Development of CCFA Partners Kids & Teens: an Internet-Based Cohort of Pediatric IBD Michael D. Kappelman Wenli Chen Christopher F. Martin Beth Jaeger Erin Stoeber Lucy Goble Tania Kamphaus Robert S. Sandler Millie D. Long
Jan 17, 2016
Development of CCFA Partners Kids & Teens: an Internet-Based Cohort of Pediatric IBD
Michael D. Kappelman Wenli ChenChristopher F. Martin Beth JaegerErin Stoeber Lucy GobleTania Kamphaus Robert S. SandlerMillie D. Long
DISCLOSURES
Nothing to disclose
Background
Prospective cohorts of pediatric IBD are needed to:• 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• 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• 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:• 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 UC n=148
Current Medications
N (%) N (%)
5-ASA (oral) 117 (27) 57 (38)
Prednisone 41 (9) 23 (15)
Immunomodulator 186 (43) 52 (34)
Biologic 220 (51) 28 (18)
Medications ever-used
5-ASA (oral) 251 (58) 116 (76)
Prednisone 354 (82) 129 (85)
Immunomodulator 294 (68) 84 (55)
Biologic 249 (57) 57 (38)
Surgery 71 (16) 26 (17)
Patient Reported Outcomes
1 2 3 430
40
50
60
70
Mean PROMIS domain scores by IMPACT Quartile*
Depressive Symptoms
Anxiety
Peer relations
Fatigue
Pain Interference
IMPACT Quartile
*Higher IMPACT scores indicate better HRQOL
Patient Reported Outcomes
Remiss
ion (
sym
ptom
-free
)
A Few
Sym
ptom
s
Sympt
oms
Somet
imes
A Lot
of S
ympt
oms
Sympt
oms
All the
Tim
e30
40
50
60
70
Mean PROMIS domain scores by patient global assessment
Depressive Symptoms
Anxiety
Peer relations
Fatigue
Pain Interference
Patient Global Assessment
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
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