Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta
| Autor: | Danielle Gomez, Brady Slater, V. Reid Sutton, Mahim Jain, David Cuthbertson, Eric S. Orwoll, Frank Rauch, Deborah Krakow, Francis H. Glorieux, Tracy Hart, Chaya N. Murali, Jeffrey P. Krischer, Sandesh C.S. Nagamani, Jean-Marc Retrouvey, David R. Eyre, Dianne Nguyen, Michael B. Bober, Gerald F. Harris, Peter A. Smith, Alicia Turner, Laura L. Tosi, Paul Esposito, Cathleen L. Raggio, Eric T. Rush, Brendan Lee |
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| Rok vydání: | 2020 |
| Předmět: |
Male
medicine.medical_specialty Adolescent Population Psychological intervention Validity Prom Motor Activity Pediatrics Severity of Illness Index Article Cohort Studies Surveys and Questionnaires Outcome Assessment Health Care Humans Medicine Patient Reported Outcome Measures Child education Genetics (clinical) education.field_of_study business.industry Osteogenesis Imperfecta medicine.disease health-related quality of life Pediatric Outcomes Data Collection Instrument Clinical trial patient-reported outcome measure Osteogenesis imperfecta Sample size determination Child Preschool Quality of Life Physical therapy Female Patient-reported outcome business clinical trial readiness |
| Zdroj: | Genetics in medicine : official journal of the American College of Medical Genetics |
| ISSN: | 1098-3600 |
| DOI: | 10.1038/s41436-019-0688-6 |
| Popis: | Purpose: Patient-reported outcome measures (PROMs) are increasingly recognized as valuable endpoints in clinical trials. 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: 459 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. |
| Databáze: | OpenAIRE |
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