Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn’s Disease

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn’s Disease

Miller TL; Schuchard J; Carle Adam C; Forrest CB; Kappelman MD; Adler J; Ammoury RF; Bass D; Bass J; Benkov K; Bousvaros A; Boyle B; Cabrera JM; Colletti R; Dorsey JM; Ebach DR; Firestine AM; Gulati A; Hoffenberg EJ; Jester TW; Kaplan JL; Kugathasan S; Kusek ME; Leibowitz I; Linville TM; Margolis P; Minar P; Rios ZM; Moses J; Palomo PJ; Pappa H; Pashankar DS; Saeed SA; Samson CM; Sandberg KC; Steiner SJ; Strople J; Sullivan JS; Tung J; Wali P

The Journal of Pediatrics

2021

outcome measure; child; patient-reported outcomes; inflammatory bowel disease; PROMIS; Crohn’s disease

Objectives To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness. Study design We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with Crohn's disease (CD). We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating PRO score changes among patients who did and patients who did not experience improvement in disease activity. Results Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for the Pain Interference, Fatigue, and inflammatory bowel disease (IBD) Symptoms domains and higher scores for the Positive Affect domain. Conclusions This study demonstrates the responsiveness of the PROMIS pediatric measures of Fatigue and Pain Interference as study endpoints in a large, multicenter pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS pediatric measures as reliable PRO endpoints for clinical trials.

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