Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases
child; chronic disease; patient-reported outcomes; PROMIS
Objectives To identify pediatric patient-reported outcomes (PROs) that are associated with chronic conditions and to evaluate the effects of chronic disease activity on PROs. Study design Participants 8-24 years-old and their parents were enrolled into 14 studies that evaluated PROMIS® PROs across 10 chronic conditions--asthma, atopic dermatitis, cancer, cancer survivors, chronic kidney disease, Crohn’s disease, juvenile idiopathic arthritis, lupus, sickle cell disease, and type 1 diabetes mellitus. PRO scores were contrasted with the United States general population of children using nationally representative percentiles. PRO-specific coefficients of variation were computed to illustrate the degree of variation in scores within versus between conditions. Condition-specific measures of disease severity and Cohens d effect sizes were used to examine PRO scores by disease activity. Results Participants included 2,975 child respondents and 2,392 parent respondents who provided data for 3,409 unique children: 52% were 5-12 years-old, 52% female, 25% African-American/Black, and 14% Hispanic. Across all 10 chronic conditions, children reported more anxiety, fatigue, pain, and mobility restrictions than the general pediatric population. Variation in PRO scores within chronic disease cohorts was equivalent to variation within the general population, exceeding between-cohort variation by factors of 1.9 (mobility) to 5.7 (anxiety). Disease activity was consistently associated with poorer self-reported health, and these effects were weakest for peer relationships. Conclusions Chronic conditions are associated with symptoms and functional status in children and adolescents across 10 different disorders. These findings highlight the need to complement conventional clinical evaluations with those obtained directly from patients themselves using PROs.
Forrest CB; Schuchard J; Bruno C; Amaral S; Cox ED; Flynn KE; Hinds PS; Huang IC; Kappelman MD; Krishnan JA; Kumar RB; Lai JS; Paller AS; Phipatanakul W; Schanberg LE; Sumino K; Weitzman ER; Reeve BB
The Journal of Pediatrics
2022
<a href="http://doi.org/10.1016/j.jpeds.2022.02.052" target="_blank" rel="noreferrer noopener">10.1016/j.jpeds.2022.02.052</a>
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
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.
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
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.jpeds.2021.10.053" target="_blank" rel="noreferrer noopener">10.1016/j.jpeds.2021.10.053</a>