Electrical impedance myography to assess outcome in amyotrophic lateral sclerosis clinical trials
Electrical impedance myography to assess outcome in amyotrophic lateral sclerosis clinical trials
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DOI：
10.1016/j.clinph.2007.08.004
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发表时间：
2007-11-01
影响因子：
4.7
通讯作者：
Shiffman, Carl A.
中科院分区：
文献类型：
--
作者：
Rutkove, Seward B.;Zhang, Hui;Shiffman, Carl A.
目的：用于肌萎缩侧索硬化（ALS）临床试验的标准结局指标，包括ALS功能评定量表修订版（ALSFRS-R）、最大随意等长收缩试验（MVICT）和手动肌肉试验（MMT），在检测细微疾病进展方面的能力有限。电阻抗肌电图（EIM）是一种新的非侵入性技术，通过测量局部组织阻抗提供肌肉健康的定量数据。本研究探讨EIM是否可以提供一个新的结果测量用于ALS临床试验works.Methods：15 ALS患者进行了重复EIM测量的一个或多个肌肉在一段时间内长达18个月的主要结果变量，θ（Z-最大值），测量。然后，使用与过去应用于MVICT的方法相同的方法计算theta（z-max）megascore。这和MMT的数据，然后被用来评估每个措施的统计权力，以检测一个给定的影响，在一个假设的计划临床治疗trial.Results的疾病进展：θ（z-max）的平均下降约21%的测试期间，平均在所有患者和所有测试的肌肉。在我们计划的假设试验中，theta（z-max）megascore检测10%治疗效果的功效为73%，而MMT的功效为28%。这些结果也有利地比较ALSFRS-R和MVICT臂Megascore的历史数据从试验塞来昔布在ALS，其中两个措施只有23%的权力，以检测同样的10%治疗effects.Conclusions：theta（z-max），megascore可能会提供一个强大的新的成果措施ALS临床试验。将EIM应用于未来的ALS试验可能会允许更小，更快的研究，提高检测疾病和治疗效果的微妙进展的能力。(c)2007年国际临床神经生理学联合会。由Elsevier爱尔兰有限公司出版。保留所有权利。
Objective: Standard outcome measures used for amyotrophic lateral sclerosis (ALS) clinical trials, including the ALS functional rating scale-revised (ALSFRS-R), maximal voluntary isometric contraction testing (MVICT), and manual muscle testing (MMT), are limited in their ability to detect subtle disease progression. Electrical impedance myography (EIM) is a new non-invasive technique that provides quantitative data on muscle health by measuring localized tissue impedance. This study investigates whether EIM could provide a new outcome measure for use in ALS clinical trials work.Methods: Fifteen ALS patients underwent repeated EIM measurements of one or more muscles over a period of up to 18 months and the primary outcome variable, theta(z-max), measured. The theta(z-max) megascore was then calculated using the same approach as has been applied in the past for MVICT. This and the MMT data were then used to assess each measure's statistical power to detect a given effect on disease progression in a hypothetical planned clinical therapeutic trial.Results: theta(z-max) showed a mean decline of about 21% for the test period, averaged across all patients and all tested muscles. The theta(z-max) megascore had a power of 73% to detect a 10% treatment effect in our planned hypothetical trial, as compared to a 28% power for MMT. These results also compared favorably to historical data for ALSFRS-R and MVICT arm megascore from the trial of celecoxib in ALS, where both measures had only a 23% power to detect the same 10% treatment effect.Conclusions: The theta(z-max), megascore may provide a powerful new outcome measure for ALS clinical trials.Significance: The application of EIM to future ALS trials may allow for smaller, faster studies with an improved ability to detect subtle progression of the disease and treatment effects. (c) 2007 International Federation of Clinical Neurophysiology. Published by Elsevier Ireland Ltd. All rights reserved.