TY - JOUR
T1 - Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta
AU - Members of the BBD Consortium
AU - Murali, Chaya N.
AU - Cuthbertson, David
AU - Slater, Brady
AU - Nguyen, Dianne
AU - Turner, Alicia
AU - Harris, Gerald
AU - Sutton, V. Reid
AU - Lee, Brendan
AU - Rauch, Frank
AU - Glorieux, Francis
AU - Retrouvey, Jean Marc
AU - Esposito, Paul
AU - Rush, Eric
AU - Bober, Michael
AU - Eyre, David
AU - Gomez, Danielle
AU - Hart, Tracy
AU - Jain, Mahim
AU - Krakow, Deborah
AU - Krischer, Jeffrey
AU - Orwoll, Eric
AU - Raggio, Cathleen
AU - Smith, Peter
AU - Tosi, Laura
AU - Nagamani, Sandesh C.S.
N1 - Publisher Copyright:
© 2019, American College of Medical Genetics and Genomics.
PY - 2020/3/1
Y1 - 2020/3/1
N2 - 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.
AB - 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.
KW - Pediatric Outcomes Data Collection Instrument
KW - clinical trial readiness
KW - health-related quality of life
KW - osteogenesis imperfecta
KW - patient-reported outcome measure
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U2 - 10.1038/s41436-019-0688-6
DO - 10.1038/s41436-019-0688-6
M3 - Article
C2 - 31772349
AN - SCOPUS:85075585053
SN - 1098-3600
VL - 22
SP - 581
EP - 589
JO - Genetics in Medicine
JF - Genetics in Medicine
IS - 3
ER -