Journal article
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
The Journal of pediatrics, Vol.242, pp.86-92.e3
03/2022
DOI: 10.1016/j.jpeds.2021.10.053
PMCID: PMC8882140
PMID: 34740588
Abstract
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.
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.
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.
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.
Details
- Title: Subtitle
- 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
- Creators
- Talya L. Miller - University of North Carolina at Chapel HillJulia Schuchard - Children's Hospital of PhiladelphiaAdam C. Carle - University of Cincinnati Medical CenterChristopher B. Forrest - Children's Hospital of PhiladelphiaMichael D. Kappelman - University of North Carolina at Chapel HillJeremy AdlerRana F. AmmouryDorsey BassJulie BassKeith BenkovAthos BousvarosBrendan BoyleJosé M. CabreraRichard CollettiJill M. DorseyDawn R. EbachAnn M. FirestineAjay GulatiEdward J. HoffenbergTraci W. JesterJess L. KaplanSubra KugathasanMark E. KusekIan LeibowitzTiffany M. LinvillePeter MargolisPhillip MinarZarela Molle RiosJonathan MosesPablo J. PalomoHelen PappaDinesh S. PashankarShehzad A. SaeedCharles M. SamsonKelly C. SandbergSteven J. SteinerJennifer StropleJillian S. SullivanJeanne TungPrateek WaliClinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) Study Group
- Resource Type
- Journal article
- Publication Details
- The Journal of pediatrics, Vol.242, pp.86-92.e3
- DOI
- 10.1016/j.jpeds.2021.10.053
- PMID
- 34740588
- PMCID
- PMC8882140
- NLM abbreviation
- J Pediatr
- ISSN
- 0022-3476
- eISSN
- 1097-6833
- Publisher
- Elsevier Inc
- Grant note
- DOI: 10.13039/100000069, name: National Institute of Arthritis and Musculoskeletal and Skin Diseases, award: U19AR069525; DOI: 10.13039/100006093, name: Patient-Centered Outcomes Research Institute; DOI: 10.13039/100000002, name: National Institutes of Health; DOI: 10.13039/100007028, name: Leona M. and Harry B. Helmsley Charitable Trust
- Language
- English
- Date published
- 03/2022
- Academic Unit
- Stead Family Department of Pediatrics; Gastroenterology, Hepatology, Pancreatology, and Nutrition
- Record Identifier
- 9984354041302771
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