PIH33 Generic Patient-Reported Outcome Measures (PROMS) for Use in Pediatric Age Group: Review of Available Measures

Abstract

Various generic patient-reported outcomes measures (PROMs) are available to measure health-related quality of life (HRQoL), symptoms, psychological and behavioral function in children. Pediatric PROMs are required to appropriately accommodate children's cognitive development, reading comprehension and language skills. Generic PROMs are suitable to be used in clinical trials when appropriate disease-specific PROMs are unavailable. This review aimed to identify and systematically evaluate generic PROMs (self- and proxy-reported) for children and adolescents below 18 years of age. Literature searches were performed in Medline and Embase until January 2019. Keywords like “pediatric”, “HRQoL” were used to identify generic PROMs developed or used in the pediatric population. Selected PROMs underwent domain-level evaluation to inform on conceptual coverage. Psychometric properties were evaluated on the basis of PRO review considerations of the FDA Guidance (2009). A total of 846 abstracts were identified and were screened to obtain a list of generic PROMs. Based on domain-level analysis, PROMs were classified as: HRQoL (n=39), health utility (n=7), psychological functioning (n=40), and behavioral (n=20). For further in-depth evaluation, 22 PROMs which had evidence of use in clinical trials were selected. The Pediatric Quality of Life Inventory 4.0 (PedsQL) was the most commonly (41.3%) used PROM in the included studies. For establishing the content validity, all except KINDL, and Childhood Health Assessment questionnaire (CHAQ) involved children (or parents). Complete psychometric testing including reliability, validity, sensitivity to change (responsiveness) and minimal clinically interpretable difference (MCID) was reported for the CHAQ, PROMIS- Pediatric Global Health Measure (PGH-7), PedsQL, and Pediatric Outcomes Data Collection Instrument (PODCI). The PedsQL, KINDL, and DISABKIDS also had disease-specific modules. Many generic pediatric PROMs are available but only PedsQL, PGH-7 and PODCI have evidence of complete psychometric validation. Selection of pediatric PROMs for use in clinical trials should be based on conceptual coverage, content validity and psychometric strength.