patient counseling or clinical decision making Presentation support Transplant center assessments Clinical trial planning Market assessments Coordinating Center staff members fulfill requests related to clinical decision making within three days and most other requests within three weeks. If a request will take more than an estimated four weeks to fulfill, a Coordinating Center staff member will contact the requestor to discuss an appropriate timeline. Table 3.7. Data Requests Addressed by the CIBMTR in 2015 Requestor Number of Requests Physician / Researcher 352 Patient or Relative 41 Pharmaceutical / Biotech Company 38 Market Research Firm 22 Student 6 Patient Advocacy Group 4 News Media 4 Federal Government Agency 1 Cord Blood Bank 1 TOTAL 469 How to Access For more information about requesting data from the Research Database, visit the CIBMTR How to Request Data webpage. If you would like a one-time, custom analysis, complete the Custom Information Request Form. If you have questions about requesting CIBMTR data, please contact inforequest@mcw.edu. Accomplishments In 2015, the CIBMTR fulfilled 469 requests for information and data (Table 3.7). Page | 33 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.2 INTERNET PRESENCE The CIBMTR Internet presence provides the scientific community and the public with access to HCT information. Current websites include general information about HCT and CIBMTR activities; training and support; a shared communications and collaborative environment for member centers; and secure web access to CIBMTR data for Working Committees, transplant centers, scientific investigators, and CIBMTR staff. 3.2.1 CIBMTR Public Website The CIBMTR public website (cibmtr.org) is unrestricted and provides information about the CIBMTR and its research. It supports the Working Committees and BMT CTN with information regarding proposal submission, access to a listing and summaries of all studies in process, and access to a summary of all CIBMTR publications. The website facilitates data and information requests, and it provides access to all current and past data collections forms, training manuals, and videos as well as other materials for both investigators and data professionals. The website information is, in part, supported by DISCO (Data and Information for Statistical Center Operations), an application which maintains data on >750 studies, >1,000 publications, and >1,800 authors and their institutions at time of publication. In 2015, the CIBMTR public website had approximately 418,000 unique page views. About CIBMTR Administrative and Progress Reports (663 unique page views in 2015) Provides access to the CIBMTR’s annual Progress Report, annual Summary of Accomplishments, and Manual of Operations. Studies Working Committee Studies Lists (4,835 unique page views in 2015) A summary of the planned, in-progress, and recently published clinical outcomes studies for each Working Committee. Meetings Annual BMT Tandem Meetings Materials (34,073 unique page views in 2015) Provides access to agendas, handouts, and educational materials from specific meetings at the BMT Tandem Meetings: Working Committee Meetings and Clinical Research Professionals / Data Management Conferences. Reference Center Summary Slides (10,654 unique page views in 2015) Includes charts and figures summarizing current uses and outcomes of allogeneic and autologous HCT. Web-based US Transplant Reports (31,964 unique page views in 2015) Directs users to the Be The Match US Center Listing Report and customizable reports of patient survival and transplant available through the HRSA Blood Cell Transplant website. Publication List (5,836 unique page views in 2015) Searchable descriptive list of >1,000 publications resulting from the use of CIBMTR data and statistical resources. Page | 34 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE Newsletter (3,199 unique page views in 2015) Published 3-4 times per year. Articles feature updates on Working Committees, the SCTOD, data management and collection, and noteworthy events in the HCT community. Data Collection Forms (47,303 unique page views in 2015) Provides access to current and retired versions of the forms used by the CIBMTR to collect standard data elements for all transplant recipients. Patient Resources (4,590 unique page views in 2015) Includes lay summaries of CIBMTR research articles as well as post-transplant care recommendations for adult and pediatric autologous and allogeneic HCT recipients to help patients and clinicians understand and plan for the specialized care of transplant recipients. 12 lay summaries were published in 2015. Training and Reference (68,004 unique page views in 2015) Provides access to a wide variety of CIBMTR data management training and reference materials. Statistical Resources (4,183 unique page views in 2015) Provides access to resources offered through the unique partnership between the CIBMTR and MCW Division of Biostatistics, including biostatistical publications, a series of statistical lectures targeted at basic and clinical investigators, and research tools, such as the Disease Risk Index Assignment Tool. Data Management Data Management Manual (38,071 unique page views in 2015) A comprehensive reference document for completing CIBMTR data collection forms. The manual also details reporting requirements, describes protocols and the consent process, and includes downloadable versions of report forms. 3.2.2 CIBMTR Collaborative Site The CIBMTR Collaborative site (collaborate.cibmtr.org) uses the SharePoint Enterprise Collaboration platform to promote cooperative work among CIBMTR staff members and provides a communication platform for specific studies and initiatives. This site is secured by username and password, and user-specific security credentials are assigned centrally. The CIBMTR uses the site for storing and sharing protocol and consent documents, donor / recipient tracking tools, confidential committee information, data, manuscript drafts, and other relevant information. While only two Working Committees currently use the Collaborate site on a regular basis, it is available to all Working Committees for sharing information. Data Back to Centers Application (628 unique page views) Links to the Data Back to Centers (DBtC) application that provides CIBMTR member centers the ability to retrieve all the TED-level data their center has reported to the CIBMTR through FormsNet or AGNIS. Page | 35 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.2.3 CIBMTR Portal Site The CIBMTR Portal site (portal.cibmtr.org) delivers applications and data to CIBMTR transplant centers and other partners. In 2015, unique, external visitors viewed 2,675 portal pages. Four applications are currently hosted on this site: Data Back to Centers The current DBtC application provides authorized users the ability to download CIBMTR TED-level data variables for their centers. The data have been validated and processed in the CIBMTR Research Database and are reviewed and refreshed quarterly. Legacy International Bone Marrow Transplant Registry (IBMTR) data from as far back as 1964 and some legacy NMDP/Be The Match data from as far back as 1987 are available. In 2015, 628 unique, non-CIBMTR visitors viewed 914 DBtC pages and downloaded data 418 times. In Spring 2016, the CIBMTR plans to release an enhanced DBtC (eDBtC) application that will give centers access to both TED- and CRF-level source data as well as a variety of analytic tools via a user-friendly application called Qlikview. Center Volumes Portal The Center Volumes Portal allows centers to preview; correct, if necessary; and approve center volume data published annually to the HRSA Blood Cell Transplant website. The CIBMTR gives centers access to display and download the previous six years (2009-2014) of volume data as well as the current year under review (2014). In 2015, 188 unique, external visitors viewed 706 Center Volumes Portal pages. Patient One-Year Survival Calculator – Allogeneic Transplants Accessible by medical directors, the Patient One-Year Survival Calculator for Allogeneic Transplants provides centers with a tool to predict one year survival for individual allogeneic HCT recipients. The calculator data are updated annually to reflect new information contained in the center outcomes analysis. In 2015, 448 unique, non-CIBMTR visitors viewed 1,357 survival calculator pages. Qlikview In 2015, the CIBMTR selected Qlikview, a third party business intelligence tool, to provide transplant centers with self-service access to their data and information, including descriptive statistics, outcomes, and trends. Using Qlikview, centers will be able to more readily visualize their data in predefined dashboards, interact with their data through ad hoc analytics, and export their data to files that will support further analysis. The CIBMTR will introduce two Qlikview applications at the 2016 BMT Tandem Meetings: eDBtC, which will provide transplant centers with access to their TED- and CRF-level data, and CenterSpecific Analysis Tool, which will provide transplant centers with access to their SCTOD database data. Page | 36 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.2.4 Be The Match Public Website 3.2.6 HRSA Blood Cell Transplant Website The Be The Match Public website (bethematch.org) is designed for patients and families, donors, and supporters. It incorporates detailed information about transplantation and donation written for the public. The website provides scientific information in lay terms for donors related to the donation process and for patients related to specific diseases, various treatment options, the process of transplantation, and life after transplant. It also addresses concerns related to specific populations, including children and caregivers. The CIBMTR collaborates with NMDP/Be The Match to provide content for several areas of this website, including data for the US Center Listing Report. The HRSA Blood Cell Transplant website (bloodcell.transplant.hrsa.gov) provides information for the public, physicians, and other constituents. It incorporates transplant resources, donor information, and cord blood information as well as research, data, and outcomes. CIBMTR data and research findings are incorporated in numerous ways, including through CIBMTR-created reports: US Center Listing Report Transplant Center Directory Provides transplant center specific information about facilities, personnel, diseases treated, cost, and transplant experience, including the number of transplants performed and survival rates by age, disease type, and disease stage. 3.2.5 Be The Match Clinical Website The Be The Match Clinical website (bethematchclinical.org) is designed for clinicians, network participants, payors, and bioinformatics professionals. For clinicians, the website provides access to evidence-based tools, clinical guidelines, outcomes data, and education courses on HCT. The website also provides information specific to types of network participants: transplant centers, donor centers, apheresis and collection centers, and cord blood banks. For payors, the website offers information to help individuals understand BMT, determine coverage, and answer employer and patient questions. Related to bioinformatics, the website provides resources for immunogenetic-focused research and operational bioinformatics as well as frequently used HLA tools. Transplant Outcomes and Data US Patient Survival Report Provides disease-specific post-HCT survival estimates by the length of time after transplant: 100 days, 1 year, and 3 years. Survival estimates are also available by patient age, patient gender, patient race, or cell source. Transplant Data by US Center Report Displays the number of adult donor and cord blood transplants performed at a specific transplant center. Transplant Data by Disease Report Displays the number of adult donor and cord blood transplants reported for a specific disease. Totals are also available by patient age, patient gender, patient race, cell source, and the year the transplant was performed. Transplant Activity Report Displays the number of transplants performed at US transplant centers, including autologous as well as related and unrelated allogeneic. Numbers are also available by patient age, patient gender, patient race, cell source, disease, transplant center location by state, and year in which the transplant was performed. Page | 37 CIBMTR 2015 Annual Report 3.2.7 Other Applications and Data Exchange Standards The CIBMTR has 7 methods for sharing data. The 4 hosted on the CIBMTR Portal site (DBtC, Center Volumes Portal, Patient One-Year Survival Calculator, and Qlikview) were described in Section 3.2.3. The other 3 methods for sharing data are: AGNIS AGNIS (A Growable Network Information System) allows participating centers to electronically collect and share data with the CIBMTR as well as others who link to AGNIS. Data are entered once and then distributed and synchronized among databases. In 2015, a total of 12,873 forms for 1,840 patients were submitted through AGNIS by 20 US transplant centers and by EBMT for 47 of their affiliated centers. 3.0 HOW WE SHARE KNOWLEDGE Disease Risk Index Assignment Tool In March 2015, the CIBMTR launched a Disease Risk Index Assignment Tool developed by investigators at the Dana Farber Cancer Institute and validated in a large CIBMTR study. It is intended for use by clinical researchers. The tool was developed for the primary outcome of overall survival after HCT and, at present, only applies to adult patients with hematologic malignancies. It is NOT intended to give an accurate prognosis for individual patients. In 2015, 1,370 unique, non-CIBMTR visitors viewed the Disease Risk Index Assignment Tool 3,764 times. BRIDG The BRIDG (Biomedical Research Integrated Domain Group) Model is an information model, representing a shared view of the concepts of basic, pre-clinical, clinical, and translational research. Common data elements for certain standard CIBMTR forms have been extracted and associated in the BRIDG model to one of three contexts: recipient, donor, or stem cell product. Future expansion of the BRIDG model will add HCT content as the basis for a physical database model, which will help remove barriers that transplant centers experience in electronic transfer of HCT data to the SCTOD. It will provide a foundation upon which centers and vendors can develop their own in-house data systems and, eventually, develop electronic medical record integration engines to submit data to the CIBMTR Research Database. Page | 38 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.3 BMT TANDEM MEETINGS The BMT Tandem Meetings are co-sponsored with the ASBMT and are held annually in February. They include 5 days of plenary sessions, concurrent scientific sessions, and other meetings. Reports on recent progress and updates in basic science, translational research, and clinical studies are targeted to worldwide physicians, scientists, and other health professionals with an interest in HCT. 2015 BMT Tandem Meetings With 3,132 attendees from 47 countries, the 2015 BMT Tandem Meetings included 5 plenary sessions, 11 concurrent sessions, 108 oral abstracts, 2 poster sessions, 6 corporatesupported symposia, and 3 product theaters. Continuing Medical Education (CME) and Continuing Education credits were issued through MCW to physicians and allied health professionals. In addition to the extensive scientific agenda, many educational opportunities focused on young investigators and other allied health professionals. Clinical Research Professionals / Data Management Conference With almost 200 attendees, this conference provided forms training, which increases the accuracy with which CIBMTR forms are completed. BMT CTN Coordinators and Investigators Meetings With approximately 100 and 300 attendees, respectively, these meetings focused on treatment options; study management, such as promoting studies and reporting adverse events; processes, such as endpoint review; and specific clinical trials. IT Forum With almost 100 attendees, this forum discussed the Research Data Life Cycle and various ways to share data, including from electronic medical records via BRIDG. BMT Center Administrators Conference With approximately 165 attendees, this conference focused on many topics related to quality and outcomes, including benchmarking and key metrics, such as readmission rates, as well as Medicare coverage and workforce capacity. BMT Pharmacists Conference With almost 200 attendees, this conference presented the latest research and best practices with a focus on specific diseases and preparative regimens. Transplant Nursing Conference With almost 500 attendees, this conference presented the latest research as well as disease-specific information and communication techniques. BMT Clinical Education Conference This conference is designed for Nurse Practitioners, Physician Assistants, Fellows, and Junior Faculty. With almost 200 attendees, the conference focused not only on the latest clinical research but also informed consent and mental health. 2016 BMT Tandem Meetings The 2016 BMT Tandem Meetings will include 5 plenary sessions, 9 concurrent sessions, 133 oral abstracts, 2 poster sessions, 5 corporatesupported symposia, and 5 product theaters. They are expected to attract approximately 3,200 attendees. Page | 39 CIBMTR 2015 Annual Report 3.0 HOW WE SHARE KNOWLEDGE 3.4 DATA MANAGEMENT TRAINING The CIBMTR has developed comprehensive, secure and efficient applications to allow you to electronically submit data to the CIBMTR. Visit the CIBMTR Data Management Training and Reference webpage to access resources. Center Reference Guide Learn about participation in CIBMTR research, center membership, access to FormsNet, data manager education, mentor program, forms submission process and many useful tips and links. Manuals Find the answers to your data submission questions by accessing the Forms Instructions Manual, which includes general instructions and instructions for each form type. FormsNet Learn how to submit data to the CIBMTR via the FormsNet application, a secure clinical research management system, which is in compliance with SCTOD requirements. Conference Materials Access meeting materials as well as audio and visual presentations on the form submission process presented at Clinical Research Professionals / Data Management Conferences. AGNIS Learn how to retrieve and transmit form data, extracted directly from your own institution’s database, directly to the FormsNet application using AGNIS, a secure, standards-based system. Legacy Data Review retired data manuals, forms, and other archived documents for reference purposes and to assist in making changes to legacy data. Adverse Events Learn how to report adverse events and product issues through FormsNet. Newsletters and eBlasts Read archived issues of the Data Matters Training Newsletter and eBlasts. Tip Sheets Access sheets providing tips and instructions for various CIBMTR forms. Online Training Review educational modules developed for new and seasoned data managers through the NMDP/Be The Match Learning Center. Learning Center Courses Data Back to Centers Overview HLA SERIES Introduction to HLA HLA Reporting (Form 2005) Basic Biology of HLA Advanced Biology of HLA ESSENTIAL FORM SERIES Baseline Form 2000 CRID 2804 Form 2400 Pre-TED Indication for CRID Assignment (Form 2814) Infusion Data (Form 2006) Reporting Overview Reporting Preparative Regimen on PreTED (Form 2400) and Baseline (Form 2000) DISEASE SPECIFIC SERIES Form 2016 / 2116 Plasma Cell Disorders Form 2018 / 2118 Lymphoma Page | 40 CIBMTR 2015 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA 4.0 HOW WE COLLECT AND MANAGE DATA 4.1 RESEARCH DATA LIFE CYCLE The Research Data Life Cycle (Figure 4.1) describes the path of data from the point of capture to its ultimate use in analysis, reporting, and publication. The process begins with data collection. Most centers enter data in FormsNet, a web application now in its third generation. Centers that have implemented local or third party systems can also capture and submit data electronically using AGNIS. An overriding goal of these applications is to ease the data capture burden on the centers. Data Sharing completes the cycle, providing data for analysis that have been collected and curated to ensure research value. These data are extracted from the Research Database in monthly and quarterly retrievals to serve a range of research and stakeholder needs. The data retrievals provide the basis for research study data files, reports, and externallyrequested datasets. TED-level data are also directly available to centers through use of the DBtC application (Section 3.2.3). Following collection, data undergo quality assessment and validation and are extracted 4 times per month and loaded into the CIBMTR Research Database. Figure 4.1. Research Data Life Cycle Abbreviations: AGNIS = A Growable Network Information System, CRID = CIBMTR Recipient Identification Number, CRF = Comprehensive Report Form, FN = FormsNet, IDW = Integrated Data Warehouse, RDB = Research Database Page | 41 CIBMTR 2015 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA 4.2 COLLECTING AND STORING DATA 4.3 ENSURING DATA QUALITY 4.2.1 FormsNet 4.3.1 Continuous Process Improvement More than 95% of data collected by the CIBMTR is submitted electronically via FormsNet, a comprehensive electronic data submission system containing >240 forms related to capturing HCT outcomes for donors and recipients. The application was updated in 2015 to provide key enhancements supporting operational efficiencies and enhanced data collection. For instance, a more flexible CIBMTR Recipient ID (CRID) Assignment Form establishes a patient ID independent of treatment, allowing the CIBMTR to expand data collection to patients who receive cellular therapy as well as non-HCT treatments. In addition, the CIBMTR released several forms to support data collection needs of the Radiation Injury Treatment Network (RITN), which will collect incident, medical history, and follow-up data on casualties exposed to or contaminated by radioactive material in the event of a national radiation event. Robust data collection is critical to the success of the CIBMTR. The CPI program ensures timeliness and completeness of data forms submissions (Appendix H). 4.2.2 Research Database The Donor Data Management Team oversees submission of transplant donor forms from NMDP/Be The Match donor, collection, and apheresis centers. Donor CPI reports are generated 4 times per year (January, April, July, and October). To be compliant, centers must submit 100% of the forms required for that CPI period. The CIBMTR Research Database now contains information on >425,000 patients. Submission of outcomes data is mandatory for allogeneic HCTs in the US and those outside the US that use a US donor; all other submissions are voluntary. The CIBMTR estimates that almost 100% of US allogeneic transplants and about 80% of US autologous HCTs are reported. Recipient Forms Transplant centers receive CPI reports 3 times per year (January, May, and September), listing the number of follow-up forms that were due in the previous trimester and the number and percentage of each submitted within the trimester. A form is not officially submitted until all errors are resolved and all applicable information is submitted and approved. To be compliant, centers must submit ≥90% of forms due for the trimester, for all unrelated donor transplants and for related donor and autologous transplants that have occurred since December 3, 2007. Donor Forms Page | 42 CIBMTR 2015 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA 4.3.2 Verification and Validation FormsNet When data are entered into FormsNet, a series of entry level validation checks takes place to ensure data consistency. This process flags certain errors at the time of entry and allows the CIBMTR to contact the center data manager so errors can be corrected immediately while source documents are readily available. If a data field does not pass the FormsNet validation checks, an error comment is generated, and the data manager is navigated to an error review page to review, resolve, or override the unresolved errors. Lastly, an error report is generated that lists any unresolved errors as well as errors that have been overridden. Research Database Data extracted from FormsNet and loaded to the Research Database each month undergoes comprehensive validation and verification. These data are rigorously validated for consistency, completeness, and uniqueness using business rules implemented in custom logic for the categories provided below. Finally, the Data Quality Team reviews errors and works with transplant centers to correct data. Extraction Validation Checks Fields that cannot be null Cross form consistency Entry Level Validation Checks Longitudinal consistency Mandatory field validation Logical relationships Range validation Cross form consistency Within form consistency Core field validations Rules across these categories were updated and tested as part of the recent Forms Revision. The CIBMTR continues to work toward reducing the number of data entry errors. Currently, the rate of form rejection due to inconsistently reported data is <2%, which is attributed to recent enhancements in transplant center education as well as to enhanced validations built in at the point of entry in FormsNet. Page | 43 CIBMTR 2015 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA 4.3.3 On-Site Data Audit Program On-going data audits are performed at all CIBMTR participating transplant centers. The audit compares data in source documents maintained at the transplant center with data contained in the CIBMTR Research Database. Clinical Research Associates perform the onsite transplant center audits, spending 3-4 days at each center reviewing original source documents. The overall audit process is displayed in Figure 4.2. The Foundation for the Accreditation of Cellular Therapy (FACT) and the CIBMTR agreed in 2015 to consolidate transplant center data audits. Therefore, beginning in 2016, FACT auditors will use the results of the CIBMTR data audit and discontinue use of the current independent FACT data audit. Consolidating audits of transplant center data collection and reporting eases the reporting and compliance burdens of transplant centers and demonstrates the value of the CIBMTR’s data collection and management to an international accrediting agency. In 2015, 62 centers were scheduled for audit (48 domestic, 16 international). As of December 31, 57 centers were notified of their final audit results, including requested corrective action follow-up. Of the centers sent reports, 80% passed with ≤3% critical field errors. Of the 11 centers that did not pass the audit, 7 completed all required corrective action; the remaining 4 centers are in the process of completing requested corrective action. Figure 4.2. Audit Process Page | 44 CIBMTR 2015 Annual Report 4.0 HOW WE COLLECT AND MANAGE DATA 4.4 PROTECTING PATIENTS AND DATA 4.4.1 Human Subjects / HIPAA Compliance 4.4.2 Information Security and Data Privacy The CIBMTR is committed to the ethical conduct of research. All Coordinating Center personnel maintain Collaborative IRB Training Initiative (CITI) certification. The NMDP/Be The Match IRB, which is fully accredited by the Association for the Accreditation of Human Research Protection Programs, reviews all human subject research conducted by the CIBMTR. The CIBMTR maintains compliance with the Health Insurance Portability and Accountability Act (HIPAA) Privacy Rule, as applicable. CIBMTR rules requiring the registration of all consecutive HCT recipients ensure the inclusion of women, minorities, and children, so the Research Database population includes women and minorities in the same proportion as found in the general HCT population. Children are included in most CIBMTR studies; their inclusion is dependent on the study focus. The CIBMTR protects the data and information received from transplant centers and patients. The SCTOD contract requires specific protections through minimum security controls, policies, and standards. The CIBMTR’s data systems are maintained in accordance with the Federal Information Systems Management Act of 2002, with information security guidance provided by the National Institute of Standards and Technology. In accordance with National Institute of Standards and Technology Special Publication 800-18, and supervised by HRSA’s Office of Information Technology, the CIBMTR maintains a System Security Plan that outlines management, operational, and technical controls. Since 2008, the CIBMTR holds an Authority to Operate from the Chief Information Officer of HRSA. The certification was renewed in June 2015, and security audits are performed annually, most recently in September 2015. The NMDP/Be The Match also holds an Authority to Operate from HRSA, ensuring similar standards of information security are applied to all CIBMTR and NMDP/Be The Match systems. These controls, maintained by the CIBMTR and NMDP/Be The Match, protect the data and information in these systems in ways beyond those required by HIPAA. Page | 45 CIBMTR 2015 Annual Report 5.0 WHAT WE WILL DO NEXT 5.0 WHAT WE WILL DO NEXT In 2016, the CIBMTR will continue to conduct high quality research through a collaborative process. Specific plans are listed in Figure 5.1 and Tables 5.2-5.4. Figure 5.1. Plans for 2016 Enhance the quality and scope of CIBMTR data Expand data and knowledge sharing Increase research productivity and scientific impact Details provided in Tables 5.2-5.4. Page | 46 CIBMTR 2015 Annual Report Table 5.2. Plans to Enhance Data Enhance the quality and scope of CIBMTR data Further develop a cellular therapy registry Expand data collection to patients who receive non-HCT treatments Continually review data collection forms for scientific content updates Implement key data validations in FormsNet, close to the point of data capture Implement consolidated transplant center data audits with FACT to enhance data monitoring while decreasing reporting and compliance burdens on transplant centers Work toward an Integrated Data Warehouse to strengthen flexibility of data use and ease of data access 5.0 WHAT WE WILL DO NEXT Table 5.3. Plans to Expand Knowledge Sharing Expand data and knowledge sharing Implement two data sharing products using Qlikview software applications to enhance transplant centers’ self-service access to their data and information • eDBtC will provide centers with access to a wide range of TED- and CRF-level data • Performance data tool will provide centers with comparison data from the SCTOD annual center-specific analysis of posttransplant survival in US centers Publish easy to read summaries of key HCT publications Strengthen collaborations with international partners to facilitate data sharing Provide onsite and online data management training support for transplant centers Page | 47 CIBMTR 2015 Annual Report 5.0 WHAT WE WILL DO NEXT Table 5.4. Plans to Increase Impact Increase research productivity and scientific impact Focus on studies with highest scientific impact and important clinical and policy implications; limit accepted studies to a number that can be completed in a timely manner Support Working Committee Chairs to be active leaders in their committees Regularly share metrics as a Working Committee management resource Expand use of outcomes data to assist in the design, implementation, and longterm follow-up of clinical trials Develop new statistical methodologies Support Health Services Research initiatives Page | 48 CIBMTR 2015 Annual Report 2015 KEY ACCOMPLISHMENTS 2015 KEY ACCOMPLISHMENTS CIBMTR by the Numbers RESEARCH DATABASE ~400 participating centers >425,000 patients ~21,000 new patients annually PUBLICATIONS >1,000 publications since inception 76 publications in 2015 38 from Working Committees 8 from BMT CTN 4 from Health Services Research Program 9 from Bioinformatics Research Program 5 from Statistical Methodology Research Program 14 from Coordinating Center (2 cross-listed between programs) PRESENTATIONS 65 presentations in 2015 (41 oral and 24 poster) 25 abstracts (17 oral and 8 poster) presented at the 2015 ASH Annual Meeting 17 abstracts (13 oral and 4 poster) presented at the 2015 BMT Tandem Meetings 6 abstracts (4 oral and 2 poster) presented at the 2015 EBMT Annual Meeting 17 abstracts (7 oral and 10 poster) presented at other national and international conferences Clinical Outcomes Research Program WORKING COMMITTEES Administered 15 committees in which 2,300 worldwide researchers participate Collaborated with the 54 global experts in the HCT field who voluntarily chair the committees Conducted 176 ongoing studies Reviewed 193 new study proposals for presentation at the 2016 BMT Tandem Meetings, >75% of which were submitted by new investigators Presented 38 abstracts (26 oral and 12 poster) at national and international conferences Supported approximately 400 scientific authors at 200 institutions worldwide to publish research findings Published 38 manuscripts in peerreviewed journals CELLULAR THERAPIES AND NONTRANSPLANT THERAPY Initiated a Cellular Therapies Task Force charged with determining the type and process for cellular therapies data collection Hosted a Cellular Therapies Forum to develop recommendations for a cellular therapy registry Page | 49 CIBMTR 2015 Annual Report Immunobiology Research Program Collected samples from 175 centers (128 transplant centers, 32 donor centers, and 15 cord blood banks) Curated 12,656 samples in the Research Repository (137,464 overall) Completed high resolution HLA and presence / absence KIR typing on 226 related and 2,772 unrelated HCT donor / cord and recipient pairs Distributed 8,770 samples to investigators for various studies Presented 5 abstracts (2 oral and 3 poster) at national and international conferences Published 6 manuscripts in peerreviewed journals RESEARCH REPOSITORY Added 4,392 unrelated recipient samples (57,018 overall) Added 1,264 related recipient samples (5,377 overall) Added 4,819 adult unrelated donor samples (59,375 overall) Added 1,258 related donor samples (5,128 overall) Added 923 unrelated cord blood samples (10,566 overall) 2015 KEY ACCOMPLISHMENTS Clinical Trials Support Program BMT CTN Opened 3 new trials to accrual, bringing the total number of launched trials to 37 Accrued >1,100 patients to trials, increasing the total number of accrued patients to >8,500 Managed 12 open protocols with overall accrual for open studies at about 125% of projections Presented 11 abstracts (8 oral and 3 poster) at national and international conferences Published 8 manuscripts in peerreviewed journals RCI BMT Opened 3 new trials to accrual, bringing the total number of launched trials to 15 Accrued just under 3,000 patients, bringing the total number of accrued patients to approximately 28,000 Presented 4 oral abstracts at national and international conferences Survey Research Group Supported 8 active studies, and participated in the development of one upcoming study Page | 50 CIBMTR 2015 Annual Report Health Services Research Program Established the HCT Health Economics Interest Group, which held its first meeting at the 2015 BMT Tandem Meetings Opened a multi-center randomized controlled study to evaluate the effectiveness of an individualized survivorship care plan template; 17 centers will participate in this study Conducted a national survey of community hematologists / oncologists on referral practices, perceptions of HCT, and education preferences to inform an educational intervention on timing of community-based referral for HCT Completed two studies: Identifying HCT patient housing and caregiver challenges and potential interventions and The financial impact of allogeneic HCT on patient and family: a pilot study Conducted >30 evaluations for the HRSA Office of Patient Advocacy / Single Point of Contact contract and NMDP/Be The Match programs Provided technical plain language writing consultation; developed 3 easy-to-read consent forms and 6 patient information sheets for BMT CTN protocols Presented 3 oral abstracts at a national conference Published 4 manuscripts in peerreviewed journals 2015 KEY ACCOMPLISHMENTS Bioinformatics Research Program Developed guidelines for reporting HLA and KIR genotyping via Next Generation Sequencing Investigated the role of genetic ancestry in transplantation Developed methods for HLA association studies Investigated HLA data from other countries Presented 11 abstracts (5 oral and 6 poster) at national and international conferences Published 9 manuscripts in peerreviewed journals Statistical Methodology Research Program Developed new statistical models Ensured the statistical integrity of CIBMTR scientific activities Contributed to results in articles on HCTrelated statistical issues for clinical audiences Supported Working Committee study investigators in developing scientific study protocols using CIBMTR data Published 4 peer-reviewed manuscripts and 1 editorial Page | 51 CIBMTR 2015 Annual Report 2015 KEY ACCOMPLISHMENTS SCTOD Published annual Center-Specific Survival Report, Transplant Center Volumes Data for 2009-2013, and a Transplant Activity Report for transplants performed 20092013 Began implementing recommended changes to tools to enhance transplant centers’ quality improvement efforts in response to recommendations from the 2014 Center Outcomes Forum Continued analysis of a quality of life project, which is unique in that the CIBMTR collected data directly from patients after HCT In conjunction with Be The Match’s Patient and Health Professional Services, published 12 lay summaries of CIBMTR publications Page | 52 CIBMTR 2015 Annual Report APPENDIX A: TRANSPLANT CENTERS APPENDIX A: TRANSPLANT CENTERS Figure A.1. Location of Transplant Centers that Submit Data to the CIBMTR Page | 53 CIBMTR 2015 Annual Report APPENDIX A1: US CENTERS APPENDIX A1: US CENTERS The following table lists the US-based transplant centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF. Participating Center City State MUD RELATED AUTO Birmingham AL CRF CRF CRF Providence Hospital HPC Transplant Center Mobile AL N/A N/A CRF University of Arkansas for Medical Sciences Little Rock AR CRF TED N/A Gilbert AZ CRF CRF CRF Phoenix AZ CRF CRF CRF Phoenix AZ CRF CRF CRF Scottsdale AZ CRF CRF CRF University Medical Center - Tucson Tucson AZ CRF TED TED City of Hope National Medical Center Duarte CA CRF TED TED La Jolla CA CRF CRF CRF La Jolla CA CRF CRF CRF Loma Linda University Cancer Center Loma Linda CA CRF CRF CRF Cedars Sinai Medical Center Los Angeles CA CRF TED N/A Children's Hospital of Los Angeles Los Angeles CA CRF TED TED UCLA Center for Health Sciences Los Angeles CA CRF CRF TED USC BMT Program Los Angeles CA CRF CRF CRF Children's Hospital of Oakland Oakland CA CRF CRF CRF Children's Hospital of Orange County Orange CA CRF CRF CRF University of Alabama Birmingham Banner MD Anderson Cancer Center Banner Blood and Marrow Transplant Program Mayo Clinic Arizona and Phoenix Children's Hospital Cancer Transplant Institute at Virginia G. Piper Cancer Center Scripps Blood & Marrow Transplant Program University of California, San Diego Medical Center Page | 54 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO St Joseph’s Hospital Irvine Orange CA N/A N/A CRF University of California Irvine Medical Center Orange CA CRF CRF CRF Palo Alto CA CRF CRF CRF Sutter Cancer Center Sacramento CA TED TED TED University of California - Davis Cancer Center Sacramento CA CRF TED TED San Diego CA CRF CRF CRF San Francisco CA CRF CRF CRF San Francisco CA CRF CRF CRF Stanford Health Care Stanford CA CRF CRF TED Stanford University Medical Center Stanford CA CRF CRF TED The Children's Hospital of Denver Aurora CO CRF CRF CRF University of Colorado Hospital Aurora CO CRF TED TED Colorado Blood Cancer Institute Denver CO CRF TED TED University of Colorado Medical School Denver CO N/A N/A N/A University of Connecticut Farmington CT N/A N/A N/A Yale New Haven Hospital New Haven CT CRF CRF CRF Children's National Medical Center Washington DC CRF TED TED Medstar Georgetown University Hospital Washington DC CRF N/A CRF Newark DE CRF CRF CRF Alfred I. duPont Hospital for Children Wilmington DE CRF CRF CRF Shands HealthCare & University of Florida Gainesville FL CRF CRF CRF Lucile Packard Children’s Hospital Rady Children's Hospital San Diego University of California - San Francisco Adults University of California - San Francisco Pediatrics Christiana Care Page | 55 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO BMT Program of Mayo Clinic / Nemours and Wolfson Children's Hospital Jacksonville FL CRF CRF CRF Mayo Clinic Florida - Jacksonville Jacksonville FL CRF CRF CRF Miami Children's Hospital Miami FL CRF TED TED University of Miami - Adults Miami FL CRF CRF CRF Miami FL CRF TED TED Orlando FL CRF CRF CRF Pembroke Pines FL TED TED TED St. Petersburg FL CRF CRF CRF H Lee Moffitt Cancer Center Tampa FL CRF TED TED Children's Healthcare of Atlanta at Egleston Atlanta GA CRF CRF CRF Emory University Atlanta GA CRF CRF CRF The Blood and Marrow Transplant Program at Northside Hospital Atlanta GA CRF CRF CRF Georgia Regents University Cancer Center Augusta GA CRF CRF CRF Hawaii Medical Center Honolulu HI N/A N/A N/A Kapi'olani Medical Center for Women and Children Honolulu HI CRF CRF CRF University of Iowa Hospital & Clinics Iowa City IA CRF CRF CRF St. Luke's Mountain States Tumor Institute Boise ID N/A N/A CRF Ann & Robert H. Lurie Children's Hospital of Chicago Chicago IL CRF CRF CRF Northwestern Memorial Hospital Chicago IL CRF CRF TED Chicago IL CRF TED N/A Chicago IL CRF CRF CRF University of Miami / Jackson Memorial Hospital Blood & Marrow Transplant Center, Florida Hospital Medical Group Memorial Cancer Institute All Children's Hospital Northwestern Memorial Hospital Department of Immunotherapy The Coleman Foundation Blood and Marrow Transplant Center, Rush University Page | 56 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO University of Chicago Medical Center Chicago IL CRF CRF CRF University of Illinois at Chicago Medical Center Chicago IL CRF CRF CRF Loyola University Medical Center Maywood IL CRF CRF CRF Advocate Lutheran General Hospital Park Ridge IL N/A TED TED Zion IL CRF CRF CRF Indiana Blood & Marrow Transplantation Beech Grove IN CRF CRF CRF Indiana University Hospital / Riley Hospital for Children Indianapolis IN CRF CRF TED St Vincent Hospital Indianapolis Indianapolis IN N/A CRF CRF University of Kansas Kansas City KS CRF CRF CRF Wichita KS N/A CRF CRF Lexington KY CRF TED TED Louisville KY CRF TED TED New Orleans LA CRF CRF CRF Ochsner Medical Center New Orleans LA N/A CRF TED Tulane University Medical Center New Orleans LA CRF CRF CRF Shreveport LA CRF CRF CRF Beth Israel Deaconess Medical Center Boston MA CRF TED TED Boston Medical Center Boston MA N/A N/A TED Boston MA CRF CRF TED Boston MA CRF TED TED Boston MA CRF TED TED Cancer Treatment Centers of America Midwest Via Christi Hospitals Wichita University of Kentucky Medical Center University of Louisville Hospital / James Brown Cancer Center Louisiana State University Children's Hospital Louisiana State University Health Sciences Center - Shreveport Dana Farber Cancer Institute at Brigham and Women's Hospital - Adults Dana Farber Cancer Institute at Brigham and Women's Hospital - Pediatrics Massachusetts General Hospital Page | 57 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO Boston MA CRF TED TED Lahey Clinic Medical Center Burlington MA N/A N/A TED UMass Memorial Medical Center Worcester MA CRF CRF CRF Johns Hopkins Oncology Center Baltimore MD TED TED TED University of Maryland School of Medicine Baltimore MD CRF CRF CRF National Heart Lung & Blood Institute Bethesda MD N/A TED N/A National Institutes of Allergy & Infectious Disease Bethesda MD N/A TED N/A National Institutes of Health Bethesda MD N/A TED N/A Bethesda MD N/A CRF CRF Bethesda MD CRF N/A N/A Ann Arbor MI CRF TED TED Children's Hospital of Michigan Detroit MI CRF CRF CRF Henry Ford Hospital Bone Marrow Transplant Program Detroit MI CRF CRF CRF Karmanos Cancer Institute Detroit MI CRF CRF CRF Helen DeVos Children's Hospital Grand Rapids MI CRF CRF CRF Spectrum Health Grand Rapids MI CRF CRF CRF Abbott Northwest Hospital Minneapolis MN N/A N/A TED Masonic Cancer Center University of Minnesota Minneapolis MN CRF CRF CRF Mayo Clinic Rochester Rochester MN CRF CRF CRF Saint Luke’s Blood & Marrow Transplant Program Kansas City MO CRF CRF CRF Tufts New England Medical Center NIH - NCI Experimental Transplantation and Immunology Branch (Related Donor Program) NIH - NCI Matched Unrelated Donor Program The University of Michigan Page | 58 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO Kansas City MO CRF CRF CRF Cardinal Glennon Children's Hospital St. Louis MO CRF CRF CRF SSM Health Saint Louis University Hospital St. Louis MO CRF TED TED Washington University School of Medicine St. Louis MO CRF CRF TED St. Louis MO CRF CRF CRF Jackson MS CRF TED TED Billings MT N/A N/A TED Chapel Hill NC CRF CRF CRF Charlotte NC CRF CRF CRF Levine Cancer Institute Charlotte NC N/A CRF CRF Duke University - Adults Durham NC CRF TED TED Durham NC CRF CRF CRF Durham NC N/A TED N/A NC N/A N/A N/A NC CRF TED TED The Children's Mercy Hospitals and Clinics Washington University / St Louis Children's Hospital University of Mississippi Medical Center Jackson Billings Clinic Cancer Center University of North Carolina Hospitals Chapel Hill BMT Program at Levine Children's Hospital / Carolinas Medical Center Duke University Medical Center, Pediatric BMT Duke University, Immunology / BMT, Pediatrics Novant Health Oncology Specialists Wake Forest Baptist Health WinstonSalem WinstonSalem CHI Health Bergan Mercy Omaha NE N/A N/A TED CHI Health Immanuel Omaha NE N/A N/A TED Nebraska Methodist Hospital Omaha NE N/A N/A TED University of Nebraska Medical Center Omaha NE CRF CRF CRF Dartmouth-Hitchcock Medical Center Lebanon NH CRF CRF CRF Hackensack University Medical Center Hackensack NJ CRF CRF CRF Page | 59 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO New Brunswick NJ CRF CRF CRF Albuquerque NM N/A N/A CRF Childrens Hospital at Montefiore Bronx NY CRF TED TED Montefiore Medical Center Bronx NY CRF CRF TED Roswell Park Cancer Institute Buffalo NY CRF CRF CRF Westchester Medical Center Hawthorne NY CRF CRF CRF North Shore University Hospital Manhasset NY CRF TED TED Cohen Children's Medical Center of New York Memorial Sloan Kettering Cancer Center Adults Memorial Sloan Kettering Cancer Center Pediatrics Morgan Stanley Children's Hospital of New York New Hyde Park NY CRF CRF CRF New York NY CRF TED TED New York NY CRF TED TED New York NY CRF CRF CRF Mount Sinai Medical Center - New York New York NY CRF CRF TED New York Presbyterian Hospital New York NY CRF CRF CRF New York Presbyterian Hospital / Columbia University Medical Center New York NY CRF CRF CRF New York University Medical Center New York NY TED TED TED St Vincent's Hospital Manhattan New York NY N/A TED TED University of Rochester Medical Center Rochester NY CRF CRF CRF Stony Brook University Medical Center Stony Brook NY TED TED CRF State University of NY Upstate Medical University Syracuse NY N/A CRF CRF Akron OH CRF CRF CRF Cincinnati OH CRF CRF CRF Cancer Institute of New Jersey University of New Mexico - Pediatrics Akron Children's Hospital Cincinnati Children's Hospital Page | 60 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO Jewish Hospital Blood and Marrow Transplant Center Cincinnati OH CRF CRF CRF University of Cincinnati Medical Center Cincinnati OH CRF CRF CRF Cleveland Clinic Cleveland OH CRF CRF CRF Seidman Cancer Center - University Hospitals Case Medical Center Cleveland OH CRF CRF CRF Nationwide Children's Hospital Columbus OH CRF CRF CRF The Ohio State University Medical Center Columbus OH CRF CRF CRF Miami Valley Hospital BMT Program Dayton OH N/A N/A CRF Oklahoma University Medical Center Oklahoma City OK CRF CRF CRF Cancer Treatment Centers of America Tulsa OK CRF CRF CRF Saint Francis Hospital - Oklahoma Tulsa OK N/A TED TED Legacy Good Samaritan Hospital and Medical Center Portland OR N/A N/A CRF Oregon Health and Science University Portland OR CRF CRF CRF Pediatric BMT Program, Doernbecher Children's Hospital (OHSU) Portland OR CRF CRF CRF Providence Portland Medical Center Portland OR N/A N/A CRF Geisinger Medical Center Danville PA CRF TED TED Penn State Hershey Medical Center Hershey PA CRF CRF CRF Abramson Cancer Center University Pennsylvania Medical Center Philadelphia PA CRF CRF CRF Eastern Regional Medical Center Philadelphia PA N/A CRF CRF Hahnemann University Hospitals Philadelphia PA CRF TED TED Philadelphia Children's Hospital Philadelphia PA TED TED N/A St Christopher's Hospital for Children Philadelphia PA TED TED TED Page | 61 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO Temple Bone Marrow Transplant Program Philadelphia PA CRF TED TED Thomas Jefferson University Philadelphia PA TED TED TED Children's Hospital of Pittsburgh of UPMC Pittsburgh PA CRF CRF CRF University of Pittsburgh Medical Center Pittsburgh PA CRF CRF CRF West Penn Hospital Pittsburgh PA CRF CRF CRF Roger Williams Medical Center Providence RI CRF TED TED Charleston Hematology Oncology Charleston SC N/A CRF CRF Medical University of South Carolina Charleston SC CRF TED TED Richland Memorial Hospital Columbia SC N/A N/A N/A Cancer Centers of the Carolinas Greenville SC CRF CRF CRF Saint Francis Hospital - Greenville Greenville SC N/A CRF CRF Avera Mckennan Transplant Institute Sioux Falls SD CRF CRF CRF Thompson Cancer Survival Center Knoxville TN N/A N/A TED Baptist Blood and Marrow Transplant Memphis TN CRF CRF CRF Baptist Centers for Cancer Care Memphis TN TED TED TED St Jude Children's Research Hospital Memphis TN CRF TED N/A The West Clinic / Methodist Healthcare Blood and Marrow Transplant Center Memphis TN N/A N/A TED University of Tennessee Memphis TN CRF CRF CRF Sarah Cannon BMT Center at Centennial Medical Center Nashville TN CRF TED TED Vanderbilt University Nashville TN CRF TED TED Vanderbilt University Veterans Center Nashville TN TED TED N/A Page | 62 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO Amarillo TX CRF TED TED Southwest Regional Cancer Center Austin TX N/A N/A N/A Baylor University Medical Center Dallas TX CRF CRF CRF Children's Medical Center - Dallas Dallas TX CRF CRF CRF Medical City Dallas Hospital Dallas TX CRF CRF CRF UT Southwestern Medical Center - BMT Program Dallas TX CRF CRF CRF Fort Worth TX CRF CRF CRF Baylor College of Medicine Houston TX CRF CRF CRF MD Anderson Cancer Center Houston TX CRF CRF N/A Wilford Hall Medical Center Lackland AFB TX TED TED TED Covenant Health System Hematopoietic Transplant Program Lubbock TX N/A TED TED Texas Tech University Medical Center Lubbock TX CRF CRF CRF South Texas Veterans Health Care System San Antonio TX N/A CRF CRF Texas Transplant Institute San Antonio TX CRF CRF CRF University of Texas Health Science Center San Antonio TX N/A CRF CRF Temple TX N/A N/A CRF Latter Day Saints Hospital Salt Lake City UT CRF CRF CRF University of Utah Medical Center Salt Lake City UT CRF CRF CRF Salt Lake City UT CRF CRF CRF Salt Lake City UT CRF CRF CRF Charlottesville VA CRF CRF CRF Texas Oncology Cook Children's Medical Center Scott and White Memorial Hospital Utah Blood and Marrow Transplant Program - Adults Utah Blood and Marrow Transplant Program - Pediatrics University of Virginia Health System Page | 63 CIBMTR 2015 Annual Report Participating Center APPENDIX A1: US CENTERS City State MUD RELATED AUTO Fairfax - Northern Virginia Hospital Fairfax VA CRF CRF CRF Virginia Oncology Associates Norfolk VA N/A N/A TED Virginia Commonwealth University Massey Cancer Center BMT Program Richmond VA CRF CRF CRF University of Vermont Cancer Center Burlington VT N/A N/A TED Fred Hutchinson Cancer Center Seattle WA CRF CRF CRF VA Puget Sound Healthcare System Seattle WA TED TED N/A Madison WI CRF CRF CRF Marshfield Clinic Marshfield WI N/A N/A CRF Aurora St Luke's Medical Center Milwaukee WI N/A N/A CRF Children's Hospital of Wisconsin Milwaukee WI CRF CRF CRF Froedtert & Medical College of Wisconsin Milwaukee WI CRF CRF TED Morgantown WV CRF CRF CRF University of Wisconsin Hospital and Clinics Osborn Hematopoietic Malignancy & Transplantation Program Page | 64 CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS APPENDIX A2: INTERNATIONAL CENTERS The following table lists the international transplant centers that submited data to the CIBMTR Research Database for matched unrelated donor (MUD) allogeneic, related donor allogeneic, and autologous transplants in the past three years. Centers submit data at two levels: TED and CRF. Participating Center City Country MUD RELATED AUTO Fundaleu-Angelica Ocampo Buenos Aires Argentina CRF CRF CRF Hospital Universitario Austral Buenos Aires Argentina CRF CRF CRF Hospital Privado de Cordoba Cordoba Argentina CRF CRF CRF Royal Adelaide Hospital / SA Pathology Adelaide Australia CRF CRF N/A Royal Prince Alfred Hospital Camperdown Australia CRF CRF TED St Vincent's Hospital Darlinghurst Australia TED TED N/A Herston Australia CRF CRF CRF Melbourne Australia TED TED TED Parkville Australia CRF CRF N/A Fiona Stanley Hospital Perth Australia CRF CRF N/A Princess Margaret Hospital for Children Perth Australia CRF CRF TED Royal Perth Hospital Perth Australia CRF CRF N/A Sydney Children's Hospital Randwick Australia CRF CRF N/A Lady Cilento Children’s Hospital South Brisbane Australia N/A CRF CRF Royal Melbourne Hospital Victoria Australia CRF CRF N/A Calvary Mater Newcastle Hospital Waratah Australia N/A N/A CRF Children's Hospital at Westmead Westmead Australia CRF CRF TED Westmead Hospital Westmead Australia CRF CRF N/A Royal Brisbane & Women's Hospital Alfred Hospital Royal Children's Hospital Page | 65 CIBMTR 2015 Annual Report Participating Center APPENDIX A2: INTERNATIONAL CENTERS City Country MUD RELATED AUTO Vienna Austria TED TED N/A Children's University Hospital Bruxelles Belgium TED TED TED University Hospital Antwerp Edegem Belgium TED TED N/A University Hospital Gasthuisberg Leuven Belgium TED TED N/A University Estadual de Campinas Campinas Brazil CRF CRF CRF Curitiba Brazil CRF CRF CRF Hospital Amaral Carvalho Jau Brazil CRF CRF CRF Hospital de Porto Alegre Porto Alegre Brazil TED TED TED Rio de Janeiro Brazil CRF CRF CRF Albert Einstein Hospital Sao Paolo Brazil TED TED TED Hospital Sirio Libanes Sao Paolo Brazil CRF CRF CRF Instituto de Oncologia Pediatrica Sao Paolo Brazil TED TED TED Alberta Children's Hospital Calgary Canada CRF CRF CRF Tom Baker Cancer Centre Calgary Canada TED TED TED Queen Elizabeth II Health Sciences Center Halifax Canada TED TED TED Hamilton Health Sciences Hamilton Canada CRF CRF CRF Kingston General Hospital Kingston Canada N/A N/A TED Centre Hospitalier Montreal Canada CRF CRF CRF Maisonneuve - Rosemont Hospital Montreal Canada TED TED N/A McGill University Health Centre Royal Victoria Hospital Montreal Canada TED TED N/A Montreal Children's Hospital Montreal Canada TED TED TED St Anna Children's Hospital Hospital de Clinicas Curitiba Instituto Nacional de Cancer Page | 66 CIBMTR 2015 Annual Report Participating Center APPENDIX A2: INTERNATIONAL CENTERS City Country MUD RELATED AUTO Quebec Canada TED TED TED Quebec City Canada TED TED TED Saint John Regional Hospital Saint John Canada N/A N/A CRF St John's Health Sciences Center St. John's Canada N/A N/A TED Health Sciences North Sudbury Canada N/A N/A TED Princess Margaret Hospital (alloHCT) Toronto Canada CRF CRF N/A Princess Margaret Hospital (autoHCT) Toronto Canada N/A N/A CRF British Columbia Children's Hospital Vancouver Canada CRF CRF CRF Vancouver General Hospital Vancouver Canada N/A N/A N/A Winnipeg Canada CRF CRF CRF Barranquilla Colombia TED TED TED Clinica de Marly Bogota Colombia CRF CRF CRF Charles University Hospital - Pilsen Pilsen CRF CRF CRF Institute of Hem-Blood Transfusion Praha TED TED N/A University Hospital, Rigshospitalet Copenhagen Denmark CRF CRF N/A Children's Cancer Hospital - Egypt Cairo Egypt N/A TED TED NCI Cairo University Cairo Egypt TED TED N/A Helsinki Finland CRF CRF N/A Turku University Turku Finland TED TED N/A Centre Hospitalier Regional University D'Angers Angers France TED TED N/A Besançon France CRF CRF N/A Hotel-Dieu de Quebec CHA-Enfant-Jesus Hospital CancerCare Manitoba / University of Manitoba Instituto de Trasplante de Medula Osea de la Costa Caribe Helsinki University Central Hospital Hopital Jean Minjoz Czech Republic Czech Republic Page | 67 CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS Participating Center City Country MUD RELATED AUTO Hopital Claude Huriez, Lille Lille France MED-A MED-A N/A Hospital Edouard Herriot Lyon France MED-A MED-A N/A Institute Paoli Calmettes Marseille France CRF CRF N/A Paris France CRF CRF N/A Poitiers France CRF CRF N/A Essen Germany CRF CRF CRF University Children’s Hospital Frankfurt Frankfurt Germany CRF CRF CRF Albert-Ludwig University - Freiburg Freiburg Germany CRF CRF N/A UKE Hamburg, Klinik und Poliklinik für Stammzelltransplantation Hamburg Germany CRF CRF N/A Heidelberg Germany CRF CRF N/A Christian Albrechts University Kiel Germany CRF CRF CRF University Leipzig, BMT Center Leipzig Germany CRF N/A N/A University Hospital Mainz Mainz Germany TED TED TED Munich Germany CRF CRF N/A Regensburg Germany MED-A MED-A N/A UniversitÓ“ts Klinikum Tubingen Tübingen Germany CRF TED N/A UniversitÓ“ts Kinderklinik Tubingen Tübingen Germany CRF TED N/A Ulm Germany CRF CRF TED Wiesbaden Germany CRF CRF N/A Rio Patras Greece TED TED TED Shatin Hong Kong CRF CRF N/A Hospital Saint Louis Hospital Jean Bernard University Hospital of Essen University of Heidelberg University of Munich Klinikum der UniversitÓ“t Regensburg UniversitÓ“t Ulm - Adults Deutsche Klinik für Diagnostik Wiesbaden University Hospital of Patras, Patras University Medical Center Chinese University of Hong Kong Page | 68 CIBMTR 2015 Annual Report Participating Center APPENDIX A2: INTERNATIONAL CENTERS City Country MUD RELATED AUTO Ahmedabad India CRF CRF CRF Delhi India CRF CRF CRF Gandhinagar India N/A TED TED Gurgaon India N/A CRF CRF Ludhiana India TED TED TED New Delhi India CRF CRF CRF Puducherry India N/A TED TED Pune India CRF CRF CRF Vellore India CRF CRF N/A Haifa Israel CRF CRF CRF Haddasah University Hospital Jerusalem Israel CRF CRF N/A Davidoff Cancer Center, Rabin Medical Center, Beilinson Hospital Petah Tikva Israel CRF CRF CRF Tel-Aviv Sourasky Medical Center Tel-Aviv Israel CRF CRF CRF Israel CRF CRF CRF Israel CRF CRF N/A Gujrat Cancer & Research Institute Sir Ganga Ram Hospital Apollo Hospital International Ltd Fortis Memorial Research Institute Bone Marrow Transplant Unit, Christian Medical College, Ludhiana Rajiv Gandhi Cancer Institute and Research Centre Jawaharlal Institute of Postgraduate Medical Education and Research Sahyadri Speciality Hospital Christian Medical College Hospital Rambam Medical Center Chaim Sheba Medical Center Pediatrics Sheba Medical Center TelHashomer TelHashomer Instituto di Ematologia e Oncologia Medica Seragnoli Bologna Italy MED-A MED-A N/A University Bologna - Pediatrics Bologna Italy TED TED TED St Eugenio Hospital Rome Italy CRF CRF N/A Ospedale Molinette Torino Italy MED-A MED-A N/A University Torino Torino Italy TED TED N/A Osaka City University Osaka Japan CRF CRF N/A Page | 69 CIBMTR 2015 Annual Report APPENDIX A2: INTERNATIONAL CENTERS Participating Center City Country MUD RELATED AUTO Hospital Universitario Monterrey Mexico CRF CRF CRF Puebla Mexico TED TED TED Academic Medical Center Amsterdam Netherlands CRF TED TED VU Medical Center - Amsterdam Amsterdam Netherlands CRF TED TED University Medical Center Groningen Groningen Netherlands CRF N/A N/A Leiden Netherlands CRF TED N/A Academic Hospital Maastricht Maastricht Netherlands CRF TED N/A University Hospital of Nijmegen Nijmegen Netherlands CRF TED N/A Erasmus MC - Daniel den Hoed Cancer Center Rotterdam Netherlands CRF TED N/A University Hospital Utrecht Utrecht Netherlands CRF N/A N/A University Medical Center Utrecht Pediatrics Utrecht Netherlands CRF N/A N/A Auckland City Hospital Auckland New Zealand CRF CRF N/A Starship Children's Hospital Auckland New Zealand CRF CRF N/A Christchurch New Zealand CRF CRF TED Wellington Blood and Cancer Centre Wellington New Zealand CRF CRF N/A Rikshospitalet - The National Hospital Oslo Norway CRF N/A N/A Karachi Pakistan CRF CRF CRF Rawalpindi Pakistan N/A CRF CRF San Borja Peru N/A TED N/A Katowice Poland CRF CRF N/A Centro de Hematologia y Medicina Interna Clinica RUIZ de Puebla Leiden University Medical Center Christchurch Hospital Hill Park Hospital - National Institute of Blood Diseases and Bone Marrow Transplantation Armed Forces Bone Marrow Transplant Center Instituto Nacional Salud Del Niño San Borja Silesian Medical Academy Page | 70 CIBMTR 2015 Annual Report Participating Center APPENDIX A2: INTERNATIONAL CENTERS City Country MUD RELATED AUTO Poznan Poland MED-A MED-A N/A Wroclaw Poland CRF CRF N/A Lisbon Portugal CRF CRF N/A Porto Portugal N/A N/A N/A St. Petersburg Russian Federation TED TED TED Riyadh Saudi Arabia CRF CRF N/A Riyadh Saudi Arabia CRF CRF N/A Singapore Singapore CRF CRF CRF Singapore Singapore CRF CRF CRF Parkway Cancer Centre Singapore Singapore CRF CRF CRF Singapore General Hospital Singapore Singapore CRF CRF N/A Slovak Medical University Bratislava Slovak Republic TED TED N/A Constantiaberg Medi-Clinic Cape Town South Africa CRF CRF CRF University of Witwatersrand Parktown South Africa CRF CRF CRF Asan Medical Center Seoul South Korea CRF CRF N/A Samsung Medical Center Seoul South Korea CRF CRF CRF Seoul St. Mary's Hospital Catholic BMT Center Seoul South Korea CRF CRF CRF Barcelona Spain TED TED N/A Gregorio Maranon University General Hospital Madrid Spain CRF CRF CRF Hospital Puerta Hierro Madrid Spain MED-A MED-A N/A Poznan University of Medical Sciences Lower-Silesian Center for Cellular Transplantation and National Bone Marrow Donor Registry Institute Portugues de Oncologia Lisbon Institute Portugues de Oncologia Porto St Petersburg State Medical University King Faisal Specialist Hospital Pediatrics King Faisal Specialist Hospital & Research Center - Adults National University Health System Adults National University Health System Pediatrics University Barcelona Page | 71 CIBMTR 2015 Annual Report Participating Center APPENDIX A2: INTERNATIONAL CENTERS City Country MUD RELATED AUTO Santander Spain TED TED TED Valencia Spain CRF CRF N/A Gothenborg Sweden CRF CRF N/A Lund Sweden MED-A MED-A N/A Stockholm Sweden CRF CRF N/A Uppsala Sweden CRF CRF N/A Basel Kantonsspital Basel Switzerland CRF CRF N/A University Hospital-Zurich Zurich Switzerland MED-A MED-A N/A Chang Gung Children's Hospital Taoyuan Taiwan CRF CRF CRF King Chulalongkorn Memorial Hospital Bangkok Thailand TED TED TED Ankara University Faculty of Medicine Ankara Turkey TED TED N/A Gulhane Military Medical Academy Ankara Turkey CRF CRF CRF Medical Park Hospital - Antalya Istanbul Turkey CRF CRF CRF Birmingham Children's Hospital Birmingham TED TED N/A Queen Elizabeth Hospital Birmingham Birmingham CRF CRF N/A Bristol Children's Hospital Bristol CRF CRF N/A Addenbrooke's NHS Trust Cambridge CRF CRF N/A MED-A MED-A N/A CRF CRF N/A CRF CRF N/A Hospital Universitario Marques de Valdecilla Hospital Universitario La Fe Sahlgrenska University Hospital University Hospital of Lund Karolinska University Hospital, Centre for Allogeneic Stem Cell Transplantation University Hospital - Uppsala Beatson West of Scotland Cancer Centre Glasgow Royal Hospital for Sick Children Glasgow Imperial College / Hammersmith Hospital London United Kingdom United Kingdom United Kingdom United Kingdom United Kingdom United Kingdom United Kingdom Page | 72 CIBMTR 2015 Annual Report Participating Center APPENDIX A2: INTERNATIONAL CENTERS City Imperial College - St Mary's Hospital London The Royal Free Hampstead NHS Trust London Royal Marsden Hospital Sutton Country MUD RELATED AUTO United Kingdom United Kingdom United Kingdom CRF CRF N/A MED-A MED-A N/A N/A N/A N/A British Hospital Montevideo Uruguay CRF CRF CRF Centro de Trasplante del Servicio Medico Integral (SMI) Montevideo Uruguay CRF CRF CRF Hospital Maciel Montevideo Uruguay CRF CRF CRF Valencia Venezuela TED TED TED Ciudad Hospitalaraia Dr Enrique Tejera Page | 73 CIBMTR 2015 Annual Report APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND PERSONNEL APPENDIX B: COORDINATING CENTER ORGANIZATIONAL STRUCTURE AND LEADERSHIP The CIBMTR Coordinating Center resides on two campuses: one is located at MCW in Milwaukee, WI, and the other is located at NMDP/Be The Match in Minneapolis, MN. The Coordinating Center provides administrative, statistical, data management, clinical trials, IT, and personnel support for CIBMTR activities, and it benefits from a unique, collegial partnership with the Division of Biostatistics of MCW. CIBMTR Milwaukee has approximately 70 employees and is an academic division of the MCW Department of Medicine. The Milwaukee office receives administrative support from the MCW departments of Grants and Contracts, Development, Office of Technology Development, Public Affairs, Human Resources and the Department of Medicine Administration. CIBMTR Minneapolis has approximately 120 employees, and several NMDP/Be The Match departments provide support for CIBMTR activities, including Finance, Contracts, and Marketing & Communications. Page | 74 CIBMTR 2015 Annual Report APPENDIX B: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS APPENDIX B1: ORGANIZATIONAL STRUCTURE – MILWAUKEE CAMPUS Chief Scientific Director M Horowitz, MD, MS Administrator Medical Faculty CIBMTR Milwaukee P Steinert, PhD, MBA Program Director, Data Operations J Brunner-Grady, PA-C Grants and Contracts Financial Manager C Gonzalez Program Manager, SCTOD C Doleysh Meetings Program Manager D Waldoch Snyder 1 Program Manager, Business Office P Vespalec Program Coordinator II R Dunn Administrative Assistant, Sr K Jackson Administrative Assistant DL Campbell J Claas University of Minnesota NMDP/Be The Match 3 Fred Hutchinson Cancer Research Center 4 University of North Carolina 2 Study Clinical Research Coordinator S Kulkarni Clinical Research Assistant III C Abel Clinical Research Assistant II M Patel Data Entry A Pereles Program Director, Advancement S Fisher Manager, Data Operations S Meiers Program Coordinator II K Bhavsar Clinical Research Coordinator II A Prentice T Hunt Program Coordinator I A Pope Clinical Research Coordinator I A Benoit Program Director, Statistics & Clinical Outcomes Research W Pérez, MPH Medical Writer J Gillis-Smith Program Coordinator II M Brey M Simaytis Communications Specialist V Vlach Senior Biostatistician J Carreras M Chen H Wang Biostatistician K Bo-Subait A DiGilio M Fei N He M Hemmer K Hu J Huang H Millard A St. Martin X Zhu Program Coordinator I M Geronime Director, IT E Bergman, MBA, MS Program/IS Manager T Moerke Bus Systems Analyst Assoc TBD Information Security Analyst J Rineck Desktop Support Technician V Buendia Database Administrator B Liu Programmer Analyst IV H Tian Programmer Analyst III P Gengler J Gier Database Analyst III X Zhang Programmer Analyst II T Graczkowski Database Analyst I M Desai Metadata Analyst A Kummerow Manager IS I (part time) C Zhang Senior Research Advisor D Weisdorf, MD1 Senior Scientific Director L Burns, MD2 M Eapen, MD, MS S Lee, MD, MPH3 M Pasquini, MD, MS JD Rizzo, MD, MS B Shaw, MD, PhD Scientific Director M Arora, MD,MS1 A D’Souza, MBBS, MD M Hamadani, MD P Hari, MD, MS M Riches, MD, MS4 W Saber, MD, MS E Thiel, MD, MS Page | 75 CIBMTR 2015 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 1) Chief Scientific Director Mary M Horowitz, MD, MS Associate Scientific Director D Confer, MD Vice President, CIBMTR Minneapolis R King, MPH Director, CIBMTR IT Systems M Prestegaard Senior Manager, Program Management K Gee Project Manager S Freeman K O’Connor A Pull B Wakaruk Project Coordinator DM Campbell Principal Business Systems Analyst E Narr Senior Business Systems Analyst S Ewer Business Systems Analyst D Kloker Solutions Manager E Chan Senior System Administrator C Yang Business Intelligence Solutions Architect TBD Principal Software Engineer C Giddegowda Senior Software Engineer A Gomez N Hood J Smith Software Engineer Z Ahmed C Jordahl G Ogega Software Engineer ETL N Aryal S Stagg Associate System Administrator A Westin Director, Bioinformatics M Maiers, MS1 Director, Immunobiology and Observational Research S Spellman, MBS1 Data Solutions Manager R Renner Quality Assurance Manager V Yarra Senior Metadata Analyst W Zhang Metadata Analyst M Nych S Sorensen Senior Data Analyst D McDonell K Schaper T Wirth Senior Quality Assurance Analyst B Samba Quality Assurance Analyst V Murukurthy J Oakes Principal Data Architect E Zink Business Architect B Burgess Senior Biostatistician P Chitphakdiathai M Haagenson Biostatistician D Kiefer S Rouse Project Coordinator H Severance Administrative Specialist A Carlson Senior Bioinformatics Scientists A Madbouly E Williams Bioinformatics Scientists S Fingerson M Halagan D Roe Principal Immunobiology Research Scientist A Howard C Vierra-Green Senior Immunobiology Research Scientist M Brown Immunobiology Research Scientist C Brady D Scheller S Waldvogel Associate Immunobiology Research Scientist A Spahn 1 Also serves as Scientific Director Page | 76 CIBMTR 2015 Annual Report APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS APPENDIX B2: ORGANIZATIONAL STRUCTURE – MINNEAPOLIS CAMPUS (PART 2) Chief Scientific Director Mary M Horowitz, MD, MS Associate Scientific Director D Confer, MD Vice President, CIBMTR Minneapolis R King, MPH Senior Manager, Monitoring & Auditing D Christianson Survey Research Group Supervisor D Mattila BMT CTN Project Manager A Foley Senior Human Research Protection Specialist - IRB T Rouse Senior Human Research Protection Specialist C Jobe Human Research Protection Specialist J Tkachenko Administrative Specialist M Young Senior Manager, Prospective Research R Drexler Senior Clinical Research Associate W Affield A Birch M Petcoff L Wendland Clinical Research Associate A Hendrickson K Kane C Newcomb K Phalen M Proue K Reilly Clinical Research Specialist E Eich Audit Coordinator E Nordquist Research Interviewer C Griffith C Jacox C Kunakom S Lease TBD Survey Research Assistant E Michem Senior Clinical Research Specialist A Adams S Flesch C Johnson H Kobusingye Clinical Research Specialist R Besser C Petroske J Vogel Clinical Research Assistant TBD Prospective Research Coordinator J Dworski Senior Manager, Data Management M Matlack Supervisor, Clinical Research, Recipient Data Management K Gardner Supervisor, Quality Control B Levesque Supervisor, Clinical Research, Donor Data Management A Hauck Clinical Research Coordinator III E Johnson Clinical Research Coordinator II A Draxler A Mitsch A Mussetter K Nutter C Olson T Thole Clinical Research Coordinator I E Mitchem P Wallace Quality Control Specialist K Kutzner Data Entry Coordinator II L Horne Data Entry Coordinator I P Lee K Xong Imaging Assistant II N Gibitz T Winder Imaging Assistant J Lund Clinical Research Coordinator III A Ewer Clinical Research Coordinator II R Krunkkala K Malum S Tasky Clinical Research Coordinator III J Bloomquist S Logan E Love Training & Development Specialist L Colt Sr Research Developer M Unekis TBD Administrative Assistant M Ammi Page | 77 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL APPENDIX B3: COORDINATING CENTER LEADERSHIP SENIOR LEADERSHIP Mary M. Horowitz, MD, MS • Chief Scientific Director for the CIBMTR • Principal Investigator for the Data and Coordinating Center of the BMT CTN • Research Director for the SCTOD • Robert A. Uihlein Professor of Hematologic Research at MCW • Chief of the Division of Hematology and Oncology at MCW • Attending physician in the MCW HCT program Jeffrey Chell, MD • Executive Director for the CIBMTR • Chief Executive Officer of NMDP/Be The Match Dennis Confer, MD • Associate Scientific Director for CIBMTR Minneapolis • Co-PI of the Data and Coordinating Center of the BMT CTN • Co-Scientific Director of the RCI BMT • Scientific Director of the Donor Health and Safety Working Committee • Chief Medical Officer of NMDP/Be The Match • Treasurer of the Executive Board of the Worldwide Network for Blood and Marrow Transplantation Daniel Weisdorf, MD • Senior Research Advisor for the CIBMTR • Scientific Director of the Acute Leukemia Working Committee • Vice President of the Worldwide Network for Blood and Marrow Transplantation • Professor of Medicine at the University of Minnesota • Chief of the Division of Hematology, Oncology, and Transplant at the University of Minnesota • Associate Chair of Clinical Research in the Department of Medicine at the University of Minnesota • Attending physician in the University of Minnesota HCT program Page | 78 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL Mei-Jie Zhang, PhD • Chief Statistical Director for the CIBMTR • Biostatistician for the Acute Leukemia and Graft Sources and Manipulation Working Committees • Professor of Biostatistics at MCW Mary Eapen, MBBS, MS • Senior Scientific Director of Research Operations for the CIBMTR • Scientific Director for the Graft Sources and Manipulation Working Committee as well as the Primary Immune Deficiencies, Inborn Errors of Metabolism and Other Non-Malignant Marrow Disorders Working Committee • Protocol Officer for several BMT CTN trials • Professor of Medicine at MCW • Attending physician in the MCW HCT program J. Douglas Rizzo, MD, MS • Senior Scientific Director and Principal Investigator of the SCTOD for the CIBMTR • Professor of Medicine at MCW • Associate Director of Clinical Operations for the Froedtert and MCW Cancer Center • Attending physician in the MCW HCT program Bronwen Shaw MD, PhD • Senior Scientific Director of Data Operations for the CIBMTR • Co-Scientific Director of the RCI BMT • Scientific Director of the Late Effects and Quality of Life Working Committee as well as the Donor Health and Safety Working Committee • Professor of Medicine at MCW • Attending physician in the MCW HCT program Page | 79 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL Linda Burns, MD • Senior Scientific Director of the Health Services Research Program for the CIBMTR • Co-Scientific Director of the RCI BMT • Vice President and Medical Director of NMDP/Be The Match • Immediate Past President of the American Society of Hematology Marcelo Pasquini, MD, MS • Senior Scientific Director of CIBMTR Clinical Trials Support – BMT CTN for the CIBMTR • Scientific Director for the Autoimmune Diseases and Cellular Therapies Working Committee as well as the Regimen-Related Toxicity and Supportive Care Working Committee • Protocol Officer and Director of Medical Monitors for the BMT CTN • CIBMTR representative to the Worldwide Network for Blood and Marrow Transplantation • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program Roberta King, MPH • Vice President for CIBMTR Minneapolis • Oversees the administrative, scientific, and statistical support activities of CIBMTR Minneapolis, which include research administration, human subject protection program, data management, auditing and monitoring, observational research, prospective research, and IT • Staff Liaison to the NMDP/Be The Match Donor and Patient Safety Monitoring Advisory Group Patricia Steinert, PhD, MBA • Administrator for CIBMTR Milwaukee • Oversees the administrative, scientific, and statistical support activities of CIBMTR Milwaukee, which include data operations, development, observational research and prospective research, IT and meetings Page | 80 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL SCIENTIFIC DIRECTORS Mukta Arora, MD, MBBS, MS • Scientific Director of the Graft-versus-Host Disease Working Committee • Associate Professor of Medicine at the University of Minnesota • Attending physician in the University of Minnesota HCT program Anita D’Souza, MD • Assistant Scientific Director of the Plasma Cell Disorders and Adult Solid Tumors Working Committee • Assistant Professor of Medicine at MCW • Attending physician in the MCW HCT program Parameswaran Hari, MD, MS • Scientific Director of the Plasma Cell Disorders and Adult Solid Tumors Working Committee • Armand J. Quick – William F. Stapp Professor of Hematology at MCW • Section Head of Hematologic Malignancies and Adult BMT Program at MCW • Attending physician in the MCW HCT program Mehdi Hamadani, MD • Scientific Director of the Lymphoma Working Committee • Associate Professor of Medicine at MCW • Attending physician in the MCW HCT program Page | 81 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL Stephanie J. Lee, MD, MPH • Senior Scientific Director of Immunobiology Research for the CIBMTR • Scientific Director of the Immunobiology Working Committee • Professor of Medicine at the University of Washington • Attending physician in the Fred Hutchinson Cancer Research Center HCT program Martin Maiers, MS • Director of Bioinformatics Research for the CIBMTR • Staff liaison to the NMDP/Be The Match Histocompatibility Advisory Group (also known as the CIBMTR Immunobiology Steering Committee) • Co-Chair of Informatics: International Histocompatibility and Immunogenetics Workshop • Member of World Health Organization HLA Nomenclature Committee Marcie Riches, MD, MS • Scientific Director of the Infection and Immune Reconstitution Working Committee • Protocol Officer and Medical Monitor for several BMT CTN trials • Clinical Associate Professor of Medicine at the University of North Carolina at Chapel Hill • Director of BMT Clinical Research and Data Quality at the University of North Carolina at Chapel Hill • Attending physician in the University of North Carolina Lineberger Comprehensive Cancer Center HCT program Wael Saber, MD, MS • Scientific Director of the Chronic Leukemia and Health Services and International Studies Working Committees • Assistant Scientific Director of the Acute Leukemia Working Committee • Assistant Professor of Medicine at MCW • Attending physician in the MCW HCT program Page | 82 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL Stephen Spellman, MBS • Scientific Director of the Graft-versus-Host Disease and Immunobiology Working Committees • Director of Immunobiology and Observational Research for the CIBMTR • Principal Investigator for the Research Repository • Staff liaison to the NMDP/Be The Match Cord Blood Advisory Group Research subcommittee and Histocompatibility Advisory Group (also known as the CIBMTR Immunobiology Steering Committee) • Program Manager for the NMDP/Be The Match Office of Naval Research Grant Elizabeth Thiel, MD, MS • Scientific Director of the Pediatric Cancer Working Committee • Associate Professor of Medicine at MCW • Attending physician in the MCW Palliative Care program STATISTICAL DIRECTORS Kwang Woo Ahn, PhD • Biostatistician for the Chronic Leukemia, Infection and Immune Reconstitution, and Lymphoma Working Committees • Associate Professor of Biostatistics at MCW Ruta Brazauskas, PhD • Biostatistician for the Health Services and International Studies Working Committee as well as the Late Effects and Quality of Life and Autoimmune Disorders and Cellular Therapies Working Committees • Assistant Professor of Biostatistics at MCW Page | 83 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL Brent Logan, PhD • Biostatistician for the Donor Health and Safety Working Committee and the Regimen-Related Toxicity and Supportive Care Working Committee • Lead Statistician for the BMT CTN and Statistical Consultant to the NMDP/Be The Match • Professor of Biostatistics at MCW • Director of the Division of Biostatistics at MCW Tao Wang, PhD • Biostatistician for the Graft-versus-Host Disease and Immunobiology Working Committees • Associate Professor of Biostatistics at MCW OTHER LEADERSHIP STAFF Erik Bergman, MBA, MS • Director of IT for the CIBMTR in Milwaukee • Leads the IT staff in Milwaukee, which is organized in four teams: Database, Applications Development, Technology Services, and Project Management • Oversees management of the Research Database, including extraction of data from source systems as well as their transformation and loading to the database • Responsible for data retrievals from the Research Database as well as key solutions for sharing data with stakeholders Janet Brunner-Grady, PA-C • Program Director of Data Operations for the CIBMTR • Manages the clinical research coordinators, develops training programs, and monitors transplant center CPI • Assists clinical research coordinators on both campuses with clinical transplant-related questions Page | 84 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL Debra Christianson • Senior Manager of Auditing and Monitoring for the CIBMTR • Oversees the monitoring program for RCI BMT clinical trials and the on-site source document audits on data submitted to the Research Database • Responsible for the FormsNet Instruction Manual Rebecca Drexler • Senior Manager of Prospective Research for the CIBMTR • Manages the activities of the RCI BMT, including the Survey Research Group • Oversees the administration of the Clinical Trials Advisory Committee Sherry Fisher • Director of Advancement for the CIBMTR • Manages the advancement activities and the Corporate Program, which generates new revenue to create a continued source of nonfederal financial support • Oversees meetings and communications activities, including the annual BMT Tandem Meetings Marie Matlack • Senior Manager of Data Management for the CIBMTR • Manages clinical research coordinators and senior research programmers as well as data entry and imaging staff • Oversees form revision and development and the CPI program • Collaborates with the Minneapolis IT staff to develop enhancements to FormsNet Page | 85 CIBMTR 2015 Annual Report APPENDIX B3: COORDINATING CENTER PERSONNEL Waleska S. Pérez, MPH • Program Director of Statistics and Clinical Outcomes Research for the CIBMTR • Oversees the Master’s-level statisticians of CIBMTR Milwaukee • Provides administrative oversight of the Clinical Outcomes Research Program Matt Prestegaard • Director of IT for the CIBMTR in Minneapolis • Leads the IT staff in Minneapolis, including project managers, programmer analysts, business systems and data analysts, quality assurance analysts, metadata analysts, and managers • Develops, implements, and supports CIBMTR electronic data management, capture, and messaging systems (FormsNet and AGNIS); data marts and data warehouses; infrastructure for these applications; and the curation of common data elements within the Cancer Data Standards Registry and Repository Page | 86 CIBMTR 2015 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP APPENDIX C: COMMITTEE MEMBERSHIP CIBMTR committees provide input and advice to the leadership team, ensuring the continued support of both the needs and priorities of its scientific and medical communities. All committees and their functions are listed in Table 1.3. APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP The Advisory Committee provides oversight for CIBMTR policies and scientific agenda and also partners with the Working Committees to prioritize scientific studies. Members are elected to threeyear terms by the CIBMTR Assembly and must be from qualifying CRF transplant centers. All Advisory Committee terms begin on March 1. ELECTED MEMBERS Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA Immediate Past Chair Thomas Shea, MD, University of North Carolina at Chapel Hill, NC VICE CHAIRS North America Helen Heslop, MD, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, Birmingham, United Kingdom Central / South America Carmem Bonfim, MD, Hospital de Clinicas – UFPR, Curitiba, Brazil Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia MEMBERS AT LARGE North America Steven Pavletic, MD, MS, NIH-NCI Experimental Transplantation and Immunology Branch, Bethesda, MD Joseph Pidala, MD, MS, H Lee Moffitt Cancer Center and Research Institute, Tampa, FL Michael Pulsipher, MD, Primary Children’s Hospital, Salt Lake City, UT Bipin Savani, MD, Vanderbilt University Medical Center, Brentwood, TN Marcel van den Brink, MD, PhD, Memorial Sloan Kettering Cancer Center, New York, NY Kirsten Williams, MD, Children’s National Medical Center, Washington, DC Non-North America Ernst Holler, MD, Klinikum der Universitaet Regensburg, Germany William Hwang, MBBS, MRCP, Singapore General Hospital, Singapore Shinichiro Okamoto, MD, PhD, Keio University, Shinjuku-ku Tokyo, Japan Page | 87 CIBMTR 2015 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP Miguel Sanz, Hospital Universitario La Fe, Valencia, Spain Alok Srivastava, MD, Christian Medical College, Vellore, India Afonso Vigorito, MD, PhD, Unicamp – Hemocentro, Campinas, Brazil APPOINTED MEMBERS ASBMT Representative Christopher Bredeson, MD, MSc, The Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Canada Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA Business Representative Theresa Franco, MSN, BSN, Nebraska Medicine, Omaha, NE Collection Center Representative Lee Ann Weitekamp, MD, Michigan Blood Cord Blood Bank, Grand Rapids, MI Cord Blood Bank Representative Elizabeth Shpall, MD, MD Anderson Cord Blood Bank, Houston, TX Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN Patient / Family Representatives James Omel, MD, Chair, Consumer Advocacy Committee Maureen Beaman, MBA, Chair, Consumer Advocacy Committee EX OFFICIO MEMBERS Executive Director Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI Senior Scientific Director for SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI Associate Scientific Director CIBMTR Minneapolis Dennis Confer, MD, CIBMTR, Minneapolis, MN Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN Research Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI Vice President CIBMTR Minneapolis Roberta King, CIBMTR, Minneapolis, MN Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN NMDP / MCW / HRSA Contracting Officer Representative Shelley Grant, MHSA, Rockville, MD NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret) MCW / HRSA Contracting Officer Representative Christine Nishiguchi, MS, MPH, Rockville, MD Page | 88 CIBMTR 2015 Annual Report APPENDIX C1: ADVISORY COMMITTEE MEMBERSHIP MCW / NCI Project Officer Roy Wu, PhD, Bethesda, MD MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD Nominating Committee Chair Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH Page | 89 CIBMTR 2015 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP The Executive Committee, a subcommittee of the Advisory Committee, ensures that the organization carries out its mission and adheres to CIBMTR policies and procedures; it also provides advice and counsel to the Coordinating Center between meetings of the Advisory Committee. Specifically, the Executive Committee is responsible for providing scientific and policy advice to the Chief Scientific Director and Coordinating Center, reviewing audit results and making recommendations for improvement, and appointing a CIBMTR Co-Chair and additional CIBMTR representatives to the BMT Tandem Meetings Scientific Organizing Committee for the annual BMT Tandem Meetings. All Executive Committee terms begin on March 1. ELECTED MEMBERS Chair Paul Martin, MD, Fred Hutchinson Cancer Research Center, Seattle, WA Immediate Past Chair Thomas Shea, MD, University of North Carolina at Chapel Hill, NC VICE CHAIRS North America Helen Heslop, MD, Baylor College of Medicine Center for Cell and Gene Therapy, Houston, TX Europe Charles Craddock, MD, PhD, Queen Elizabeth Hospital, Birmingham, United Kingdom Central / South America Carmem Bonfim, MD, Hospital de Clinicas – UFPR, Curitiba, Brazil Asia / Africa / Australia Mahmoud Aljurf, MD, MPH, King Faisal Specialist Hospital Center & Research, Riyadh, Saudi Arabia APPOINTED MEMBERS ASBMT Representative Christopher Bredeson, MD, MSc, The Ottawa Hospital Blood & Marrow Transplant Program, Ottawa, Canada Bioethicist Steven Joffe, MD, MPH, Abramson Cancer Center University of Pennsylvania Medical Center, Philadelphia, PA Business Representative Theresa Franco, MSN, BSN, Nebraska Medicine, Omaha, NE Collection Center Representative Lee Ann Weitekamp, MD, Michigan Blood Cord Blood Bank, Grand Rapids, MI Cord Blood Bank Representative Elizabeth Shpall, MD, MD Anderson Cord Blood Bank, Houston, TX Donor Center Representative Jason Gangewere, NMDP/Be The Match, Minneapolis, MN Patient / Family Representatives James Omel, MD, Chair, Consumer Advocacy Committee Maureen Beaman, MBA, Chair, Consumer Advocacy Committee Page | 90 CIBMTR 2015 Annual Report APPENDIX C2: EXECUTIVE COMMITTEE MEMBERSHIP EX OFFICIO MEMBERS Executive Director Jeffrey Chell, MD, NMDP/Be The Match, Minneapolis, MN Chief Scientific Director Mary M. Horowitz, MD, MS, CIBMTR, Milwaukee, WI Chief Statistical Director Mei-Jie Zhang, PhD, CIBMTR, Milwaukee, WI Senior Scientific Director for SCTOD J. Douglas Rizzo, MD, MS, CIBMTR, Milwaukee, WI Associate Scientific Director CIBMTR Minneapolis Dennis Confer, MD, CIBMTR, Minneapolis, MN Senior Research Advisor Daniel Weisdorf, MD, CIBMTR, Minneapolis, MN Research Administrator CIBMTR Milwaukee Patricia Steinert, PhD, MBA, CIBMTR, Milwaukee, WI Vice President CIBMTR Minneapolis Roberta King, CIBMTR, Minneapolis, MN Vice President Patient Services Elizabeth Murphy, EdD, RN, NMDP/Be The Match, Minneapolis, MN NMDP / MCW /HRSA Contracting Officer Representative Shelley Grant, MHSA, Rockville, MD NMDP / Navy Project Officer Robert Hartzman, MD, Capt. MC, USN (ret) MCW / HRSA Contracting Officer Representative Christine Nishiguchi, MS, MPH, Rockville, MD MCW / NCI Project Officer Roy Wu, PhD, Bethesda, MD MCW / NHLBI Project Officer Nancy DiFronzo, PhD, Bethesda, MD MCW / NIAID Project Officer Linda Griffith, MD, PhD, Bethesda, MD Nominating Committee Chair Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH Page | 91 CIBMTR 2015 Annual Report APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP APPENDIX C3: CONSUMER ADVOCACY COMMITTEE MEMBERSHIP The Consumer Advocacy Committee communicates research results and data to the non-medical community and provides patient and donor perspectives during the development of the CIBMTR research agenda. Many committee members have personal experience as a donor, recipient, or family member of a recipient. CHAIRS James Omel, MD Maureen Beaman, MBA MEMBERS Beatrice Abetti, MSW, Leukemia and Lymphoma Society Jack Aiello, MS Gerardo Camarillo, JD, MD Anderson Cancer Center Jeffrey Haertling, ARK Air Express Hilary Hall, Dana-Farber Cancer Institute Barry Schatz, Cardinal Bernardin Cancer Center, Loyola University Medical Center Kristin Scheeler, University of Wisconsin Hospital and Clinics SCIENTIFIC DIRECTOR J. Douglas Rizzo, MD, MS, CIBMTR EX OFFICIO MEMBERS Robyn Ashton, RN, MSN, HRSA Jeffrey Chell, MD, NMDP/Be The Match Dennis Confer, MD, NMDP/Be The Match Ellen Denzen, (NMDP/Be The Match liaison) NMDP/Be The Match Carol Doleysh, (CIBMTR liaison) CIBMTR Rebecca Drexler, CIBMTR Jessica Gillis-Smith, MPH, CIBMTR Shelley Grant, MHSA, HRSA Darlene Haven, NMDP/Be The Match Mary Horowitz, MD, MS, CIBMTR Elizabeth Murphy, EdD, RN, (NMDP/Be The Match liaison) NMDP/Be The Match Christine Nishiguchi, MS, MPH, HRSA Patricia Steinert, PhD, MBA, CIBMTR Page | 92 CIBMTR 2015 Annual Report APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP APPENDIX C4: NOMINATING COMMITTEE MEMBERSHIP The Nominating Committee consists of five members elected by the CIBMTR Assembly. Following an annual call for nominations, the Nominating Committee prepares a slate of candidates for open positions on the Advisory, Nominating, and Clinical Trials Advisory Committees. Elections are held annually by confidential electronic ballot. The Nominating Committee also makes recommendations to the Advisory Committee for open Working Committee Chair and other leadership appointments after seeking recommendations from the CIBMTR Assembly, Advisory Committee, and incumbent Working Committee Chairs. All terms begin on March 1. CHAIR Stella Davies, PhD, MBBS, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH MEMBERS Daniel Couriel, MD, University of Michigan, Ann Arbor, MI Richard Champlin, MD, MD Anderson Cancer Center, Houston TX Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center, Seattle, WA David Vesole, MD, PhD, Hackensack University Medical Center, Hackensack, NJ Page | 93 CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP For information on Scientific Working Committee structure and organization, see Section 1.3.1. For information on Working Committee studies and their accomplishments, see Section 2.1.1. ACUTE LEUKEMIA WORKING COMMITTEE Chairs Marcos de Lima, MD, University Hospitals Case Medical Center Brenda Sandmaier, MD, Fred Hutchinson Cancer Research Center Hanna Khoury, MD, Emory University Hospital Scientific Director Daniel Weisdorf, MD, CIBMTR Asst Scientific Dir Wael Saber, MD, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Hailin Wang, MPH, CIBMTR AUTOIMMUNE DISEASES AND CELLULAR THERAPIES WORKING COMMITTEE Chairs Mitchell Cairo, MD, New York Medical College Ian Lewis, MBBS, PhD, Royal Adelaide Hospital David McKenna, MD, University of Minnesota Medical Center, Fairview Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Kyle Bo-Subait, MPH, CIBMTR CHRONIC LEUKEMIA WORKING COMMITTEE Chairs Edwin Alyea, MD, Dana Farber Cancer Institute Uday Popat, MD, MD Anderson Cancer Center Ronald Sobecks, MD, Cleveland Clinic Foundation Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Zhenhuan Hu, MS, CIBMTR DONOR HEALTH AND SAFETY WORKING COMMITTEE Chairs Paul O’Donnell, MD, PhD, Fred Hutchinson Cancer Research Center Michael Pulsipher, MD, Primary Children’s Hospital Galen Switzer, PhD, University of Pittsburgh Medical Center - Cancer Center Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Ex Officio Sr Advisor Dennis Confer, MD, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statisticians Deidre Kiefer, MPH, CIBMTR Pintip Chitphakdithai, PhD, CIBMTR Consumer Advocacy Committee Representatives Beatrice Abetti, MSW, Leukemia and Lymphoma Society Maureen Beaman, MBA Jeffrey Haertling, ARK Air Express Page | 94 CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP GRAFT SOURCES AND MANIPULATION WORKING COMMITTEE Chairs Miguel-Angel Perales, MD, Memorial Sloan Kettering Cancer Center Vanderson Rocha, MD, PhD, Churchill Hospital Asad Bashey, MD, PhD, The Blood and Marrow Transplant Program at Northside Hospital Scientific Director Mary Eapen, MBBS, MS, CIBMTR Statistical Director Mei-Jie Zhang, PhD, CIBMTR Statistician Andrew St. Martin, MS, CIBMTR GRAFT-VERSUS-HOST DISEASE WORKING COMMITTEE Chairs Daniel Couriel, MD, University of Michigan Amin Alousi, MD, MD Anderson Cancer Center Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center and Research Institute Scientific Directors Mukta Arora, MD, MS, CIBMTR Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Statistician Michael Hemmer, MS, CIBMTR Consumer Advocacy Committee Representatives Hilary Hall, Dana-Farber Cancer Institute James Omel, MD HEALTH SERVICES AND INTERNATIONAL STUDIES WORKING COMMITTEE Chairs Yoshiko Atsuta, MD, PhD, Nagoya University Graduate School of Medicine Carmem Bonfim, MD, Hospital de Clinicas Curitiba Jignesh Dalal, MD, The Children’s Mercy Hospitals and Clinics Theresa Hahn, PhD, Roswell Park Cancer Institute Nandita Khera, MD, Mayo Clinic Arizona and Phoenix Children's Hospital Scientific Director Wael Saber, MD, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Naya He, MPH, CIBMTR Consumer Advocacy Committee Representatives Jack Aiello, MS Gerardo Camarillo, JD, MD Anderson Cancer Center IMMUNOBIOLOGY WORKING COMMITTEE Chairs Michael Verneris, MD, University of Minnesota Medical Center, Fairview Katharina Fleischhauer, MD, Universitätsklinikum Essen KMT Katharine Hsu, MD, PhD, Memorial Sloan Kettering Cancer Center Scientific Directors Stephanie J. Lee, MD, MPH, CIBMTR, Fred Hutchinson Cancer Research Center Stephen Spellman, MBS, CIBMTR Statistical Director Tao Wang, PhD, CIBMTR Statistician Michael Haagenson, MS, CIBMTR Page | 95 CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP INFECTION AND IMMUNE RECONSTITUTION WORKING COMMITTEE Chairs Jeffery Auletta, MD, Nationwide Children’s Hospital Caroline Lindemans, MD, PhD, University Medical Center Utrecht Krishna Komanduri, MD, University of Miami Scientific Director Marcie Riches, MD, MS, CIBMTR, H Lee Moffitt Cancer Center and Research Institute Statistical Directors Kwang Woo Ahn, PhD, CIBMTR Soyoung Kim, PhD, CIBMTR Statistician Min Chen, MS, CIBMTR LATE EFFECTS AND QUALITY OF LIFE WORKING COMMITTEE Chairs Bipin Savani, MD, Vanderbilt University Medical Center Mary Flowers, MD, Fred Hutchinson Cancer Research Center Minoo Battiwalla, MD, MS, National Heart Lung and Blood Institute - NIH Scientific Director Bronwen Shaw, MD, PhD, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician Heather Millard, MPH, CIBMTR Consumer Advocacy Committee Representative Barry Schatz, Loyola University Medical Center LYMPHOMA WORKING COMMITTEE Chairs Sonali Smith, MD, University of Chicago Hospitals Anna Sureda, MD, PhD Timothy Fenske, MD, MS, Froedtert Memorial Lutheran Hospital Scientific Director Mehdi Hamadani, MD, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Alyssa DiGilio, MS, CIBMTR PEDIATRIC CANCER WORKING COMMITTEE Chairs Gregory Hale, MD, All Children’s Hospital Parinda Mehta, MD, Cincinnati Children's Hospital Medical Center Angela Smith, MD, MS, University of Minnesota Medical Center, Fairview Scientific Director Elizabeth Thiel, MS, CIBMTR Statistical Director Kwang Woo Ahn, PhD, CIBMTR Statistician Heather Millard, MPH, CIBMTR Page | 96 CIBMTR 2015 Annual Report APPENDIX C5: SCIENTIFIC WORKING COMMITTEE LEADERSHIP PLASMA CELL DISORDERS AND ADULT SOLID TUMORS WORKING COMMITTEE Chairs Amrita Krishnan, MD, City of Hope National Medical Center Cristina Gasparetto, MD, Duke University Medical Center Yago Nieto, MD, PhD, MD Anderson Cancer Center Tomer Mark, MD, New York Presbyterian Hospital at Cornell Scientific Director Parameswaran Hari, MD, MS, CIBMTR Asst Scientific Dir Anita D’Souza, MD, CIBMTR Statistical Directors Mei-Jie Zhang, PhD, CIBMTR Raphael Fraser, PhD, CIBMTR Statistician Jiaxing Huang, MS, CIBMTR Consumer Advocacy Committee Representative James Omel, MD PRIMARY IMMUNE DEFICIENCIES, INBORN ERRORS OF METABOLISM, AND OTHER NON-MALIGNANT MARROW DISORDERS WORKING COMMITTEE Chairs Paolo Anderlini, MD, MD Anderson Cancer Center Neena Kapoor, MD, Children’s Hospital of Los Angeles Jaap-Jan Boelens, MD, PhD, University Medical Center Utrecht Vikram Mathews, MD, Christian Medical College Hospital Scientific Director Mary Eapen, MBBS, MS, CIBMTR Statistical Director Ruta Brazauskas, PhD, CIBMTR Statistician TBD REGIMEN-RELATED TOXICITY AND SUPPORTIVE CARE WORKING COMMITTEE Chairs Andrew Artz, MD, MS, University of Chicago Hospitals Alison Loren, MD, MS, Abramson Cancer Center University of Pennsylvania Medical Center Shin Mineishi, MD, University of Alabama at Birmingham Scientific Director Marcelo Pasquini, MD, MS, CIBMTR Statistical Director Brent Logan, PhD, CIBMTR Statistician Xiaochun Zhu, MS, CIBMTR Page | 97 CIBMTR 2015 Annual Report APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP APPENDIX C6: IMMUNOBIOLOGY STEERING COMMITTEE MEMBERSHIP The NMDP/Be The Match Histocompatibility Advisory Group also serves as the CIBMTR Immunobiology Steering Committee. This committee reviews and approves the use of donorrecipient specimens from the Research Repository in CIBMTR studies. CHAIR Carolyn K. Hurley, PhD, Diplomate ABHI, Georgetown University Hospital ADVISORY GROUP MEMBERS Juliet Barker, MD, Memorial Sloan Kettering Cancer Center Sarah Cooley, MD, University of Minnesota Mary Eapen, MD, MS, Medical College of Wisconsin Marcelo Fernandez-Viña, PhD, Stanford Hospital and Clinics Brent Logan, PhD, Medical College of Wisconsin Carlheinz Mueller, MD, PhD, German National Bone Marrow Donor Registry (ZKRD) Joseph Pidala, MD, PhD, H. Lee Moffitt Cancer Center David Porter, MD, Abramson Cancer Center University of Pennsylvania Medical Center Raja Rajalingham, PhD, University of California, San Francisco Bronwen Shaw, MD, PhD, Medical College of Wisconsin EX OFFICIO MEMBERS Dennis Confer, MD, NMDP/Be The Match Jason Dehn, MPH, NMDP/Be The Match Karen Dodson, NMDP/Be The Match Melissa Greenwald, MD, HRSA Robert Hartzman, MD, Capt. MC, USN (Ret.), Navy Representative, C.W. Bill Young Marrow Donor Recruitment and Research Program Martin Maiers, CIBMTR Stephen Spellman, MBS, CIBMTR John Wingard, MD, NMDP/Be The Match Board Chair Page | 98 CIBMTR 2015 Annual Report APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP APPENDIX C7: CLINICAL TRIALS ADVISORY COMMITTEE MEMBERSHIP The Clinical Trials Advisory Committee assists in the review, approval, and oversight of proposals and protocols for Phase I and Phase II clinical trials submitted to the RCI BMT (Section 2.3.2). CHAIR John Levine, MD, MS, Mount Sinai Medical Center MEMBERS Colleen Delaney, MD, MSc, Fred Hutchinson Cancer Research Center Marcos de Lima, MD, University Hospitals Case Medical Center Hugo Fernandez, MD, H Lee Moffitt Cancer Center and Research Institute John Koreth, MBBS, DPhil, Dana Farber Cancer Institute Mark Litzow, MD, Mayo Clinic Rochester Margaret MacMillan, MD, MSc, University of Minnesota Medical Center, Fairview Sophie Paczesny, MD, PhD, Indiana University Hospital/Riley Hospital for Children Katayoun Rezvani, MD, PhD, M.D. Anderson Cancer Center Jan Storek, MD, PhD, University of Calgary APPOINTED MEMBERS Maureen Beaman, MBA James Omel, MD EX OFFICIO MEMBERS Dennis Confer, MD, CIBMTR Rebecca Drexler, BS, CIBMTR Robert Hartzman, MD, Capt MC, USN (Ret.) Mary Horowitz, MD, MS, CIBMTR Roberta King, CIBMTR Brent Logan, PhD, CIBMTR Nancy Poland, MA, NMDP/Be The Match Operations Marcie Riches, MD, MS, The University of North Carolina at Chapel Hill Kevin Weber, NMDP/Be The Match Operations Daniel Weisdorf, MD, CIBMTR Page | 99 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS APPENDIX D: PUBLICATIONS The PMCID number is assigned by PubMed Central, the NIH’s free digital archive of biomedical and life sciences journal literature, and is in compliance with the NIH policy on public access. APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS The following publications were generated by Scientific Working Committees within the Clinical Outcomes Research Program. For more information about the Working Committees, see Section 2.1.1. SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Logan AC, Wang Z, Alimoghaddam K, Wong RM, Lai T, Negrin RS, Grumet C, Logan BR, Zhang M-J, Spellman SR, Lee SJ, Miklos DB ABO mismatch is associated with increased nonrelapse mortality after allogeneic hematopoietic cell transplantation PMC4363312 Gadalla SM, Wang T, Haagenson M, Spellman SR, Lee SJ, Williams KM, Wong JY, De Vivo I, Savage SA Association between donor leukocyte telomere length and survival after unrelated allogeneic hematopoietic cell transplantation for severe aplastic anemia Impact of conditioning regimen on outcomes for patients with lymphoma undergoing high-dose therapy with autologous hematopoietic cell transplantation Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):746-754. doi:10.1016/j.bbmt. 2014.12.036. Epub 2015 Jan 5 JAMA: The Journal of the American Medical Association. 2015 Feb 10; 313(6):594-602. doi:10.1001/ jama.2015.7. Epub 2015 Feb 10 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):10461053. doi:10.1016/ j.bbmt.2015.02.005. Epub 2015 Feb 13 Chen Y-B, Lane AA, Logan BR, Zhu X, Akpek G, Aljurf MD, Artz AS, Bredeson CN, Cooke KR, Ho VT, Lazarus HM, Olsson RF, Saber W, McCarthy PL, Pasquini MC PMC4388056 PMC4426014 Page | 100 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Veys PA, Nanduri V, Baker KS, He W, Bandini G, Biondi A, Dalissier A, Davis JH, Eames GM, Egeler RM, Filipovich AH, Fischer A, Jürgens H, Krance R, Lanino E, Leung WH, Matthes S, Michel G, Orchard PJ, Pieczonka A, Ringdén O, Schlegel PG, Sirvent A, Vettenranta K, Eapen M Inamoto Y, Shah NN, Savani BN, Shaw BE, Abraham AA, Ahmed IA, Akpek G, Atsuta Y, Baker KS, Basak GW, Bitan M, DeFilipp Z, Gregory TK, Greinix HT, Hamadani M, Hamilton BK, Hayashi RJ, Jacobsohn DA, Kamble RT, Kasow KA, Khera N, Lazarus HM, Malone AK, LupoStanghellini MT, Margossian SP, Muffly LS, Norkin M, Ramanathan M, Salooja N, Schoemans H, Wingard JR, Wirk B, Wood WA, Yong A, Duncan CN, Flowers MED, Majhail NS Holter-Chakrabarty JL, Pierson N, Zhang M-J, Zhu X, Akpek G, Aljurf MD, Artz AS, Baron F, Bredeson CN, Dvorak CC, Epstein RB, Lazarus HM, Olsson RF, Selby GB, Williams KM, Cooke KR, Pasquini MC, McCarthy PL Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning British Journal of PMC4433436 Haematology. 2015 Jun 1; 169(5):711-718. doi:10.1111/bjh.13347. Epub 2015 Mar 27 Secondary solid cancer screening following hematopoietic cell transplantation Bone Marrow Transplantation. 2015 Aug 1; 50(8):10131023. doi:10.1038/ bmt.2015.63. Epub 2015 Mar 30 N/A The sequence of cyclophosphamide and myeloablative total body irradiation in hematopoietic cell transplantation for patients with acute leukemia Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1251-1257. doi:10.1016/j.bbmt. 2015.03.017. Epub 2015 Mar 31 PMC4465990 Page | 101 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Sorror ML, Logan BR, Zhu X, Rizzo JD, Cooke KR, McCarthy PL, Ho VT, Horowitz MM, Pasquini MC Prospective validation of the predictive power of the hematopoietic cell transplantation comorbidity index: a Center for International Blood and Marrow Transplant Research study PMC4512746 Mehta PA, Zhang M-J, Eapen M, He W, Seber A, Gibson B, Camitta BM, Kitko CL, Dvorak CC, Nemecek ER, Frangoul HA, Abdel-Azim H, Kasow KA, Lehmann L, Gonzalez Vicent M, Diaz Pérez MA, Ayas M, Qayed M, Carpenter PA, Jodele S, Lund TC, Leung WH, Davies SM Transplant outcomes for children with hypodiploid acute lymphoblastic leukemia Olsson RF, Logan BR, Chaudhury S, Zhu X, Akpek G, Bolwell BJ, Bredeson CN, Dvorak CC, Gupta V, Ho VT, Lazarus HM, Marks DI, Ringdén OTH, Pasquini MC, Schriber JR, Cooke KR Goyal SD, Zhang MJ, Wang HL, Akpek G, Copelan EA, Freytes C, Gale RP, Hamadani M, Inamoto Y, Kamble RT, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Reshef R, Ritchie DS, Saber W, Savani BN, Seber A, Shea TC, Tallman MS, Wirk B, Bunjes DW, Devine SM, de Lima M, Weisdorf DJ, Uy GL Primary graft failure after myeloablative allogeneic hematopoietic cell transplantation for hematologic malignancies Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):14791487. doi:10.1016/ j.bbmt.2015.04.004. Epub 2015 Apr 7 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1273-1277. doi:10.1016/j.bbmt. 2015.04.008. Epub 2015 Apr 10 Leukemia. 2015 Aug 1; 29(8):1754-1762. doi:10.1038/leu. 2015.75. Epub 2015 Apr 24 Bone Marrow Transplantation. 2015 Aug 1; 50(8):10571062. doi:10.1038/ bmt.2015.82. Epub 2015 Apr 27 PMC4527880 Allogeneic hematopoietic cell transplant for AML: no impact of pretransplant extramedullary disease on outcome PMC4465998 PMC4527886 Page | 102 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Sobecks RM, Wang T, Askar M, Gallagher MM, Haagenson M, Spellman S, Fernandez-Vina M, Malmberg K-J, Müller C, Battiwalla M, Gajewski J, Verneris MR, Ringdén O, Marino S, Davies S, Dehn J, Bornhäuser M, Inamoto Y, Woolfrey A, Shaw P, Pollack M, Weisdorf D, Milller J, Hurley C, Lee SJ, Hsu K Bachanova V, Burns LJ, Ahn KW, Laport GG, Akpek G, Kharfan-Dabaja MA, Nishihori T, Agura E, Armand P, Jaglowski SM, Cairo MS, Cashen AF, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Ghosh N, Holmberg LA, Inwards DJ, Kanate AS, Lazarus HM, Malone AK, Munker R, Mussetti A, Norkin M, Prestidge TD, Rowe JM, Satwani P, Siddiqi T, Stiff PJ, William BM, Wirk B, Maloney DG, Smith SM, Sureda AM, Carreras J, Hamadani M Impact of KIR and HLA genotypes on outcomes after reduced-intensity conditioning hematopoietic cell transplantation Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):15891596. doi:10.1016/ j.bbmt.2015.05.002. Epub 2015 May 8 PMC4537837 Impact of pretransplantation 18Ffluorodeoxy glucosepositron emission tomography status on outcomes after allogeneic hematopoietic cell transplantation for non-Hodgkin Lymphoma Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):16051611. doi:10.1016/ j.bbmt.2015.05.007. Epub 2015 May 14 PMC4558181 Page | 103 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Urbano-Ispizua A, Pavletic SZ, Flowers ME, Klein JP, Zhang M-J, Carreras J, Montoto S, Perales M-A, Aljurf MD, Akpek G, Bredeson CN, Costa LJ, Dandoy C, Freytes CO, Fung HC, Gale RP, Gibson J, Hamadani M, Hayashi RJ, Inamoto Y, Inwards DJ, Lazarus HM, Maloney DG, Martino R, Munker R, Nishihori T, Olsson RF, Rizzieri DA, Reshef R, Saad A, Savani BN, Schouten HC, Smith SM, Socié G, Wirk B, Yu LC, Saber W Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, AlSeraihy A, Ayas M, Bonfim CM, Boulad F, Lund T, Buchbinder DK, Kapoor N, O'Brien TA, Diaz Perez MA, Veys PA, Eapen M Hahn T, Sucheston-Campbell LE, Preus L, Zhu X, Hansen JA, Martin PJ, Yan L, Liu S, Spellman S, Tritchler D, Clay A, Onel K, Pasquini M, McCarthy PL The impact of graftversus-host disease on the relapse rate in patients with lymphoma depends on the histological subtype and the intensity of the conditioning regimen Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):17461753. doi:10.1016/ j.bbmt.2015.05.010. Epub 2015 May 15 PMC4568162 Hematopoietic stem cell transplantation for infantile osteopetrosis Blood. 2015 Jul 9; 126(2):270-276. doi:10.1182/blood2015-01-625541. Epub 2015 May 26 PMC4497967 Establishment of definitions and review process for consistent adjudication of causespecific mortality after allogeneic unrelateddonor hematopoietic cell transplantation Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Sep 1; 21(9):16791686. doi:10.1016/ j.bbmt.2015.05.019. Epub 2015 May 29 PMC4537799 Page | 104 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Inamoto Y, Flowers MED, Wang T, Urbano-Ispizua A, Hemmer MT, Cutler CS, Couriel DR, Alousi AM, Antin JH, Gale RP, Gupta V, Hamilton BK, Kharfan-Dabaja MA, Marks DI, Ringdén OTH, Socié G, Solh MM, Akpek G, Cairo MS, Chao NJ, Hayashi RJ, Nishihori T, Reshef R, Saad A, Shah A, Teshima T, Tallman MS, Wirk B, Spellman SR, Arora M, Martin PJ Verneris MR, Lee SJ, Ahn KW, Wang H-L, Battiwalla M, Inamoto Y, Fernandez-Vina MA, Gajewski J, Pidala J, Munker R, Aljurf M, Saber W, Spellman S, Koreth J Tacrolimus versus cyclosporine after hematopoietic cell transplantation for acquired aplastic anemia Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):17761782. doi:10.1016/ j.bbmt.2015.05.023. Epub 2015 May 30 PMC4568149 HLA mismatch Is associated with worse outcomes after unrelated donor reduced-intensity conditioning hematopoietic cell transplantation: an analysis from the Center for International Blood and Marrow Transplant Research Comparison of characteristics and outcomes of trial participants and nonparticipants: example of Blood and Marrow Transplant Clinical Trials Network 0201 trial Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):17831789. doi:10.1016/ j.bbmt.2015.05.028. Epub 2015 Jun 6 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):18151822. doi:10.1016/ j.bbmt.2015.06.004. Epub 2015 Jun 11 PMC4568127 Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ PMC4568172 Page | 105 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Ayas M, Eapen M, LeRademacher J, Carreras J, Abdel-Azim H, Alter BP, Anderlini P, Battiwalla M, Bierings M, Buchbinder DK, Bonfim C, Camitta BM, Fasth AL, Gale RP, Lee MA, Lund TC, Myers KC, Olsson RF, Page KM, Prestidge TD, Radhi M, Shah AJ, Schultz KR, Wirk B, Wagner JE, Deeg HJ Shaw BE, Logan BR, Kiefer DM, Chitphakdithai P, Pedersen TL, Abdel-Azim H, Abidi MH, Akpek G, Diaz MA, Artz AS, Dandoy C, Gajewski JL, Hematti P, Kamble RT, Kasow KA, Lazarus HM, Liesveld JL, Majhail NS, O'Donnell PV, Olsson RF, Savani BN, Schears RM, Stroncek DF, Switzer GE, Williams EP, Wingard JR, Wirk BM, Confer DL, Pulsipher MA Ciurea SO, Zhang M-J, Bacigalupo AA, Bashey A, Appelbaum FR, Aljitawi OS, Armand P, Antin JH, Chen J, Devine SM, Fowler DH, Luznik L, Nakamura R, O'Donnell PV, Perales M-A, Pingali SR, Porter DL, Riches MR, Ringdén OTH, Rocha V, Vij R, Weisdorf DJ, Champlin RE, Horowitz MM, Fuchs EJ, Eapen M Second allogeneic hematopoietic cell transplantation for patients with Fanconi anemia and bone marrow failure Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):17901795. doi:10.1016/ j.bbmt.2015.06.012. Epub 2015 Jun 23 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):18301838. doi:10.1016/ j.bbmt.2015.06.013. Epub 2015 Jun 23 PMC4568139 Blood. 2015 Aug 20; 126(8):1033-1040. doi:10.1182/blood2015-04-639831. Epub 2015 Jun 30 PMC4543223 Analysis of the effect of race, socioeconomic status, and center size on unrelated National Marrow Donor Program donor outcomes: donor toxicities are more common at low-volume bone marrow collection centers Haploidentical transplant with posttransplant cyclophosphamide vs matched unrelated donor transplant for acute myeloid leukemia PMC4568129 Page | 106 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Satwani P, Ahn KW, Carreras J, Abdel-Azim H, Cairo MS, Cashen A, Chen AI, Cohen JB, Costa LJ, Dandoy C, Fenske TS, Freytes CO, Ganguly S, Gale RP, Ghosh N, Hertzberg MS, Hayashi RJ, Kamble RT, Kanate AS, Keating A, Kharfan-Dabaja MA, Lazarus HM, Marks DI, Nishihori T, Olsson RF, Prestidge TD, Rolon JM, Savani BN, Vose JM, Wood WA, Inwards DJ, Bachanova V, Smith SM, Maloney DG, Sureda A, Hamadani M Klyuchnikov E, Bacher U, Kröger NM, Hari PN, Ahn KW, Carreras J, Bachanova V, Bashey A, Cohen JB, D'Souza A, Freytes CO, Gale RP, Ganguly S, Hertzberg MS, Holmberg LA, KharfanDabaja MA, Klein A, Ku GH, Laport GG, Lazarus HM, Miller AM, Mussetti A, Olsson RF, Slavin S, Usmani SZ, Vij R, Wood WA, Maloney DG, Sureda AM, Smith SM, Hamadani M Petersdorf EW, Malkki M, O'hUigin C, Carrington M, Gooley T, Haagenson MD, Horowitz MM, Spellman SR, Wang T, Stevenson P A prognostic model predicting autologous transplantation outcomes in children, adolescents and young adults with Hodgkin lymphoma Bone Marrow Transplantation. 2015 Nov 1; 50(11):14161423. doi:10.1038/ bmt.2015.177. Epub 2015 Aug 3 PMC4633349 Reduced-intensity allografting as first transplantation approach in relapsed/refractory grades one and two follicular lymphoma provides improved outcomes in long-term survivors Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):20912099. doi:10.1016/ j.bbmt.2015.07.028. Epub 2015 Aug 4 PMC4639453 High HLA-DP expression and graft-versus-host disease PMC4560117 Knight JM, Rizzo JD, Logan BR, Wang T, Arevalo JM, Ma J, Cole SW Low socioeconomic status, adverse gene expression profiles, and clinical outcomes in hematopoietic stem cell transplant recipients New England Journal of Medicine. 2015 Aug 13; 373(7):599-609. doi:10.1056/ NEJMoa1500140. Epub 2015 Aug 13 Clinical Cancer Research. Epub 2015 Aug 18 [PMC Journal – In Process] Page | 107 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Uy GL, Costa LJ, Hari PN, Zhang M-J, Huang J-X, Anderson KC, Bredeson CN, Callander NS, Cornell RF, Perez MAD, Dispenzieri A, Freytes CO, Gale RP, Garfall A, Gertz MA, Gibson J, Hamadani M, Lazarus HM, Kalaycio ME, Kamble RT, Kharfan-Dabaja MA, Krishnan AY, Kumar SK, Kyle RA, Landau HJ, Lee CH, Maiolino A, Marks DI, Mark TM, Munker R, Nishihori T, Olsson RF, Ramanathan M, Rodriguez TE, Saad AA, Savani BN, Schiller GJ, Schouten HC, Schriber JR, Scott E, Seo S, Sharma M, Ganguly S, Stadtmauer EA, Tay J, To LB, Vesole DH, Vogl DT, Wagner JL, Wirk B, Wood WA, D'Souza A Burke MJ, Verneris MR, Le Rademacher J, He W, AbdelAzim H, Abraham AA, Auletta JJ, Ayas M, Brown VI, Cairo MS, Chan KW, Diaz Perez MA, Dvorak CC, Egeler RM, Eldjerou L, Frangoul H, Guilcher GMT, Hayashi RJ, Ibrahim A, Kasow KA, Leung WH, Olsson RF, Pulsipher MA, Shah N, Shah NN, Thiel E, Talano JA, Kitko CL Contribution of chemotherapy mobilization to disease control in multiple myeloma treated with autologous hematopoietic cell transplantation Bone Marrow Transplantation. doi:10.1038/ bmt.2015.190. Epub 2015 Aug 24 PMC4548821 Transplant outcomes for children with T cell acute lymphoblastic leukemia in second remission: a report from the Center for International Blood and Marrow Transplant Research Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):21542159. doi:10.1016/ j.bbmt.2015.08.023. Epub 2015 Aug 29 PMC4654112 Page | 108 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Ponce DM, Eapen M, Sparapani R, O'Brien TA, Chan KW, Chen J, Craddock J, Schultz KR, Wagner JE, Perales M-A, Barker JN In vivo T cell depletion with myeloablative regimens on outcomes after cord blood transplantation for acute lymphoblastic leukemia in children Citation Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Dec 1; 21(12):21732179. doi:10.1016/ j.bbmt.2015.08.022. Epub 2015 Aug 29 Pasquini MC, Zhang M-J, Hematopoietic cell Biology of Blood and Medeiros BC, Armand P, Hu transplantation outcomes Marrow Z-H, Nishihori T, Aljurf MD, in monosomal karyotype Transplantation: Akpek G, Cahn J-Y, Cairo MS, myeloid malignancies Journal of the Cerny J, Copelan EA, Deol A, American Society for Freytes CO, Gale RP, Ganguly Blood and Marrow S, George B, Gupta V, Hale Transplantation. GA, Kamble RT, Klumpp TR, doi:10.1016/ Lazarus HM, Luger SM, j.bbmt.2015.08.024. Liesveld JL, Litzow MR, Marks Epub 2015 Aug 29 DI, Martino R, Norkin M, Olsson RF, Oran B, Pawarode A, Pulsipher MA, Ramanathan M, Reshef R, Saad AA, Saber W, Savani BN, Schouten HC, Ringdén O, Tallman MS, Uy GL, Wood WA Jr, Wirk B, Pérez WS, Batiwalla M, Weisdorf DJ Jindra PT, Conway SE, Analysis of a genetic Biology of Blood and Ricklefs SM, Porcella SF, polymorphism in the Marrow Anzick SL, Haagenson M, costimulatory molecule Transplantation: Wang T, Spellman S, Milford TNFSF4 with HSCT Journal of the E, Kraft P, McDermott DH, outcomes American Society for Abdi R Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.08.037. Epub 2015 Sep 5 PMCID PMC4639413 [PMC Journal – In Process] [PMC Journal – In Process] Page | 109 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID D'Souza A, Dispenzieri A, Wirk B, Zhang M-J, Huang J, Gertz MA, Kyle RA, Kumar S, Comenzo RL, Gale RP, Lazarus HM, Savani BN, Cornell RF, Weiss BM, Vogl DT, Freytes CO, Scott EC, Landau HJ, Moreb JS, Costa LJ, Ramanathan M, Callander NS, Kamble RT, Olsson RF, Ganguly S, Nishihori T, Kindwall-Keller TL, Wood WA, Mark TM, Hari P Ehrhardt MJ, Brazauskas R, He W, Rizzo JD, Shaw BE Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: a Center for International Blood and Marrow Transplant Research study Journal of Clinical Oncology. 2015 Nov 10; 33(32):3741-3749. doi:10.1200/JCO. 2015.62.4015. Epub 2015 Sep 14 [PMC Journal – In Process] Survival of patients who develop solid tumors following hematopoietic stem cell transplantation PMC4570237 Klyuchnikov E, Bacher U, Woo Ahn K, Carreras J, Kröger NM, Hari PN, Ku GH, Ayala E, Chen AI, Chen Y-B, Cohen JB, Freytes CO, Gale RP, Kamble RT, KharfanDabaja MA, Lazarus HM, Martino R, Mussetti A, Savani BN, Schouten HC, Usmani SZ, Wiernik PH, Wirk B, Smith SM, Sureda A, Hamadani M Kollman C, Spellman SR, Zhang M-J, Hassebroek A, Anasetti C, Antin JH, Champlin RE, Confer D, DiPersio JF, Fernandez-Viña M, Hartzman RJ, Horowtiz MM, Hurley CK, Karanes C, Maiers M, Mueller CR, Perales M-A, Setterholm M, Woolfrey AE, Yu N, Eapen M Long-term survival outcomes of reducedintensity allogeneic or autologous transplantation in relapsed grade 3 follicular lymphoma Bone Marrow Transplantation. doi:10.1038/bmt. 2015.203. Epub 2015 Sep 14 Bone Marrow Transplantation. doi:10.1038/bmt. 2015.223. Epub 2015 Oct 5 Blood. doi:10.1182/blood2015-08-663823. Epub 2015 Nov 2 [PMC Journal – In Process] The effect of donor characteristics on survival after unrelated donor transplantation for hematologic malignancy [PMC Journal – In Process] Page | 110 CIBMTR 2015 Annual Report APPENDIX D1: SCIENTIFIC WORKING COMMITTEE PUBLICATIONS SCIENTIFIC WORKING COMMITTEE PUBLICATIONS Authors Title Citation PMCID Riches ML, Trifilio S, Chen M, Ahn KW, Langston A, Lazarus HM, Marks DI, Martino R, Maziarz RT, Papinicolou GA, Wingard JR, Young J-AH, Bennett CL Anthias C, Shaw BE, Kiefer DM, Liesveld JL, Yared J, Kamble RT, D'Souza A, Hematti P, Seftel MD, Norkin M, DeFilipp ZM, Kasow KA, Abidi MH, Savani BN, Shah NN, Anderlini P, Diaz MA, Malone AK, Halter JP, Lazarus HM, Logan BR, Switzer GE, Pulsipher MA, Confer DL, O'Donnell PV Kanate AS, Mussetti A, Kharfan-Dabaja MA, Ahn KW, DiGilio A, Beitinjaneh A, Chhabra S, Fenske TS, Freytes C, Gale RP, Ganguly S, Hertzberg M, Klyuchnikov E, Lazarus HM, Olsson R, Perales M-A, Rezvani A, Riches M, Saad A, Slavin S, Smith SM, Sureda A, Yared J, Ciurea S, Armand P, Salit R, Bolaños-Meade J, Hamadani M Seftel MD, Neuberg D, Zhang M-J, Wang HL, Ballen KK, Bergeron J, Couban S, Freytes CO, Hamadani M, Kharfan-Dabaja MA, Lazarus HM, Nishihori T, Paulson K, Saber W, Sallan SE, Soiffer R, Tallman MS, Woolfrey AE, DeAngelo DJ, Weisdorf DJ Risk factors and impact of non-Aspergillus mold infections following allogeneic HCT: a CIBMTR infection and immune reconstitution analysis Significant improvements in the practice patterns of adult related donor care in US transplant centers Bone Marrow Transplantation. doi:10.1038/bmt. 2015.263. Epub 2015 Nov 2 [PMC Journal – In Process] Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.11.008. Epub 2015 Nov 18 [PMC Journal – In Process] Reduced-intensity transplantation for lymphomas using haploidentical related donors versus HLAmatched unrelated donors Blood. doi:10.1182/ blood-2015-09671834. Epub 2015 Dec 15 [PMC Journal – In Process] Pediatric-inspired therapy compared to allografting for Philadelphia chromosome negative adult ALL in first complete remission American Journal of [PMC Journal Hematology. – In Process] doi:10.1002/ajh.24285. Epub 2015 Dec 23 Page | 111 CIBMTR 2015 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS APPENDIX D2: BMT CTN PUBLICATIONS The following publications were generated by the BMT CTN, a component of the Clinical Trials Support Program, which conducts multi-institutional Phase II and III trials focused on HCT. The BMT CTN Data and Coordinating Center maintains continuity of operations and facilitates effective communications. The Data and Coordinating Center effort is a collaboration of the CIBMTR, NMDP/Be The Match, and the Emmes Corporation. For more information, see Section 2.3.1. BMT CTN PUBLICATIONS Authors Title (2014 publication not previously reported) Levine JE, Braun TM, Harris A prognostic score for AC, Holler E, Taylor A, Miller acute graft-versus-host H, Magenau J, Weisdorf DJ, disease based on Ho VT, Bolaños-Meade J, biomarkers: a multicentre Alousi AM, Ferrara JLM study MacMillan ML, Robin M, Harris AC, DeFor TE, Martin PJ, Alousi A, Ho VT, BolañosMeade J, Ferrara JLM, Jones R, Arora M, Blazar BR, Holtan SG, Jacobsohn D, Pasquini M, Socie G, Antin JH, Levine JE, Weisdorf DJ A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality Holtan SG, Verneris MR, Schultz KR, Newell LF, Meyers G, He F, DeFor TE, Vercellotti GM, Slungaard A, MacMillan ML, Cooley SA, Blazar BR, PanoskaltsisMortari A, Weisdorf DJ Circulating angiogenic factors associated with response and survival in patients with acute graftversus-host disease: results from Blood and Marrow Transplant Clinical Trials Network 0302 and 0802 Citation PMCID The Lancet Haematology. 2(1):e21-e29. doi:10.1016/S23523026(14)00035-0. Epub 2014 Dec 23 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):761-767. doi:10.1016/j.bbmt. 2015.01.001. Epub 2015 Jan 10 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jun 1; 21(6):10291036. doi:10.1016/ j.bbmt.2015.02.018. Epub 2015 Mar 7 PMC4340092 PMC4359643 PMC4426052 Page | 112 CIBMTR 2015 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS BMT CTN PUBLICATIONS Authors Title Citation PMCID Khera N, Majhail NS, Brazauskas R, Wang Z, He N, Aljurf MD, Akpek G, Atsuta Y, Beattie S, Bredeson CN, Burns LJ, Dalal JD, Freytes CO, Gupta V, Inamoto Y, Lazarus HM, LeMaistre CF, Steinberg A, Szwajcer D, Wingard JR, Wirk B, Wood WA, Joffe S, Hahn TE, Loberiza FR, Anasetti C, Horowitz MM, Lee SJ Anderlini P, Wu J, Gersten I, Ewell M, Tolar J, Antin JH, Adams R, Arai S, Eames G, Horwitz ME, McCarty J, Nakamura R, Pulsipher MA, Rowley S, Leifer E, Carter SL, DiFronzo NL, Horowitz MM, Confer D, Deeg HJ, Eapen M Young JAH, Logan BR, Wu J, Wingard JR, Weisdorf DJ, Mudrick C, Knust K, Horowitz MM, Confer DL, Dubberke ER, Pergam SA, Marty FM, Strasfeld LM, Brown JM, Langston AA, Schuster MG, Kaul DR, Martin SI, Anasetti C Comparison of characteristics and outcomes of trial participants and nonparticipants: example of Blood and Marrow Transplant Clinical Trials Network 0201 trial Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Oct 1; 21(10):18151822. doi:10.1016/ j.bbmt.2015.06.004. Epub 2015 Jun 11 PMC4568172 Cyclophosphamide conditioning in patients with severe aplastic anaemia given unrelated marrow transplantation: a phase 1-2 dose deescalation study Lancet Haematology. 2015 Sep 1; 2(9):e367375. doi:10.1016/ S2352-3026(15)001477. Epub 2015 Sep 2 [PMC Journal – In Process] Infections following transplantation of bone marrow or peripheralblood stem cells from unrelated donors [PMC Journal – In Process] Wood WA, Le-Rademacher J, Syrjala KL, Jim H, Jacobsen PB, Knight JM, Abidi MH, Wingard JR, Majhail NS, Geller NL, Rizzo JD, Fei M, Wu J, Horowitz MM, Lee SJ Patient-reported physical functioning predicts the success of hematopoietic cell transplantation (BMT CTN 0902) Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/ j.bbmt.2015.09.013. Epub 2015 Sep 23 Cancer. doi: 10.1002/ cncr.29717. Epub 2015 Oct 6 [PMC Journal – In Process] Page | 113 CIBMTR 2015 Annual Report APPENDIX D2: BMT CTN PUBLICATIONS BMT CTN PUBLICATIONS Authors Title Citation PMCID Giralt S, Garderet L, Durie B, Cook G, Gahrton G, Bruno B, Hari P, Lokhorst H, McCarthy P, Krishnan A, Sonneveld P, Goldschmidt H, Jagannath S, Barlogie B, Mateos M, Gimsing P, Sezer O, Mikhael J, Jin L, Dimopoulos M, Mazumder A, Palumbo A, Abonour R, Anderson K, Attal M, Blade J, Bird J, Cavo M, Comenzo R, de la Rubia J, Einsele H, Sanz RG, Hillengass J, Holstein S, Johnsen H, Joshua D, Koehne G, Kumar S, Kyle R, Leleu X, Lonial S, Ludwig H, Nahi H, Nooka A, Orlowski R, Rajkumar V, Reiman A, Richardson P, Rivas E, San Miguel J, Turreson I, Usmani S, Vesole D, Bensinger W, Qazilbash M, Efebera Y, Mohty M, Gasparreto C, Gajewski J, LeMaistre C, Bredeson C, Moreau P, Pasquini M, Kroeger N, Stadtmauer E Devine SM, Owzar K, Blum W, Mulkey F, Stone RM, Hsu JW, Champlin RE, Chen Y-B, Vij R, Slack J, Soiffer RJ, Larson RA, Shea TC, Hars V, Sibley AB, Geralt S, Carter S, Horowitz MM, Linker C, Alyea EP American Society of Blood and Marrow Transplant, European Society of Blood and Marrow Transplantation, Blood and Marrow Transplant Clinical Trials Network and International Myeloma Working Group Consensus Conference on Salvage Hematopoietic Cell Transplantation in Patients with Relapsed Multiple Myeloma Biology of Blood and Marrow Transplantation. 2015 Dec 1; 21(12):20392051. doi: 10.1016/ j.bbmt.2015.09.016. Epub 2015 Sep 30 [PMC Journal – In Process] Phase II study of Journal of Clinical allogeneic transplantation Oncology. Epub 2015 for older patients with Nov 2 acute myeloid leukemia in first complete remission using a reduced-intensity conditioning regimen: results from cancer and leukemia group B 100103 (alliance for clinical trials in oncology)/blood and marrow transplant clinical trial network 0502 [PMC Journal – In Process] Page | 114 CIBMTR 2015 Annual Report APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS APPENDIX D3: HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS The following publications were generated by the Health Services Research program, through which the CIBMTR conducts research in health disparities, health policy, and system capacity issues involving HCT. For more information, see Section 2.4. HEALTH SERVICES RESEARCH PROGRAM PUBLICATIONS Authors Title Besse KL, Preussler JM, Murphy EA, Denzen EM, Lill MC, Chell JW, Senneka MK, Majhail NS, Williams EP Estimating demand and unmet need for allogeneic hematopoietic cell transplantation in the United States using geographic information systems Majhail NS, Mau L-W, National survey of Chitphakdithai P, Payton T, hematopoietic cell Eckrich M, Joffe S, Lee SJ, transplantation center LeMaistre CF, LeRademacher personnel, infrastructure, J, Loberiza F, Logan B, Parsons and models of care SK, Repaczki-Jones R, Robinett delivery P, Rizzo JD, Murphy E, Denzen EM Clauser SB, Gayer C, Murphy E, Majhail NS, Baker KS Patient centeredness and engagement in quality-ofcare oncology research Preussler JM, Mau L-W, Majhail NS, Bevans M, Clancy E, Messner C, Parran L, Pederson KA, Ferguson SS, Walters W, Murphy EA, Denzen EM Patient housing barriers to hematopoietic cell transplantation: results from a mixed-methods study of transplant center social workers Citation PMCID Journal of Oncology Practice. 11(2):e120e130. doi:10.1200/ JOP.2014.000794. Epub 2015 Mar 1 PMC4371120 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):13081314. doi:10.1016/ j.bbmt.2015.03.020. Epub 2015 Mar 31 Journal of Oncology Practice. 2015 May 1; 11(3):176-179. doi:10.1200/JOP.2015 .003749. Epub 2015 Apr 7 Supportive Care in Cancer. doi:10.1007/ s00520-015-2872-9. Epub 2015 Aug 15 PMC4466059 [PMC Journal – In Process] [PMC Journal – In Process] Page | 115 CIBMTR 2015 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS The following publications were generated by the Bioinformatics Research Program, which provides expertise in, and conducts research on, translational and operational bioinformatics. For more information, see Section 2.5. BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Besse KL, Preussler JM, Murphy EA, Denzen EM, Lill MC, Chell JW, Senneka MK, Majhail NS, Williams EP Estimating demand and unmet need for allogeneic hematopoietic cell transplantation in the United States using geographic information systems Power laws for heavytailed distributions: modeling allele and haplotype diversity for the National Marrow Donor Program Progress toward curing HIV infection with hematopoietic cell transplantation Journal of Oncology Practice. 11(2):e120e130. doi:10.1200/ JOP.2014.000794. Epub 2015 Mar 1 PMC4371120 PLoS Computational Biology. 11(4):e1004204. doi:10.1371/journal.p cbi.1004204. Epub 2015 Apr 22 Stem Cells and Cloning: Advances and Applications. 2015(8):109-116. doi:10.2147/SCCAA.S 56050. Epub 2015 Jul 28 PLoS One. doi:10.1371/journal.p one.0135960. Epub 2015 Aug 19 PMC4406525 Slater N, Louzoun Y, Gragert L, Maiers M, Chatterjee A, Albrecht M Petz LD, Burnett JC, Li H, Li S, Tonai R, Bakalinskaya M, Shpall EJ, Armitage S, Kurtzberg J, Regan DM, Clark P, Querol S, Gutman JA, Spellman SR, Gragert L, Rossi JJ Hollenbach JA, Saperstein A, Albrecht M, Vierra-Green C, Parham P, Norman PJ, Maiers M Race, ethnicity and ancestry in unrelated transplant matching for the National Marrow Donor Program: a comparison of multiple forms of selfidentification with genetics PMC4524463 PMC4545604 Page | 116 CIBMTR 2015 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Besse K, Maiers M, Confer D, Albrecht M On modeling HLAidentical sibling match probability for alloHCT: estimating the need for an unrelated donor source N/A Single RM, Strayer N, Thomson G, Paunic V, Albrecht M, Maiers M Asymmetric linkage disequilibrium: tools for assessing multiallelic LD Mack SJ, Milius RP, Gifford BD, Sauter J, Hofmann J, Osoegawa K, Robinson J, Groeneweg M, Turenchalk GS, Adai A, Holcomb C, Rozemuller EH, Penning MT, Heuer ML, Wang C, Salit ML, Schmidt AH, Parham PR, Müller C, Hague T, Fischer G, Fernandez-Viña M, Hollenbach JA, Norman PJ, Maiers M Magalon J, Maiers M, Kurtzberg J, Navarrete C, Rubinstein P, Brown C, Schramm C, Larghero J, Katsahian S, Chabannon C, Picard C, Platz A, Schmidt A, Katz G Minimum information for reporting next generation sequence genotyping (MIRING): guidelines for reporting HLA and KIR genotyping via next generation sequencing Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2 015.09.012. Epub 2015 Sep 5 Human Immunology. doi:10.1016/j.humim m.2015.09.001. Epub 2015 Sep 7 Human Immunology. doi:10.1016/j.humim m.2015.09.011. Epub 2015 Sep 25 PLoS One. 10(12):e0143440. Epub 2015 Dec 1 PMC4666404 Banking or bankrupting: strategies for sustaining the economic future of public cord blood banks N/A [PMC Journal – In Process] Page | 117 CIBMTR 2015 Annual Report APPENDIX D4: BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS BIOINFORMATICS RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Buck K, Wadsworth K, Setterholm M, Maiers M, Confer D, Hartzman R, Schmidt A, Yang SY, Dehn J 7/8 and 9/10 or better high resolution match rate for the Be The Match® Unrelated Donor Registry Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2 015.12.012. Epub 2015 Dec 24 N/A Page | 118 CIBMTR 2015 Annual Report APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS APPENDIX D5: STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS The following publications were generated by the Statistical Methodology Research Program, which develops and evaluates the statistical models used in HCT. For more information, see Section 2.6. STATISTICAL METHODOLOGY RESEARCH PROGRAM PUBLICATIONS Authors Title Citation PMCID Keiding N, Andersen PK, Zhang MJ Editorial: To the memory of John P. Klein N/A Wang T, He P, Ahn KW, Wang X, Ghosh S, Laud P A re-formulation of generalized linear mixed models to fit family data in genetic association studies The proportional odds cumulative incidence model for competing risks A proportional hazards regression model for the subdistribution with covariates-adjusted censoring weight for competing risks data Observational studies: matching or regression? Lifetime Data Analysis. 2015 Apr 1; 21(2):157-159. doi:10.1007/ s10985-015-9320-5. Epub 2015 Feb 7 Frontiers in Genetics. 6(120):110. doi:10.3389/ fgene.2015.00120. Epub 2015 Mar 31 Biometrics. 2015 Sep 1; 71(3):687-695. doi:10.1111/biom.12330. Epub 2015 May 26 Scandinavian Journal of Statistics, Theory and Applications. doi:10.1111/sjos.12167. Epub 2015 Jun 5 PMC4608382 Eriksson F, Li J, Scheike T, Zhang M-J He P, Eriksson F, Scheike TH, Zhang M-J Brazauskas R, Logan BR PMC4379931 [PMC Journal – In Process] Biology of Blood and Marrow [PMC Journal Transplantation: Journal of the – In Process] American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt.2015.12.005. Epub 2015 Dec 19 Page | 119 CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS APPENDIX D6: COORDINATING CENTER PUBLICATIONS The following publications incorporated major contributions from the CIBMTR Coordinating Center and Scientific Directors. COORDINATING CENTER PUBLICATIONS Authors Title Citation PMCID Majhail NS, Giralt S, Bonagura A, Crawford S, Farnia S, Omel JL, Pasquini M, Saber W, LeMaistre CF Guidelines for defining and implementing standard episode of care for hematopoietic stem cell transplantation within the context of clinical trials Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Apr 1; 21(4):583-588. doi:10.1016/ j.bbmt.2014.12.030. Epub 2015 Jan 29 Current Hematologic Malignancy Reports. 2015 Mar 1; 10(1):4558. doi:10.1007/ s11899-014-0246-x. Epub 2015 Feb 21 The Lancet Haematology. 2(3):e91-e100. doi:10.1016/S23523026(15)00028-9. Epub 2015 Mar 1 N/A Sucheston-Campbell LE, Clay A, McCarthy PL, Zhu Q, Preus L, Pasquini M, Onel K, Hahn T Identification and utilization of donor and recipient genetic variants to predict survival after HCT: are we ready for primetime? Gratwohl A, Pasquini MC, One million haemopoietic Aljurf M, Atsuta Y, Baldomero stem-cell transplants: a H, Foeken L, Gratwohl M, retrospective Bouzas LF, Confer D, observational study Frauendorfer K, Gluckman E, Greinix H, Horowitz M, Iida M, Lipton J, Madrigal A, Mohty M, Noel L, Novitzky N, Nunez J, Oudshoorn M, Passweg J, van Rood J, Szer J, Blume K, Appelbaum F, Kodera Y, Niederwieser D PMC4352187 N/A Page | 120 CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS COORDINATING CENTER PUBLICATIONS Authors Title Citation PMCID Shah N, Callander N, Ganguly S, Gul Z, Hamadani M, Costa L, Sengsayadeth S, Abidi M, Hari P, Mohty M, Chen Y-B, Koreth J, Landau H, Lazarus H, Leather H, Majhail N, Nath R, Osman K, Perales M-A, Schriber J, Shaughnessy P, Vesole D, Vij R, Wingard J, Giralt S, Savani BN Hematopoietic stem cell transplantation for multiple myeloma: guidelines from the American Society for Blood and Marrow Transplantation [PMC Journal – In Process] Eapen M Hematopoietic cell transplantation for acute leukemia: selecting donors Khera N From evidence to clinical practice in blood and marrow transplantation Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Jul 1; 21(7):1155-1166. doi:10.1016/j.bbmt. 2015.03.002. Epub 2015 Mar 11 Haematologica. 2015 Apr 1; 100(4):414-415. doi:10.3324/haematol. 2015.124974. Epub 2015 Apr 1 Blood Reviews. doi:10.1016/j.blre. 2015.04.001. Epub 2015 Apr 9 Graff TM, Singavi AK, Schmidt W, Eastwood D, Drobyski WR, Horowitz M, Palmer J, Pasquini M, Rizzo DJ, Saber W, Hari P, Fenske TS Safety of outpatient autologous hematopoietic cell transplantation for multiple myeloma and lymphoma National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versusHost Disease: VI. The 2014 Clinical Trial Design Working Group Report Bone Marrow Transplantation. 2015 Jul 1; 50(7):947-953. doi:10.1038/ bmt.2015.46. Epub 2015 Apr 13 Biology of Blood and Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. 2015 Aug 1; 21(8):13431359. doi:10.1016/ j.bbmt.2015.05.004. Epub 2015 May 15 PMC4490016 Martin PJ, Lee SJ, Przepiorka D, Horowitz MM, Koreth J, Vogelsang GB, Walker I, Carpenter PA, Griffith LM, Akpek G, Mohty M, Wolff D, Pavletic SZ, Cutler CS PMC4380712 PMC4610823 PMC4506719 Page | 121 CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS COORDINATING CENTER PUBLICATIONS Authors Title Gale RP, Eapen M Who is the best alternative allotransplant donor? Renner R, Carlis J, Maiers M, Rizzo JD, O'Neill C, Horowitz M, Gee K, Confer D Booth GS, Gehrie EA, Jagasia MH, Shaw BE, Savani BN Majhail NS, Farnia SH, Carpenter PA, Champlin RE, Crawford S, Marks DI, Omel JL, Orchard PJ, Palmer J, Saber W, Savani BN, Veys PA, Bredeson CN, Giralt SA, LeMaistre CF Deeg HJ, Bredeson C, Farnia S, Ballen K, Gupta V, Mesa RA, Popat U, Hari P, Saber W, Seftel M, Tamari R, Petersdorf EW Citation Bone Marrow Transplantation. 2015 Jun 1; 50(S2):S40-S42. doi:10.1038/ bmt.2015.94. Epub 2015 Jun 4 Integration of Data Integration in the hematopoietic cell Life Sciences. 2015 Jul transplantation outcomes 8; 9162:139-146. data: data standards are doi:10.1007/978-3not enough 319-21843-4_11 When can you discard Biology of Blood and stem cells? Marrow Transplantation: Journal of the American Society for Blood and Marrow Transplantation. doi:10.1016/j.bbmt. 2015.07.024. Epub 2015 Aug 4 Indications for autologous Biology of Blood and and allogeneic Marrow hematopoietic cell Transplantation: transplantation: Journal of the guidelines from the American Society for American Society for Blood and Marrow Blood and Marrow Transplantation. Transplantation doi:10.1016/j.bbmt. 2015.07.032. Epub 2015 Aug 7 Hematopoietic cell Biology of Blood and transplantation as Marrow curative therapy for Transplantation: patients with Journal of the myelofibrosis: long-term American Society for success in all age groups Blood and Marrow Transplantation. 2015 Nov 1; 21(11):18831887. doi:10.1016/ j.bbmt.2015.09.005. Epub 2015 Sep 11 PMCID PMC4520408 N/A N/A N/A PMC4604067 Page | 122 CIBMTR 2015 Annual Report APPENDIX D6: COORDINATING CENTER PUBLICATIONS COORDINATING CENTER PUBLICATIONS Authors Title Citation PMCID Pagliuca S, Gérard L, Kulasekararaj A, Eapen M, Boutboul D, Martin H, Salvino MA, Knol-Bout C, Dufour C, Peffault de Latour R, Marsh J Characteristics and outcomes of aplastic anemia in HIV patients: a brief report from the severe aplastic anemia working party of the European Society of Blood and Bone Marrow Transplantation Bone Marrow Transplantation. doi:10.1038/ bmt.2015.252. Epub 2015 Oct 19 N/A Page | 123 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS APPENDIX E: PRESENTATIONS 2015 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI BMT CTN 0201 5 year results of BMT CTN 0201: unrelated donor bone marrow is associated with better psychological well-being and less burdensome chronic graft-versus-host disease symptoms than peripheral blood * Selected as a 2016 Highlight of ASH Oral SJ Lee BMT CTN 0601 A multicenter Phase II trial of unrelated donor reduced intensity bone marrow transplantation for children with severe sickle cell disease (SCURT): Results of the Blood and Marrow Transplant Clinical Trials Network (BMT CTN 0601) Study Oral S Shenoy BMT CTN 0805 Multicenter US intergroup study of intensive chemotherapy plus dasatinib followed by allogeneic stem cell transplant in patients with Philadelphia chromosome positive acute lymphoblastic leukemia younger than 60 * Late Breaking Abstract Oral F Ravandi BMT CTN 0901 Results of a Phase III randomized, multi-center study of allogeneic stem cell transplantation after high vs reduced intensity conditioning in patients with myelodysplastic syndrome or acute myeloid leukemia * Late Breaking Abstract Oral BL Scott CK11-02 A prognostic system predictive of outcomes in persons undergoing allogeneic hematopoietic cell transplantation for myelodysplastic syndrome Oral B Shaffer CK14-01 Outcomes after umbilical cord blood transplantation for myelodysplastic syndromes: a Center for International Blood and Marrow Transplant Registry study Poster A Gerds GV12-01 Outcomes of grades II-IV acute graft-versus-host disease post allogeneic hematopoietic stem cell transplantation: How much progress was achieved? Poster H Khoury GV12-02 Upper gastrointestinal acute graft-versus-host disease adds minimal prognostic value when present in isolation or in addition to grade I or other grade II-defining GvHD manifestations Oral S Nikiforow Page | 124 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS 2015 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study Title Type PI HS13-01 The impact of pre-transplant depression on outcomes of allogeneic and autologous hematopoietic stem cell transplantation Oral A El-Jawahri HS13-02 A study of predictors of clinical outcomes and healthcare utilization in children with sickle cell disease undergoing allogeneic hematopoietic cell transplantation Oral S Arnold IB11-01 Evaluation of the impact of non-inherited maternal antigens on the outcome of HLA mismatched unrelated donor hematopoietic stem cell transplantation for hematological malignancies on behalf of the ALWP of the EBMT and the CIBMTR Poster J Pingel IB12-03 Investigating effect of genetic admixture and donor / recipient genetic disparity on transplant outcomes Poster A Madbouly LK13-04 Comparison of post allogeneic hematopoietic cell transplantation outcomes after matched related donor versus matched unrelated donor HCT in adults with acute lymphoblastic leukemia Poster E Segal LK14-02 Outcomes of allogeneic transplantation in patients aged ≤ 60 years with acute myeloid leukemia in second complete remission: a CIBMTR cohort analysis Poster F Michelis LK14-03 Autologous transplant, and not ATO alone, remains the preferred therapy for relapsed APL: a report from the CIBMTR, EBMT, and two specialized centers Oral M Tallman LY06-03 Allogeneic stem cell transplantation for relapsed / refractory follicular lymphoma: a joint study between the European Society for Blood and Marrow Transplantation and the Center for International Blood and Marrow Transplant Research Oral A Sureda LY14-02 Reduced-intensity allogeneic hematopoietic cell transplantation provides durable progression-free survival in a subset of diffuse large B-cell lymphoma patients relapsing after autologous HCT Oral T Fenske Page | 125 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS 2015 AMERICAN SOCIETY OF HEMATOLOGY (ASH) ANNUAL MEETING Study LY15-01 Title Type PI Survival after T-cell replete haploidentical related donor transplant using post-transplant cyclophosphamide compared with matched unrelated donor transplant for lymphoid malignancies * Selected as a 2016 Highlight of ASH Oral M KharafanDabaja Poster M Sharma MM13-02 Autologous hematopoietic cell transplantation in patients with high risk multiple myeloma: post-transplant responses do not translate to longer survival MM15-01 Post transplant therapy is more important than induction regimen choice in autologous hematopoietic cell transplantation recipients for multiple myeloma Oral R Cornell RCI BMT 09PLEX A Phase II study evaluating the safety and efficacy of subcutaneous plerixafor for the mobilization and transplantation of HLA-matched sibling donor hematopoietic stem cells in recipients with hematologic malignancies Oral S Devine RT09-04 / IB09-06 Combined donor and recipient non-HLA genotypes show evidence of genome-wide association with transplant related mortality after HLA-matched unrelated donor blood and marrow transplantation (DISCOVeRY BMT Study) Oral L SuchestonCampbell RT09-04 / IB09-06 Genome wide association study of overall and progressionfree survival after HLA-matched unrelated donor blood and marrow transplantation (DISCOVeRY BMT Study) Oral T Hahn RT09-04c Evidence for heterogeneous genetic associations with acute lymphoblastic leukemia by cytogenetics and sex in high risk patients treated with matched unrelated donor allogeneic blood or marrow transplant Poster A Clay SC11-06 Outcome of patients 65 years and older with myelodysplastic syndrome receiving allogeneic hematopoietic stem cell transplantation compared to patients 55-64 years of age Oral E Atallah Page | 126 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS 2015 BMT TANDEM MEETINGS Type Principal Investigator BioMetadata / BRIDG integration process informatics Oral J Pollack BioStem cell transplant interoperability using BRIDG informatics Oral RP Milius Study Title BMT CTN More infections with transplantation of bone marrow versus peripheral-blood stem cells from unrelated donors Oral J Young BMT CTN 0302 / 0802 Prognostic impact of follistatin in acute graft-versus-host disease: results from BMT CTN 0302 and 0802 Oral S Holtan CK13-02 Allogeneic hematopoietic cell transplantation for adult chronic myelomonocytic leukemia Oral H Duong DS05-02b & RCI BMT 06-DON Health-related quality of life among older adult related hematopoietic stem cells donors (>60 yrs.) is equivalent to or better than that of younger adult related donors (18-60 yrs.) Oral G Switzer DS05-02c / RCI BMT 06-DON Acute toxicities of related adult donors compared to unrelated adult Oral M Pulsipher DS09-04 The effect of race, socioeconomic status, and collection center size on bone marrow and peripheral blood stem cell donor experiences at National Marrow Donor Program collection centers Oral M Pulsipher HS13-03 Clinical outcomes among unrelated donor transplant recipients for acute myelogenous leukemia as a function of socioeconomic status and related transcriptome differences Oral J Knight HSR 13-01 Administrative claims data for cost analyses in hematopoietic cell transplantation: the good, the bad and the ugly Poster J Preussler HSR 13-02 Patient and provider preferences for survivorship care plans for allogenic hematopoietic cell transplantation survivors: a qualitative study Poster E Murphy HSR 14-02 Hematopoietic cell transplantation multidisciplinary care teams: burnout, moral distress and career satisfaction Poster J Neumann Page | 127 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS 2015 BMT TANDEM MEETINGS Study Title Type Principal Investigator IB13-05 MHC class I chain-related gene A (MICA) donor-recipient mismatches and MICA-129 polymorphism in unrelated donor hematopoietic stem cell transplants for hematological malignancies: a CIBMTR study Poster M Askar IS13-01 Impact of race on graft-versus-host disease rates after HLAmatched sibling bone marrow or peripheral blood hematopoietic cell transplantation: Comparison of North American Caucasian versus Japanese populations Oral J Kanda LK13-03 Allogeneic transplant for acute biphenotypic leukemia: characteristics and outcome in the CIBMTR database Oral R Munker LY12-01 Positive pre-allogeneic hematopoietic cell transplantation PET scan in patients with non-Hodgkin lymphoma predicts higher risk of relapse but has no impact on survival Oral V Bachanova LY13-02 Risk factors predicting outcomes of autologous hematopoietic cell transplantation in children, adolescents and young adults with relapsed / refractory classical Hodgkin lymphoma: a CIBMTR analysis Oral P Satwani 2015 AMERICAN SOCIETY OF CLINICAL ONCOLOGY ANNUAL MEETING Title Type Principal Investigator LY13-03 Reduced intensity conditioning allograft as first transplant approach in relapsed / refractory grade III follicular lymphoma is associated with improved outcomes in longterm survivors Oral E Klyuchnikov BMT CTN 0704 Updated analysis of CALGB / ECOG / BMT CTN 100104: Lenalidomide vs. placebo maintenance therapy after single autologous stem cell transplant for multiple myeloma Poster SA Holstein Study Page | 128 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS 2015 EUROPEAN GROUP FOR BLOOD AND MARROW TRANSPLANTATION (EBMT) ANNUAL MEETING Type Principal Investigator BioReporting NGS-based HLA & KIR genotyping using MIRING informatics principles HML 1.0 Oral RP Milius DS05-02f / RCI BMT 06-DON Health-related quality of life among pediatric hematopoietic stem cell donors Oral G Switzer DS14-01 (US) Significant improvements in the practice patterns of related donor care in US transplant centres Poster C Anthias DS14-01 (EBMT) JACIE accreditation significantly improves compliance with international recommendations for related donor care in EBMT transplant centres Poster C Anthias IB12-02b Uni-directional and bi-directional non-permissive HLA-DPB1 T cell epitope group mismatches have similar risk associations in 10/10 matched unrelated donor HCT Oral K Fleischhauer IB12-04b Effect of HLA-C allele matching in the context of patients HLA-C encoded KIR ligand grouping (C1 or C2) on outcomes of unrelated hematopoietic stem cell transplantation Oral J Fischer IB12-06 KIR B genotype in HLA-matched unrelated donor protects from relapse and improves progression-free survival after allogeneic transplantation for relapsed / refractory nonHodgkin lymphoma (This presentation was withdrawn because the PI was unable to attend the meeting) Poster V Bachanova Study Title 2015 EUROPEAN HEMATOLOGY ASSOCIATION CONGRESS Title Type Principal Investigator LY13-03 Reduced intensity conditioning allo transplantation is associated with superior long-term disease control in relapsed / refractory grade I/II follicular lymphoma Oral E Klyuchnikov BMT CTN 0102 Heavy light chain ratio normalization allows identification of electrophoretic non-complete response patients with improved outcomes: a long term follow up update for BMT CTN 0102 correlative study Poster A D’Souza Study Page | 129 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS 2015 EUROPEAN IMMUNOGENETICS AND HISTOCOMPATIBILITY CONFERENCE Type Principal Investigator BioUsing SNPs to improve phasing of HLA haplotypes informatics Oral V Paunic BioImpudigree: an imputation-based automated pedigree tool informatics Oral J Freeman BioImproved HLA-based race / ethnic classification using donor informatics geography and census demographic data Poster L Gragert BioHLA diversity in the Ezer Mizion Registry informatics Poster M Halagan HLA class II six-locus DRB3/4/5~DRB1~DQA1~DQB1~DPA1~ BioDPB1 high resolution haplotype frequencies of the major US informatics populations Poster M Halagan BioHLA allele and haplotype frequencies for Christian and informatics Muslim Arab donors in Hadassah Registry Poster A Bishara Study Title 2015 INSTITUTE FOR HEALTHCARE ADVANCEMENT’S ANNUAL HEALTH LITERACY CONFERENCE Study BMT CTN 1205 Title Type Principal Investigator Easy-to-read informed consent forms for multi-center hematopoietic cell transplant clinical trials Poster H Moore 2015 INTERNATIONAL CONFERENCE ON LONG-TERM COMPLICATIONS OF TREATMENT OF CHILDREN AND ADOLESCENTS FOR CANCER Study LE13-04 Title Type Principal Investigator Survival of patients who develop solid tumors following hematopoietic stem cell transplantation Poster M Ehrhardt Page | 130 CIBMTR 2015 Annual Report APPENDIX E: PRESENTATIONS 2015 PEDIATRIC BLOOD AND MARROW TRANSPLANT CONSORTIUM ANNUAL MEETING Study LE13-04 Title Type Principal Investigator Survival of patients who develop solid tumors following hematopoietic stem cell transplantation Oral M Ehrhardt 2015 WORLD CONGRESS OF PSYCHO-ONCOLOGY (hosted by the International Psycho-Oncology Society and American Psychosocial Oncology Society) Title Type Principal Investigator BMT CTN 0902 Cancer and treatment distress measurement over time in a multicenter cohort of hematopoietic cell transplantation recipients Oral K Syrjala BMT CTN 0902 Pre-transplant health-related quality of life factors as predictors of outcomes following hematopoietic cell transplantation Oral J Knight Study 2015 WORLD MARROW DONOR ASSOCIATION MEETING Type Principal Investigator BioKey driver analysis of HLA diversity: analytically focused informatics recruitment strategies for improving registry quality Poster A Sivasankaran BioEnhancing HML for electronic reporting of NGS-based HLA informatics and KIR genotyping results Poster J Kathryn Study Title Page | 131 CIBMTR 2015 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS This study development and management process pertains to studies for which the CIBMTR provides data and statistical support. Data sets are also made available to investigators who have their own statistical resources. Final analyses and manuscripts resulting from these analyses are reviewed and approved by the CIBMTR prior to journal submission. STUDY DEVELOPMENT AND MANAGEMENT PROCESS Planned Protocol pending. Proposals remain in this preliminary stage until the PI creates a draft protocol. Draft protocol received. When a PI submits a draft protocol, Coordinating Center staff review it. Protocol development. During the development process, the Working Committee biostatisticians, Scientific Director, and Chairs refine the submission into a comprehensive study protocol. They add a table with a preliminary description of the proposed study population and present the draft protocol for discussion at a weekly Coordinating Center statistical meeting. When a protocol is approved, Coordinating Center personnel invite Working Committee members to participate in a Writing Committee. In Progress Sample typing. If applicable, the PIs perform laboratory tests (e.g., genotyping) on samples from the CIBMTR Research Repository. The testing data will be used in the analysis to determine any correlation with clinical outcome. Supplemental forms / data collection. Most studies use routinely-collected data. If necessary, Coordinating Center staff, in collaboration with the PI and relevant Working Committee Chairs, develop a supplemental form, which is approved prior to soliciting centers for additional data. Use of supplemental data (e.g., data not collected on standard CIBMTR data collection forms) is discouraged unless it will result in a particularly meaningful publication and/or external funding can support the extra burden placed on transplant centers and supplement forms reimbursement costs. Page | 132 CIBMTR 2015 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS STUDY DEVELOPMENT AND MANAGEMENT PROCESS Data file preparation. The objective of data file preparation is to create a file of eligible subjects who are consecutively treated at participating centers with adequate follow-up, with minimal missing data fields, and in large enough numbers to give the analysis sufficient statistical power to meet the stated study objectives. This process involves a series of steps by the MS-level statistician, sometimes working with the Clinical Research Coordinator, to ensure data quality: In Progress (continued) • Verifying selection criteria • Including and excluding patients so that the investigators can determine whether the final study population is representative of the target population • Assessing follow-up • Determining the extent and nature of missing values and their potential effect on the study • Resolving and reconciling data discrepancies / outliers by examining data collection forms and communicating with centers and the PI Analysis in progress. Analysis proceeds in several phases. The first generally includes a detailed description of the patient population and univariate and multivariate analyses of study endpoints. Coordinating Center personnel present these data for discussion at a weekly Coordinating Center statistical meeting and then distribute them to Writing Committee members for suggestions and comments. The PI works with Coordinating Center staff in an iterative process to review comments from the Writing Committee. The process repeats until final analysis, which serves as the basis for the manuscript. Ongoing. A study in ongoing status is long-term and often involves multiple grants and/or renewals outside of the CIBMTR in order to reach its objectives. The study has its own Statistical Director for analysis, but it requires data from the CIBMTR, usually each year. Preliminary Results Manuscript preparation. The PI is primarily responsible for manuscript preparation and is expected to prepare a draft manuscript within 30 days of receiving analysis results. The Working Committee leadership review and revise the document, ensuring that the description and interpretation of the statistical analyses are accurate and contribute to the fundamental message of the manuscript. The Coordinating Center then distributes the approved first draft to the Writing Committee and solicits feedback. The PI incorporates comments from the Writing Committee and creates a revised draft, which is reviewed in an iterative process by the Writing Committee until reaching a reasonable consensus on a final manuscript. Page | 133 CIBMTR 2015 Annual Report APPENDIX F: STUDY DEVELOPMENT AND MANAGEMENT PROCESS STUDY DEVELOPMENT AND MANAGEMENT PROCESS Preliminary Results (continued) Submitted. The Coordinating Center staff is responsible for submitting the manuscript and corresponding with the chosen journal. The Working Committee Scientific Director often serves as corresponding author, and the study statistician forwards all editor and reviewer comments to the PI and Statistical Director. The PI is expected to prepare a response, working with Coordinating Center staff who provide additional analyses of data, as needed. Coordinating Center personnel communicate with the journal, including re-submissions, in most cases. In press. A publication is in press when it has been approved but does not yet have a citation. Completed Published. A manuscript is considered published when a citation is available, including a PMCID number, if applicable. For a list of 2015 publications, see Appendix D. Page | 134 CIBMTR 2015 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS APPENDIX G: CLINICAL TRIALS Through the Clinical Trials Support Program, the Coordinating Center supports clinical trial planning and interpretation; data collection, including long-term follow-up data; and real-time accrual assessment. See Section 2.3 for more information. APPENDIX G1: BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT The BMT CTN (Section 2.3.1) is the US national trials group charged with developing and conducting multicenter Phase II and III clinical trials focused on HCT. The CIBMTR is the lead institution for the BMT CTN Data and Coordinating Center, which it runs in collaboration with NMDP/Be The Match and the Emmes Corporation. A status of BMT CTN trials open for enrollment is included in this appendix and is available on the BMT CTN website. For additional information on completed Network trials, see the annual progress report on the BMT CTN website. BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Number BMT CTN 1101 BMT CTN 1102 BMT CTN 1202 BMT CTN 1203 Title Status to Date Phase III study comparing HLA• Opened to accrual Jun 2012 haploidentical related donor bone marrow versus double umbilical cord • 219 of 410 patients enrolled blood with reduced-intensity conditioning • Anticipated accrual completion in for patients with hematologic 2017 malignancies A multi-center biologic assignment trial comparing reduced intensity allogeneic • Opened to accrual Dec 2013 hematopoietic cell transplant to • 147 of 338 patients enrolled hypomethylating therapy or best • Anticipated accrual completion in supportive care in patients aged 50-75 early 2018 with intermediate-2 and high risk myelodysplastic syndrome • Opened to accrual Jun 2013 • 1,708 of 1,500 patients enrolled; Prospective multi-center cohort for the accrual target increased to evaluation of biomarkers predicting risk enhance pediatric and minority of complications and mortality following patient accrual allogeneic HCT • Anticipated accrual completion in Jun 2016 • Opened to accrual Sep 2014 A multi-center Phase II trial randomizing novel approaches for graft-versus-host • 209 of 270 patients enrolled disease prevention compared to • Anticipated accrual completion in contemporary controls April 2016 Page | 135 CIBMTR 2015 Annual Report APPENDIX G1: BMT CTN CLINICAL TRIALS BMT CTN CLINICAL TRIALS OPEN FOR ENROLLMENT Protocol Number BMT CTN 1205 BMT CTN 1301 BMT CTN 1302 BMT CTN 1304 (DFCI 10-106) BMT CTN 1505 (UT RECRUIT) Title Status to Date • Opened to accrual Nov 2013 • 138 of 160 patients enrolled, Easy-to-read informed consent (ETRIC) for although accrual target may be hematopoietic cell transplantation clinical increased trials • Anticipated accrual completion in 2016 • Opened to accrual Aug 2015 Phase III trial of calcineurin inhibitor-free • 9 of 345 patients enrolled interventions for prevention of graft• Anticipated accrual completion in versus host-disease early 2019 Phase II, double-blind Placebo controlled • Opened to accrual Aug 2015 trial of maintenance ixazomib after • 6 of 138 patients enrolled allogeneic HCT for high risk multiple • Anticipated accrual completion in myeloma June 2018 A randomized Phase III study comparing • Opened to accrual by BMT CTN conventional dose treatment using a Nov 2013 combination of lenalidomide, bortezomib, and dexamethasone (RVD) to high-dose • 514 of 660 patients enrolled treatment with peripheral stem cell • Anticipated accrual completion in transplant in the initial management of 2016 myeloma in patients up to 65 years of age • Opened to accrual by BMT CTN May 2015 • Patient enrollment: N/A, BMT CTN Randomized minority patient recruitment centers randomized to intervention intervention trial and control arms; center staff are research subjects • Anticipated accrual completion in May 2017 Page | 136 CIBMTR 2015 Annual Report APPENDIX G2: RCI BMT CLINICAL TRIALS APPENDIX G2: RCI BMT CLINICAL TRIALS The RCI BMT (Section 2.3.2) provides researchers in the field of HCT with infrastructure and expertise in HCT clinical trial conduct and analysis. The program’s goal is to help investigators generate data allowing novel and innovative ideas to move into the larger Phase II or Phase III setting into such groups as the BMT CTN or the national cancer cooperative groups. A status of its projects is included in this appendix. RCI BMT CLINICAL TRIALS Protocol # Astellas 12-MOXE Title Unrelated donor sample collection for donors whose recipient is enrolled in a randomized, double-blind, placebo-controlled, Phase III trial to evaluate the protective efficacy and safety of a therapeutic vaccine, ASP0113, in cytomegalovirus-seropositive recipients undergoing allogeneic hematopoietic cell transplant Study of the anti-CD22 recombinant immunotoxin moxetumomab pasudotox (CAT8015, HA 22) in children with B-lineage acute lymphoblastic leukemia and minimal residual disease prior to allogeneic hematopoietic cell transplantation 09-SQOL Pilot study to assess the feasibility of collecting quality of life data in collaboration with the Stem Cell Therapeutic Outcomes Database 11-TREO Multi-center study evaluating treosulfan, fludarabine, and low-dose TBI in children with AML / MDS undergoing allogeneic HCT COG-KIR A multi-center study examining donor NK-cell receptors and patient outcomes Status To Date • Donor sample collection only • Opened to accrual Apr 2014 • Two donors enrolled • RCI BMT closed study Dec 2014 • Opened to accrual May 2015 • One patient enrolled • Study closed Sep 2015 due to pharmaceutical company decision • Closed to accrual Sep 2013 • 301 adults and 89 pediatric recipients enrolled • Final subject follow-up expected by end of 2014 • Data analysis in process • Closed to accrual April 2014, about one year earlier than expected • 40 of 40 targeted enrollment • Follow-up continues • Abstract submitted to 2016 EBMT Annual Meeting • COG closed accrual May 2014 • URD sample management • 609 of 1,200 samples collected • 145 out of 400 targeted enrollment Page | 137 CIBMTR 2015 Annual Report APPENDIX G2: RCI BMT CLINICAL TRIALS RCI BMT CLINICAL TRIALS Protocol # DS05-02, 06-DON Title RDSafe: A multi-institutional study of hematopoietic stem cell donor safety and quality of life PBMTC ONC A multi-center study to determine the role of 1001 / 09- minimal residual disease testing before and MRD after HCT for pediatric acute myeloid leukemia 09-PLEX rHuG-CSF PBSC 10-CBA 10 CMSMDS-1 A phase II study evaluating the safety and efficacy of intravenous plerixafor for the mobilization and transplantation of HLAmatched sibling donor hematopoietic stem cells in recipients with hematological malignancies Long-term follow-up study evaluating hematologic and non-hematologic cancers, thrombotic events, and autoimmune disorders in unrelated donors undergoing bone marrow harvest versus peripheral blood stem cell mobilization with recombinant human granulocyte colony-stimulating factor Filgrastim-mobilized peripheral blood stem cells for allogeneic transplantation with unrelated donors A multi-center access and distribution protocol for unlicensed cryopreserved cord blood units for transplantation in pediatric and adult patients with hematologic malignancies and other indications Assessment of allogeneic HCT in Medicare beneficiaries with MDS and related disorders Status To Date • Closed to accrual July 2014 • 1,812 donors enrolled • Follow up assessments completed July 2015 • Abstract presented at 2015 BMT Tandem Meetings • Data review and analysis as well as manuscript planning in progress • Closed to accrual Oct 2014 • 150 total enrollment • Follow-up continues • Closed to accrual Dec 2014 • Follow-up completed Feb 2016 • Oral abstract presented at 2015 ASH Annual Meeting • Data review and analysis as well as manuscript planning in process • Closed to accrual Oct 2015 • 21,569 total enrollment • Follow-up continues through 2020 • Opened to accrual Apr 1996 • Just under 25,000 URDs enrolled • Will close to accrual upon FDA license; another protocol will open for all unlicensed product • Opened to accrual Oct 2010 • 2,757 enrolled • Open indefinitely to allow distribution and access to unlicensed cord units • Opened to accrual Dec 2010 • 1,294 patients enrolled Page | 138 CIBMTR 2015 Annual Report APPENDIX G2: RCI BMT CLINICAL TRIALS RCI BMT CLINICAL TRIALS Protocol # KIR-DS Statin BMT CTN 1102-QOL Title A multi-center study looking at the selection of a favorable KIR donor Impact of donor statin use on graft-versus-host disease after unrelated donor HCT; URD data collection A study comparing reduced intensity allogeneic hematopoietic cell transplant to hypomethylating therapy or best supportive care in patients aged 50-75 with intermediate2 and high risk myelodysplastic syndrome Status To Date • • • • Opened to accrual Jun 2011 URD sample management 1,942 samples facilitated Amendment in process to alter donor sample logistics, which will eliminated CIBMTR staff involvement • Opened to accrual Oct 2011 • 5,569 of 7,000 targeted donors enrolled • Survey Research Group performing QOL assessments • 210 total assessments completed 13-TLEC Prospective non-therapeutic study, assessing the long-term toxicity of HCT for childhood leukemia • Opened to accrual Mar 2015 • 53 of 340 patients enrolled 13-SCP A randomized study to evaluate the impact of survivorship care planning on cancer survivors self-management and adherence to care recommendations and utilization of follow-up care • In collaboration with Health Services Research Program • Opened to accrual Apr 2015 • 181 of 495 patients enrolled BMT CTN 1102Ancillary CEA study A cost effectiveness ancillary study to the parent study 1102 above HPD High priority donor project supporting Be The Match Operations 15-MMUD Study of transplantation of HLA-mismatched unrelated donor bone marrow for patients with hematologic malignancies • Collaborating with Fred Hutchinson Cancer Research center to perform Cost Effectiveness Analysis (CEA) study • Survey Research Group to perform CEA survey collection • First subject contacted in Oct 2015 • First team meeting held in June 2015 • First subjects contacts by the Survey Research Group in Sep 2015 • Protocol team established Oct 2015 • Protocol development in process Page | 139 CIBMTR 2015 Annual Report APPENDIX H: FORMS SUBMISSION PROCESS APPENDIX H: FORMS SUBMISSION PROCESS • • • • • • Center submits CRID Assignment Form (Form 2804), and CRID is generated Indication for CRID Assignment Form (Form 2814) is added to Forms Due list Center completes Indication Form and reports indication as HCT Pre-TED (Form 2400) is added to Forms Due list Center completes and submits Pre-TED Pre-TED data are processed through the selection algorithm resulting in CRF or TED track o If autologous recipient declines consent for research, stop here. Otherwise, follow the appropriate track below CRF Track Forms 2004, 2005, and 2006 are added, depending on 1 donor type and if the donor has been used for a prior transplant.* 2 Baseline form 2000, disease specific inserts, and Followup Forms are added to Forms Due list. 3 Center completes Baseline form after infusion. TED Track Forms 2004, 2005, and 2006 are added, depending on donor type, consent for sample repository, and if the donor has been used for a prior transplant.* Post-TED Follow-up Form 2450 is added to Forms Due list. Center completes designated Post-TED Forms at appropriate time points. Center completes designated CRF Follow-up Forms at appropriate time points. Is recipient alive? If yes, go to Step 5. If no, report the death on the follow-up form, and go to Reporting Recipient Death. Is recipient alive? If yes, go to Step 6. If no, report the 5 death on the follow-up form, and go to Reporting Recipient Death. Did recipient have a subsequent transplant? If yes, go to Step 6. If no, continue reporting at next time point (Step 3). Did recipient have subsequent transplant? If yes, go to 6 Step 7. If no, continue reporting at next time point (Step 4). Subsequent transplant is reported on the next available follow-up form. 4 When the form reporting the subsequent transplant When the form reporting the subsequent transplant is in is in complete status, future forms for the prior 7 complete status, future forms for the prior transplant will transplant will be automatically deleted from be automatically deleted from FormsNet. FormsNet. Center completes and submits Pre-TED (Form 2400) for 8 subsequent transplant. Go to Step 2 for subsequent transplant. Center completes and submits Pre-TED (Form 2400) for subsequent transplant. Go to Step 2 for subsequent transplant. Reporting Recipient Death Death Form 2900 is completed to report the recipient’s death.** The recipient’s death is reported on the Post TED. A 2900 Death Form should not be completed for patients on the TED track. * For more details regarding when Forms 2004, 2005, and 2006 are required, see “How Forms Come Due (2004, 2005, and 2006)”. ** Complete Death Form 2900 even if autopsy is pending. Another death form will be requested to confirm cause of death if autopsy was pending. Page | 140 CIBMTR 2015 Annual Report APPENDIX I: WEBSITES APPENDIX I: WEBSITES Throughout this report, electronic links to webpages and documents are provided. If you are unable to access items using the links provided, enter the underlined words into a general search engine or the search engine at the top of the CIBMTR website (cibmtr.org). URLs for the websites mentioned in this report are provided here. Name Be The Match Be The Match Clinical BMT CTN CIBMTR CIBMTR Collaborate CIBMTR Portal HRSA Blood Cell Transplant URL bethematch.org bethematchclinical.org bmtctn.net cibmtr.org collaborate.cibmtr.org portal.cibmtr.org bloodcell.transplant.hrsa.gov Page | 141 CIBMTR 2015 Annual Report APPENDIX J: GLOSSARY APPENDIX J: GLOSSARY Abbreviation/ Meaning Acronym AGNIS alloHCT AML ASBMT ASH autoHCT BMT BMT CTN BRIDG CALGB CED CIBMTR CME CMS COG CPI CRF CRID DBtC DFCI DISCO DRI EBMT eDBtC FACT FDA HCT HIPAA HIV HLA HRSA IND IRB IT KIR MCW MDS MED-A, MED-B N/A NCI A Growable Network Information System allogeneic hematopoietic cell transplantation acute myeloid (myelogenous) leukemia American Society of Blood and Marrow Transplantation American Society of Hematology autologous hematopoietic cell transplantation bone marrow transplant Blood and Marrow Transplant Clinical Trials Network Biomedical Research Integrated Domain Group Cancer and Leukemia Group B (member Alliance for Clinical Trials in Oncology) Coverage with Evidence Determination Center for International Blood and Marrow Transplant Research continuing medical education Centers for Medicare and Medicaid Services Children’s Oncology Group Continuous Process Improvement Comprehensive Report Form CIBMTR Recipient Identification Number Data Back to Centers application Dana Farber Cancer Institute Data and Information for Statistical Center Operations Disease Risk Index European Group for Blood and Marrow Transplantation enhanced Data Back to Centers application Foundation for the Accreditation of Cellular Therapy Food and Drug Administration hematopoietic cell transplantation Health Insurance Portability and Accountability Act human immunodeficiency virus human leukocyte antigen Health Resources and Services Administration Investigational new drug Institutional Review Board Information Technology killer-cell immunoglobulin-like receptors Medical College of Wisconsin myelodysplastic syndrome Minimum Essential Data forms not applicable National Cancer Institute Page | 142 CIBMTR 2015 Annual Report APPENDIX J: GLOSSARY Abbreviation/ Meaning Acronym NK NHLBI NIAID NIH NMDP PBMTC PBSC PI PMCID QOL RCI BMT RDSafe RITN SCTOD TBD TED URD US vs natural killer (cell) National Heart, Lung, and Blood Institute National Institute of Allergy and Infectious Disease National Institutes of Health National Marrow Donor Program Pediatric Blood and Marrow Transplant Consortium peripheral blood stem cell principal investigator PubMed Central unique identifier quality of life Resource for Clinical Investigations in Blood and Marrow Transplant Related Donor Safety Study Radiation Injury Treatment Network Stem Cell Therapeutic Outcomes Database to be determined Transplant Essential Data unrelated donor United States versus Page | 143 The CIBMTR is supported by Public Health Service Grant/Cooperative Agreement 5U24CA076518 from the National Cancer Institute (NCI), the National Heart, Lung and Blood Institute (NHLBI) and the National Institute of Allergy and Infectious Diseases (NIAID). CIBMTR® (Center for International Blood and Marrow Transplant Research®) is a research collaboration between the National Marrow Donor Program® (NMDP)/Be The Match® and Medical College of Wisconsin Milwaukee Campus Medical College of Wisconsin 9200 W. Wisconsin Ave., Suite C5500 Milwaukee, WI 53226 USA (414) 805-0700 cibmtr.org © 2016 The Medical College of Wisconsin, Inc. and the National Marrow Donor Program Minneapolis Campus National Marrow Donor Program/ Be The Match 500 N 5th Street Minneapolis, MN 55401 USA (612) 627-5800 cibmtr.org