REVISED UPPER LIMB MODULE FOR SPINAL MUSCULAR ATROPHY: DEVELOPMENT OF A NEW MODULE
REVISED UPPER LIMB MODULE FOR SPINAL MUSCULAR ATROPHY: DEVELOPMENT OF A NEW MODULE
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DOI：
10.1002/mus.25430
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发表时间：
2017-06-01
期刊：
影响因子：
3.4
通讯作者：
Mercuri, Eugenio
中科院分区：
文献类型：
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作者：
Mazzone, Elena S.;Mayhew, Anna;Mercuri, Eugenio
介绍：由于现有的量表在临床范围的极端情况下缺乏灵敏度，因此越来越需要一种可靠的临床测量方法来评估脊髓性肌萎缩症（SMA）的上肢运动功能。我们报告了修订的上肢模块（RULM）的开发，这是一种专门针对SMA患者上肢功能的评估。研究方法：一个具有特定神经肌肉专业知识的国际小组对目前可用于评估SMA上肢功能的量表进行了全面审查。这篇综述促进了对现有上肢功能量表的修订，使其成为一个更可靠的临床量表。结果如下：该量表的多次修订包括统计分析，并捕获临床相关变化，以满足监管机构和倡导团体的要求。结论：由此产生的RULM量表显示出良好的信度和效度，使其成为评估SMA人群上肢功能的多中心临床研究的合适工具。
Introduction: There is a growing need for a robust clinical measure to assess upper limb motor function in spinal muscular atrophy (SMA), as the available scales lack sensitivity at the extremes of the clinical spectrum. We report the development of the Revised Upper Limb Module (RULM), an assessment specifically designed for upper limb function in SMA patients. Methods: An international panel with specific neuromuscular expertise performed a thorough review of scales currently available to assess upper limb function in SMA. This review facilitated a revision of the existing upper limb function scales to make a more robust clinical scale. Results: Multiple revisions of the scale included statistical analysis and captured clinically relevant changes to fulfill requirements by regulators and advocacy groups. Conclusions: The resulting RULM scale shows good reliability and validity, making it a suitable tool to assess upper extremity function in the SMA population for multi-center clinical research.