强直性肌营养不良患者对患者报告结局指标的偏好
Myotonic dystrophy patient preferences in patient-reported outcome measures.
作者信息
Heatwole Chad, Johnson Nicholas, Dekdebrun Jeanne, Dilek Nuran, Eichinger Kate, Hilbert James, Luebbe Elizabeth, Martens William, Mcdermott Michael P, Thornton Charles, Moxley Richard
机构信息
Department of Neurology, University of Rochester Medical Center, Box 673, 601 Elmwood Avenue Rochester, New York, 14642, USA.
Department of Neurology, University of Utah Medical Center, Salt Lake City, Utah, USA.
出版信息
Muscle Nerve. 2018 Jan 12. doi: 10.1002/mus.26066.
INTRODUCTION
When preparing for clinical trials in myotonic dystrophy type-1 (DM1), it is important that researchers develop and identify patient-reported outcome measures with good measurement properties.
METHODS
Fifty-two DM1 patients enrolled in 2 clinical studies completed the Myotonic Dystrophy Health Index (MDHI), 36-Item Short Form Health Survey (version 2; SF-36v2), Individualized Neuromuscular Quality of Life questionnaire (INQoL), and a questionnaire comparing the relevance, usability, overall preference, and perceived responsiveness of each measure. The associations between instrument scores and physical function, genetic test results, and employment status were examined.
RESULTS
The MDHI was preferred over the INQoL in 13 of 13 areas and was preferred over the SF-36v2 in 9 of 13 areas. The MDHI was the only score that was associated with participant employment status, CTG repeat length, and the 3 measurements of clinical function.
DISCUSSION
The MDHI correlates well with physical function and is viewed favorably by participants in DM1 clinical studies. Muscle Nerve, 2018.
引言
在准备1型强直性肌营养不良症（DM1）的临床试验时，研究人员开发并确定具有良好测量特性的患者报告结局指标非常重要。
方法
52名参与2项临床研究的DM1患者完成了强直性肌营养不良健康指数（MDHI）、36项简明健康调查（第2版；SF-36v2）、个性化神经肌肉生活质量问卷（INQoL），以及一份比较各指标的相关性、可用性、总体偏好和感知反应性的问卷。研究了各量表得分与身体功能、基因检测结果和就业状况之间的关联。
结果
在13个领域中的13个方面，MDHI比INQoL更受青睐；在13个领域中的9个方面，MDHI比SF-36v2更受青睐。MDHI是唯一与参与者就业状况、CTG重复长度和临床功能的3项测量结果相关的得分。
讨论
MDHI与身体功能相关性良好，在DM1临床研究中受到参与者的好评。《肌肉与神经》，2018年。
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