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Pediatric Outcomes Data Collection Instrument is a Useful Patient-Reported Outcome Measure for Physical Function in Children with Osteogenesis Imperfecta.
Genetics in Medicine ( IF 6.6 ) Pub Date : 2019-11-27 , DOI: 10.1038/s41436-019-0688-6 Chaya N Murali 1, 2 , David Cuthbertson 3 , Brady Slater 1 , Dianne Nguyen 1 , Alicia Turner 1, 2 , Gerald Harris 4 , V Reid Sutton 1, 2 , Brendan Lee 1, 2 , , Sandesh C S Nagamani 1, 2
Genetics in Medicine ( IF 6.6 ) Pub Date : 2019-11-27 , DOI: 10.1038/s41436-019-0688-6 Chaya N Murali 1, 2 , David Cuthbertson 3 , Brady Slater 1 , Dianne Nguyen 1 , Alicia Turner 1, 2 , Gerald Harris 4 , V Reid Sutton 1, 2 , Brendan Lee 1, 2 , , Sandesh C S Nagamani 1, 2
Affiliation
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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.
中文翻译：
儿科结果数据收集仪器是一种有用的患者报告结果测量方法，用于测量成骨不全儿童的身体功能。
目的 患者报告的结果测量 (PROM) 越来越被认为是临床试验中有价值的终点。儿科结果数据收集仪 (PODCI) 是一种用于患有肌肉骨骼疾病的儿童的 PROM。我们评估了 PODCI 在成骨不全症 (OI) 儿童中的有效性和可靠性。方法 使用 PODCI 对参与脆性骨疾病联盟开展的一项多中心研究的大量儿童进行身体机能和心理健康评估。身体功能评分与经过验证的观察者评分量表、运动功能简要评估 (BAMF) 以及心理健康评分相关。我们计算了检测具有临床意义的身体功能差异所需的样本量。结果 417 名患有 I、III 和 IV 型成骨不全症的儿童被纳入研究。 III型OI的身体功能评分显着低于I型和IV型OI。心理健康状况没有显着差异。 PODCI 身体功能评分与 BAMF 呈中度至强相关性。整体功能量表是身体机能的综合体，与心理健康并不总是相关。结论 PODCI 可以作为成骨不全儿童身体机能的可靠衡量标准，并提供有关患者报告的健康状况的宝贵信息以及检查该人群干预措施效用的新方法。
更新日期：2019-11-27
中文翻译：
儿科结果数据收集仪器是一种有用的患者报告结果测量方法，用于测量成骨不全儿童的身体功能。
目的 患者报告的结果测量 (PROM) 越来越被认为是临床试验中有价值的终点。儿科结果数据收集仪 (PODCI) 是一种用于患有肌肉骨骼疾病的儿童的 PROM。我们评估了 PODCI 在成骨不全症 (OI) 儿童中的有效性和可靠性。方法 使用 PODCI 对参与脆性骨疾病联盟开展的一项多中心研究的大量儿童进行身体机能和心理健康评估。身体功能评分与经过验证的观察者评分量表、运动功能简要评估 (BAMF) 以及心理健康评分相关。我们计算了检测具有临床意义的身体功能差异所需的样本量。结果 417 名患有 I、III 和 IV 型成骨不全症的儿童被纳入研究。 III型OI的身体功能评分显着低于I型和IV型OI。心理健康状况没有显着差异。 PODCI 身体功能评分与 BAMF 呈中度至强相关性。整体功能量表是身体机能的综合体，与心理健康并不总是相关。结论 PODCI 可以作为成骨不全儿童身体机能的可靠衡量标准，并提供有关患者报告的健康状况的宝贵信息以及检查该人群干预措施效用的新方法。
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