脊髓性肌萎缩症上肢模块修订版：新模块的开发
Revised upper limb module for spinal muscular atrophy: Development of a new module.
作者信息
Mazzone Elena S, Mayhew Anna, Montes Jacqueline, Ramsey Danielle, Fanelli Lavinia, Young Sally Dunaway, Salazar Rachel, De Sanctis Roberto, Pasternak Amy, Glanzman Allan, Coratti Giorgia, Civitello Matthew, Forcina Nicola, Gee Richard, Duong Tina, Pane Marika, Scoto Mariacristina, Pera Maria Carmela, Messina Sonia, Tennekoon Gihan, Day John W, Darras Basil T, De Vivo Darryl C, Finkel Richard, Muntoni Francesco, Mercuri Eugenio
机构信息
Department of Paediatric Neurology, Catholic University, Largo Gemelli 8, 00168, Rome, Italy.
Institute of Genetic Medicine, Newcastle University, Newcastle, United Kingdom.
出版信息
Muscle Nerve. 2017 Jun;55(6):869-874. doi: 10.1002/mus.25430. Epub 2017 Feb 6.
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. Muscle Nerve 55: 869-874, 2017.
引言
由于现有的评估量表在临床症状谱的两端缺乏敏感性，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）患者的上肢运动功能。我们报告了修订后的上肢模块（RULM）的开发情况，这是一种专门为SMA患者上肢功能设计的评估方法。
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患者上肢功能的量表进行了全面审查。这次审查促进了对现有上肢功能量表的修订，以形成一个更可靠的临床量表。
结果
该量表经过多次修订，包括统计分析，并捕捉到了临床上相关的变化，以满足监管机构和倡导团体的要求。
结论
最终得到的RULM量表显示出良好的信度和效度，使其成为评估SMA人群上肢功能以进行多中心临床研究的合适工具。《肌肉与神经》55: 869 - 874, 2017年。
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