Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta
Genetics in Medicine
clinical trial readiness; health-related quality of life; osteogenesis imperfecta; patient-reported outcome measure; Pediatric Outcomes Data Collection Instrument
© 2019, American College of Medical Genetics and Genomics. 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.
Murali, C., Cuthbertson, D., Slater, B., Nguyen, D., Turner, A., Harris, G., Sutton, V., Lee, B., Rauch, F., Glorieux, F., Retrouvey, J., Esposito, P., Rush, E., Bober, M., Eyre, D., Gomez, D., Hart, T., Jain, M., Krakow, D., Krischer, J., Orwoll, E., Raggio, C., Smith, P., Tosi, L., & Nagamani, S. (2020). Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta. Genetics in Medicine, 22 (3). http://dx.doi.org/10.1038/s41436-019-0688-6