Health-related quality of life in adults with osteogenesis imperfecta.
Health-related quality of life in adults with osteogenesis imperfecta.
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DOI：
10.1111/cge.13939
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发表时间：
2021-06
影响因子：
3.5
通讯作者：
Nagamani SCS
中科院分区：
文献类型：
--
作者：
Murali CN;Slater B;Musaad S;Cuthbertson D;Nguyen D;Turner A;Azamian M;Tosi L;Rauch F;Sutton VR;Lee B;Members of the BBD Consortium;Nagamani SCS
患者报告的结局指标（PROM）越来越多地用作临床试验的终点。简明健康调查量表-12（SF-12 v2）是成人的通用PROM。我们试图评估SF-12 v2在成人成骨不全（OI）中的有效性。在一项多中心、观察性、自然史研究中，在一个大型成人队列中评估了身心健康相关的生活质量（HRQoL）。身体HRQoL评分与吉列功能评估问卷（GFAQ）相关。我们计算了交叉和平行组设计的临床试验所需的样本量，以检测有临床意义的身体HRQoL变化。入组了302名I型、III型和IV型OI成人。研究人群的身体HRQoL评分低于人群标准。对于I型和IV型OI，身体HRQoL评分与GFAQ中度相关。我们没有发现精神和身体HRQoL之间的相关性。从临床试验准备的角度来看，我们表明SF-12 v2可靠地测量了成人OI的身体功能，并可用于交叉试验，以检测小样本量的有意义的身体HRQoL变化。本研究表明，SF-12 v2可用于测量OI干预后身体HRQoL的变化。
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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影响因子：
6.2
作者：
Glorieux, Francis H.;Devogelaer, Jean-Pierre;Winkle, Peter J.
通讯作者：
Winkle, Peter J.
影响因子：
1.2
作者：
Pillion, Josepb P.;Shapiro, Jay
通讯作者：
Shapiro, Jay
影响因子：
2
作者：
Cohen, Julie S.;Biesecker, Barbara B.
通讯作者：
Biesecker, Barbara B.
影响因子：
3.7
作者：
Slade A;Isa F;Kyte D;Pankhurst T;Kerecuk L;Ferguson J;Lipkin G;Calvert M
通讯作者：
Calvert M
影响因子：
3.5
作者：
Patel RM;Nagamani SC;Cuthbertson D;Campeau PM;Krischer JP;Shapiro JR;Steiner RD;Smith PA;Bober MB;Byers PH;Pepin M;Durigova M;Glorieux FH;Rauch F;Lee BH;Hart T;Sutton VR
通讯作者：
Sutton VR