Development of CCFA Partners Kids & Teens:
an Internet-Based Cohort of Pediatric IBD
Michael D. Kappelman
Christopher F. Martin
Erin Stoeber
Tania Kamphaus
Millie D. Long
Wenli Chen
Beth Jaeger
Lucy Goble
Robert S. Sandler
DISCLOSURES
Nothing to disclose
Background
Prospective cohorts of pediatric IBD are needed to:
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Study disease natural history
Identify prognostic factors and/or environmental risk factors of relapse
Evaluate of the safety and effectiveness of treatments
Most pediatric IBD cohorts in the U.S. rely on center-based
recruitment and data collection
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Involve patients followed at large and/or academically oriented practices
Very time and resource intensive
Focus on physician-reported data and biosample collection
CCFA Partners is an Internet-based cohort of adults with IBD
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Large and diverse study population: >12,000 patients from all 50 states,
treated in academic and private practice settings
Web-based recruitment and data collection efficient and cost-effective
Focus on patient reported exposures, health behaviors, and outcomes
Objective
To develop an internet based cohort of pediatric IBD patients in
collaboration with the Crohn’s and Colitis Foundation of America
Methods
• Recruited children (< 18 years of age) with self-reported IBD
through email invitations to CCFA constituents, promotion on
the CCFA website, social media, and chapter events
• After informed consent/assent, parents and their children
completed a baseline web-based survey containing questions
regarding:
• Demographics
• Disease characteristics (hospitalization, surgery,
anthropometrics, location, extent)
• Disease activity (Patient Global Assessment, Short CDAI,
PUCAI, and Manitoba Index)
• Treatments
• Adherence
• Quality of life (Impact 35) and other Patient Reported
Outcomes
Methods 2
• Surveys developmentally appropriate, with increasing levels
of self-report based upon age
• Validated survey instruments used when available
• At 3 month intervals, an update on study progress and
educational materials developed by CCFA emailed to
participants
• Follow up surveys planned Q6 months
• Enrollment and linkage to adult CCFA Partners planned upon
18th birthday
www.ccfapartners.org
Preliminary Findings (First 4 months)
Enrollment:
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570 Participants from 47 U.S. states and 6 countries
316 male (55%), 254 female (45%)
422 CD (74%), 134 UC (24%), 14 IC (2%)
Median age: 13 years
Median age at diagnosis: 9 years
Treatment Patterns
CD n=422
Current
Medications
UC n=148
N (%)
N (%)
5-ASA (oral)
117 (27)
57 (38)
Prednisone
41 (9)
23 (15)
Immunomodulator
186 (43)
52 (34)
Biologic
220 (51)
28 (18)
5-ASA (oral)
251 (58)
116 (76)
Prednisone
354 (82)
129 (85)
Immunomodulator
294 (68)
84 (55)
Biologic
249 (57)
57 (38)
71 (16)
26 (17)
Medications everused
Surgery
Patient Reported Outcomes
Mean PROMIS domain scores by IMPACT Quartile*
70
60
Depressive Symptoms
50
Anxiety
Peer relations
Fatigue
Pain Interference
40
30
1
2
3
4
IMPACT Quartile
*Higher IMPACT scores indicate better HRQOL
Patient Reported Outcomes
Mean PROMIS domain scores by patient global assessment
70
60
Depressive Symptoms
50
Anxiety
Peer relations
Fatigue
Pain Interference
40
30
Remission
(symptom-free)
A Few Symptoms
Symptoms
A Lot of Symptoms
Sometimes
Patient Global Assessment
Symptoms All the
Time
Conclusion
CCFA Partners Kids & Teens is a novel internet-based cohort of
pediatric IBD
• Partnership between CCFA, patient community, and research
community
• Channel for all children with IBD to participate in research,
regardless of treating physician/practice
• Preliminary findings suggest that internet based recruitment and
data collection are feasible
• Growth in enrollment and long term cohort retention will be critical to
the success of this project
Implications and future directions
Focus on PROs will become increasingly important in
observational research
• Complement to clinical endpoints, which are rapidly moving towards
mucosal healing rather than measures of patient well being
Dual enrollment and linkage with clinical cohorts (i.e. Risk,
Protect) will allow analyses that combine clinician-reported data,
patient-reported data, and biospecimens
Long term outcomes of pediatric IBD
• Opportunity for follow up after patients transition from pediatric care
Platform for ancillary studies
• Secondary analyses, supplemental surveys, simple clinical trials,
biospecimen collection
Please Get involved
Submit ancillary studies
Promote study to your patients