Quantitative strength testing in ALS clinical trials
Quantitative strength testing in ALS clinical trials
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DOI：
10.1212/wnl.0000000000002941
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发表时间：
2016-08-09
期刊：
影响因子：
9.9
通讯作者：
Cudkowicz, Merit
中科院分区：
文献类型：
--
作者：
Shefner, Jeremy M.;Liu, Dawei;Cudkowicz, Merit
目的：研究属性的定量强度测试，使用手持测力仪（HHD）作为一个有效的措施，在2个大型3期肌萎缩侧索硬化症（ALS）trials.Methods：在3期试验头孢曲松和dexpramipexole，513和943例患者，分别参加了双盲，随机，安慰剂对照试验，计划随访至少1年。在头孢曲松研究中每3个月对患者进行一次研究，在右旋普拉克索研究中每2个月对患者进行一次研究。HHD的评价者接受了培训，并且必须证明力量测试的充分性能;测试范例涉及双侧上肢和下肢的9个肌肉群。两种药物均未显著影响任何结局指标。强度测量结果通过单个肌肉和megascore进行评估，megascore平均缩放强度测量结果以产生肌肉力量的总体测量结果。计算下降率与患者内和患者间测量变异性相结合的测量值，即变化率的变异系数，显示HHD总体表现略低于肌萎缩侧索硬化症功能评定量表修订版（ALSFRS-R），但优于肺活量。个体肌肉与对侧的相同肌肉以及同一身体区域的其他肌肉高度相关。力量下降与ALSFRS-R和肺活量相关。结论：使用HHD进行定量力量测试是ALS下降的可靠且可重复的测量方法。
Objective: To study the attributes of quantitative strength testing using hand-held dynamometry (HHD) as an efficacy measure in 2 large phase 3 amyotrophic lateral sclerosis (ALS) trials.Methods: In the phase 3 trials of ceftriaxone and dexpramipexole, 513 and 943 patients, respectively, were enrolled in double-blind, randomized, placebo-controlled trials with planned follow-up of at least 1 year. Patients were studied every 3 months in the ceftriaxone study and every 2 months in the dexpramipexole study. Evaluators of HHD were trained and had to show evidence of adequate performance of strength testing; the testing paradigm involved testing 9 muscle groups in the upper and lower extremity bilaterally. Neither drug significantly affected any outcome measure. Strength measurements were evaluated by individual muscle and by megascores, which averaged scaled strength measures to produce an overall measure of muscle strength.Results: A measure combining rate of decline with both within-and between-patient variabilities of measurement, the coefficient of variation for rate of change, was calculated, and showed that HHD overall performed slightly less well than Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) but better than vital capacity. Individual muscles were highly correlated to the identical muscles on the contralateral side, as well as to other muscles in the same body region. Strength decline was correlated both with ALSFRS-R and vital capacity.Conclusion: Quantitative strength testing using HHD is a reliable and reproducible measure of decline in ALS.