Faculty Collaboration Database

Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta. Genet Med 2020 Mar;22(3):581-589

Date

11/28/2019

Pubmed ID

31772349

Pubmed Central ID

PMC7060104

DOI

10.1038/s41436-019-0688-6

Scopus ID

2-s2.0-85075585053 (requires institutional sign-in at Scopus site)   15 Citations

Abstract

PURPOSE: Patient-reported outcome measures (PROMs) are increasingly recognized as valuable endpoints in clinical trials. The Pediatric Outcomes Data Collection Instrument (PODCI) is a PROM utilized in children with musculoskeletal disorders. We evaluated the validity and reliability of PODCI in children with osteogenesis imperfecta (OI).

METHODS: Physical functioning and psychological well-being were assessed using PODCI in a large cohort of children enrolled in a multicenter study conducted by the Brittle Bone Disorders Consortium. Physical function scores were correlated with a validated, observer-rated scale, Brief Assessment of Motor Function (BAMF), and with psychological well-being scores. We calculated sample sizes required to detect clinically meaningful differences in physical function.

RESULTS: Four hundred seventeen children with OI types I, III, and IV were enrolled. Physical function scores in OI type III were significantly lower than those in OI types I and IV. There were no significant differences in psychological well-being. PODCI physical function scores showed moderate-to-strong correlation with BAMF. The Global Functioning Scale, a composite of physical function, did not consistently correlate with psychological well-being.

CONCLUSION: PODCI can be a reliable measure of physical functioning in children with OI and offers valuable information about patient-reported health status and new ways to examine the utility of interventions in this population.

Author List

Murali CN, Cuthbertson D, Slater B, Nguyen D, Turner A, Harris G, Sutton VR, Lee B, Members of the BBD Consortium, Nagamani SCS

Author

Gerald Harris PhD Director in the Orthopaedic Research Engineering Center (OREC) department at Marquette University

MESH terms used to index this publication - Major topics in bold

Adolescent
Child
Child, Preschool
Cohort Studies
Female
Humans
Male
Motor Activity
Osteogenesis Imperfecta
Patient Reported Outcome Measures
Pediatrics
Quality of Life
Severity of Illness Index
Surveys and Questionnaires