Novel outcome measures for Charcot-Marie-Tooth disease: validation and reliability of the 6-min walk test and StepWatch Activity Monitor and identification of the walking features related to higher quality of life
Novel outcome measures for Charcot-Marie-Tooth disease: validation and reliability of the 6-min walk test and StepWatch Activity Monitor and identification of the walking features related to higher quality of life
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DOI：
10.1111/ene.13033
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发表时间：
2016-08-01
影响因子：
5.1
通讯作者：
Contini, M.
中科院分区：
文献类型：
--
作者：
Padua, L.;Pazzaglia, C.;Contini, M.
背景和目的腓骨肌萎缩症（CMT）是最常见的遗传性神经病，但治疗选择一直局限于症状管理。过去的药理学试验失败了，可能是由于不敏感的结果措施（OM）。本研究的目的是评估6分钟步行试验（6MWT）和StepWatch活动监测（SAM）与其他先前验证的OMs在CMT疾病中的有效性和可靠性。（104 CMT1A，27 CMT1B，37与X连锁CMT）从意大利中心专门在CMT carry.ResultsStatistical分析表明，6MWT是高度相关的所有以前使用的OM。一些，但不是所有的，SAM参数与常用的OM，但可能提供更多的信息quality of life.ConclusionsThe目前的研究表明，6MWT和SAM作为OM CMT的有效性和可靠性。此外，SAM提供了与生活质量指标更好相关的数据，使其在未来的康复试验中非常有用。
Background and purposeCharcot-Marie-Tooth (CMT) disease is the most common inherited neuropathy, but therapeutic options have been limited to symptom management. Past pharmacological trials have failed, possibly due to insensitive outcome measures (OMs). The aim of the current study was to evaluate the validity and reliability of the 6-min walk test (6MWT) and StepWatch Activity Monitoring (SAM) with other previously validated OMs in CMT disease.MethodsA prospective multicenter study was performed, consecutively enrolling 168 CMT patients (104 with CMT1A, 27 with CMT1B, 37 with X-linked CMT) from Italian centers specializing in CMT care.ResultsStatistical analysis showed that the 6MWT was highly related with all previously used OMs. Some, but not all, SAM parameters were related to commonly used OMs but may provide more information about quality of life.ConclusionsThe current study demonstrated the validity and reliability of the 6MWT and SAM as OMs for CMT. Moreover, SAM provides data that correlate better with quality of life measures, making it useful in future rehabilitation trials.