Journal
CLINICAL GENETICS
Volume 99, Issue 6, Pages 772-779Publisher
WILEY
DOI: 10.1111/cge.13939
Keywords
natural history; osteogenesis imperfecta; quality of life; sample size
Categories
Funding
- Eunice Kennedy Shriver National Institute of Child Health and Human Development [P50HD103555]
- National Center for Advancing Translational Sciences, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Instutute of Dental and Craniofacial Research, National Institute of Child Health and Human Development [U54AR068069]
- National Institutes of Health [T32GM0752642]
- Osteogenesis Imperfecta Foundation
- Shriners of North America
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PROMs are increasingly used in clinical trials, with SF-12v2 being a commonly used generic measure. In this study, SF-12v2 was found to be valid in assessing physical HRQoL in adults with OI. However, no correlations were found between mental and physical HRQoL in this population.
Patient-reported outcome measures (PROMs) are increasingly utilized as endpoints in clinical trials. The Short Form Health Survey-12 (SF-12v2) is a generic PROM for adults. We sought to evaluate the validity of SF-12v2 in adults with osteogenesis imperfecta (OI). Physical and mental health-related quality of life (HRQoL) were assessed in a large cohort of adults in a multicenter, observational, natural history study. Physical HRQoL scores were correlated with the Gillette Functional Assessment Questionnaire (GFAQ). We calculated sample sizes required in clinical trials with crossover and parallel-group designs to detect clinically meaningful changes in physical HRQoL. Three hundred and two adults with OI types I, III, and IV were enrolled. Physical HRQoL scores in the study population were lower than population norms. Physical HRQoL scores moderately correlated with GFAQ for OI types I and IV. We found no correlations between mental and physical HRQoL. From a clinical trial readiness perspective, we show that SF-12v2 reliably measures physical function in adults with OI and can be utilized in crossover trials to detect meaningful physical HRQoL changes with small sample sizes. This study shows that SF-12v2 can be used to measure changes in physical HRQoL in response to interventions in OI.
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