UK Stem Cell Strategic Forum Recommendations

UK Stem Cell Strategic Forum
C Craddock, University of Birmingham
Our Aims and Objectives
The UK Stem Cell Strategic Forum was established at
the request of the Minister of State for Public Health in
February 2010.
Led by NHS Blood and Transplant.
To deliver a comprehensive strategy to meet the needs
of UK patients for unrelated haemopoietic stem cells.
Working with stakeholders, service providers, experts to:
 Understand the healthcare and economic
challenges of alternative donor transplantation
 Find new ways of working together to save and
improve patients’ lives.
An important opportunity to improve patient outcomes
Allogeneic stem cell transplantation remains the only curative therapy
for children and adults with high risk haematological malignancies
Initially confined to patients with matched sibling donors but excellent
outcomes now achievable using unrelated and cord blood donors
Major barriers limit the extension of a curative option to all:
Absence of a suitable donor-particularly affects patients from BME
Delays in identifying a suitable donor
Death from transplant toxicity or disease relapse
Lack of a coordinated UK strategy to deliver alternative donor stem cells and
high quality transplant care
Historic opportunity to increase number of lives saved by Tx and establish
the UK as a leader in stem cell provision and transplant
Stakeholder Engagement
Review structured to be as inclusive as possible – hence Working
Groups supporting the Forum.
Other ‘Reference’ Groups established as required to further draw
on available expertise and opinion.
Key stakeholder engagement via workshop and/or briefings:
Orin Lewis - The African Caribbean Leukaemia Trust (Chair)
The Cord Blood Charity
The Jeanette Crizzle Trust
The Adrian Sudbury Campaign
Desi Donors
Private cord blood banks
The All-Party Parliamentary Group on Umbilical Cord Blood
and Adult Stem Cells
Requirements and Benefits Group
Professor David Marks (chair) – consultant transplant physician
Dr Mary Horowitz (Head CIBMTR, USA)
Prof Eliane Gluckman (Hospital St Louis, Paris)
Dr Fred Appelabuam (Fred Hutchinson Cancer Centre, Seattle
Dr Paul Veys - consultant transplant physician
Dr Sergio Querol - consultant transplant physician
Dr Derwood Pamphilon – Medical Director BBMR and NHS-CBB
Stephen Dobra - DH Principal O.R. Analyst
Dr. Rachael Hough - Chair BSBMT CB Working Group
Dr Paresh Vyas – Research and Development
Kate Bingham - Schroder Venture Capital
Service Delivery Group
Dr Andrew Hadley (chair) – Review co-ordinator
Dr Carlheinz Müller – Director, German Bone Marrow Registry
Henny Braund – CEO Anthony Nolan Trust
Lynda Hamlyn – CEO NHSBT
Colin Pavelin – Department of Health
Dr Derwood Pamphilon – UK Blood Services
Dr Kieran Morris – NI Blood Transfusion Service
Dr Geoff Poole – Welsh Blood Service
Keith Thompson – Scottish National Blood Transfusion Service
Commissioning Group
Prof Antonio Pagliuca (chair) – President BSBMT
Dr Jackie Cornish – Consultant Transplant Physician
Mike Millen – Specialist Commissioner
Cathy Edwards – Specialist Commissioner
Jessica Whitton, Commissioning Manager, South West
specialised Commissioning Group
Key Recommendations
Unanimous agreement that stem cell transplantation from
either unrelated donors or umbilical cord blood are highly
effective treatment strategies for children and adults with high
risk leukaemia and other haematological disorders
There is a clear and important need to significantly decrease
the time it takes to identify suitably matched unrelated donors.
This can be achieved through better tissue typing of volunteer
donors and exploitation of information technology platforms
which assist in the prediction of patient-donor compatibility
Improve the Provision of Unrelated Adult Donor Stem
To increase the number of UK patients finding an acceptable
match within a clinically appropriate time period, we
recommend that:
 In collaboration with third sector organisations, there should
be greater engagement with Black and minority ethnic
donors to increase their representation on donor registries
and cord blood banks.
 Selected donors should be retrospectively and
prospectively HLA typed to high resolution to obviate the
need for this test as part of the donor selection process
 The UK should create or purchase predictive search
technologies (such as those used by German and
US registries) to increase the chance that selected
donors are a match for the patient
 A ‘graft identification advisory service’ should
be established to ensure optimal donor selection for
each patient.
 Registries should increase contact with donors,
updating information on their contact details, health
status and willingness to donate.
These measures are intended to create a ‘fit panel’ of
volunteer donors. Collectively, they would:
 Increase the proportion of selected adult volunteers able
to donate from 60% to 90%
 Reduce the number of donor searches by around 30%
 Reduce the number of extended HLA typings performed
at donor selection by 75%
 Reduce search to transplant times by four to six weeks.
Key Recommendations
Given the unique ethnic diversity of the UK investment should
be made to establish a bank of 50,000 cord blood units
accessible to all NHS patients
Importation of cord blood units from international registries
cannot substitute for the development of a UK cord blood
Stem cell dose is a critical determinant of outcome after cord
blood transplantation and target doses should be kept under
continual review
Stem Cell Supply Recommendations
Improve the Provision of Cord Blood Stem Cells
Based on an analysis of transplantation trends, and of patient and donor
Demographics, we recommend that:
 The UK should increase its inventory of cord blood to 50,000 units over
eight years. This time period represents a cost-effective approach to
achieving the required inventory.
 The inventory should contain 30% to 50% of donations from Black and ethnic
minority women.
 Newly banked units should have a high total of nucleated count threshold
(over 9x108 TNC from ethnic minority donors, over 12x108 TNC from Caucasian
Provision of Cord Blood Stem Cells
 Satisfy 85% of unmet Caucasian need (around 150 patients each year) and
50% of unmet black and minority ethnic need (around 50 patients each
 Improve search to transplant times (around 100 patients each year who fail
to receive an adult stem cell transplant due to delays in provision would be
able to be transplanted).
 Improve donor/recipient matches (around 70 patients each year
transplanted with a 9/10 matched adult donor could be transplanted faster
or more effectively).
 Reduce the total cost of cord blood units to the NHS to £16K- investment
cost neutral within 5 years, saves money to the NHS beyond that.
Key Recommendations
Unrelated donor transplants are complex treatments with a
substantial treatment-related mortality. Regional Centres of
Excellence for unrelated donor transplantation, specifically
cord blood transplantation, should be established in order to
improve patient outcomes. Any savings from this rationalisation
should be considered for re-investment in the stem cell transplant
programme of the NHS
A system should be put into place for all units performing unrelated
donor transplants to report immediate and long term patient outcomes
to Commissioners through a national transplant outcome registry.
Drive Quality and Efficiency
Unrelated donor transplantation should reflect clinical best practice.
Acknowledging differences in policy across the UK, we recommend that:
 Educational tools and platforms should be developed to
improve understanding among commissioning bodies. With the support
of physicians, commissioners should align their strategies with the latest
clinical guidance and patient outcome data.
 Commissioning bodies should operate within a standardised
funding framework, using a baseline figure adjusted to reflect market
forces factors. This should detail the necessary costs for each stage of
the patient’s treatment to provide clarity and certainty to all
service providers.
Allograft activity by UK transplant centre, 2009
Cord transplant activity by centre, 2009
Resources and expertise for cord blood transplantation
should be concentrated into designated Regional Centres
of Excellence, promoting high quality care and the best use
of resources.
• Regional Centres of Excellence should serve a minimum
population of 4-5 million.
 Local networks should be linked into designated centres
and make appropriate referrals when necessary.
Drive Quality and Efficiency
 Standardised data collection and outcome
monitoring should be integrated into every stage of the
patient pathway so that reliable outcome data can be
used to benchmark individual performance and promote
best practice.
 Funding streams should be identified to support
the collection and analysis of outcome data
from Regional Centres of Excellence.
Key Recommendations
Given the significant mortality of unrelated donor
transplantation it is highly desirable that transplants are
performed according to disease specific registration studies
and where possible appropriately badged clinical trials
Commissioning authorities should encourage the development
of appropriate studies for patients undergoing unrelated donor
Key Recommendations
Substantial Government funding over the last two decades has
resulted in the development of a world-class science base in
stem cell biology in the U.K. A clinical trials network of centres
performing unrelated donor stem cell transplants should be
established. This will create one of the world’s largest stem cell
transplant trials networks
Collaborative links should be established between donor
registries, cord blood banks and the biopharmaceutical
industries. This initiative, coupled with the establishment of a
clinical trials network has the capacity to attract inward
investment in the U.K which will create jobs in the knowledge
rich biotechnology sector
The LLR Trials Acceleration
Statistical Design,
Regulatory Approvals, Site
Initiation, Trial
Programming, Monitoring,
Analysis, publication
Central Coordination
Drive Quality and Efficiency
 Designated transplant centres should work together to
support an alternative donor clinical trials network
 The commissioning process should encourage the
development of registration studies and early and late phase
clinical trials in alternative donor transplantation
 Funding streams should be identified to develop this
important translational initiative
Progress to date
All recommendations of Report accepted by Dept of Health
Identified by Ministers as a model of collaborative working between
clinicians, commissioners and providers
£4 million grant provided to NHSBT and AN for expansion of
unrelated donor registry and cord blood bank
Effective collaboration between NHSBT and AN has delivered a
single registry, grown a fit panel and enabled expansion of unrelated
registry and cord blood bank
Progress to date
Jan 2012: Rebid to fund further expansion of registry and bank and
secure funding of outcome registry submitted to DoH
Joint NHSBT/AN graft identification services workshops established
in order to share best practice
Active discussions with Commissioning authorities to deliver
Regional Centres of Excellence
Request for support for transplant trials network, in the context of
2011 Govt Life Sciences Review, submitted to NIHR
Strategic Oversight Committee tasked with long term review and
development of UK policy for alternative donor stem cell
Back Up
Unrelated donor HSCT trends worldwide
The increasing use of unrelated donor HSCT in
older patients
The increased use of cord blood in the UK for unrelated
donor HSCT
Patient matching rates by ethnicity (2004/5)
Search times for bone marrow donations and cord
blood units
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