Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials
Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials
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DOI：
10.1002/acn3.50958
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发表时间：
2019-12-25
影响因子：
5.3
通讯作者：
Zaidman, Craig
中科院分区：
文献类型：
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作者：
Leitner, Melanie L.;Kapur, Kush;Zaidman, Craig
目的评价电阻抗肌电图（EIM）对男性DMD患者病情进展的敏感性。方法和参与者：一项非盲的纵向队列研究，包括29名非卧床和15名非卧床DMD男孩和年龄相似的健康男孩。作为多中心研究的一部分，对受试者进行了长达1年的随访，并使用Myolex（R）mView（TM）EIM系统进行了评估。结果非卧床组EIM 100 kHz电阻值与正常组相比有显著性差异。例如，在下肢肌肉中，EIM 100 kHz电阻值在12个月内的平均变化导致估计的效应大小为1.58。基于这些结果，假设50%的治疗效果，12个月的临床试验将需要26名DMD患者/组。在不能行走的男孩中，上肢肌肉的EIM变化更大。例如，二头肌在100 kHz阻力下在12个月时的估计效应量为1.92。基于这些结果，假设治疗效果为50%，12个月的临床试验将需要18例卧床DMD患者/组。纵向变化的100 kHz电阻值的动态男孩与纵向变化的定时仰卧到站立测试。EIM在整个研究期间耐受性良好。解释本研究支持EIM 100 kHz电阻对非卧床和卧床男孩的DMD进展敏感。鉴于该技术的易用性和广泛的年龄范围的效用，它应该被用作一个探索性的终点，在未来的临床治疗试验的DMD。试用注册：Clincialtrials.gov注册编号NCT 02340923
Objective To evaluate the sensitivity of electrical impedance myography (EIM) to disease progression in both ambulatory and non-ambulatory boys with DMD. Methods and Participants A non-blinded, longitudinal cohort study of 29 ambulatory and 15 non-ambulatory boys with DMD and age-similar healthy boys. Subjects were followed for up to 1 year and assessed using the Myolex (R) mView(TM) EIM system as part of a multicenter study. Results In the ambulatory group, EIM 100 kHz resistance values showed significant change compared to the healthy boys. For example, in lower extremity muscles, the average change in EIM 100 kHz resistance values over 12 months led to an estimated effect size of 1.58. Based on these results, 26 DMD patients/arm would be needed for a 12-month clinical trial assuming a 50% treatment effect. In non-ambulatory boys, EIM changes were greater in upper limb muscles. For example, biceps at 100kHz resistance gave an estimated effect size of 1.92 at 12 months. Based on these results, 18 non-ambulatory DMD patients/arm would be needed for a 12-month clinical trial assuming a 50% treatment effect. Longitudinal changes in the 100 kHz resistance values for the ambulatory boys correlated with the longitudinal changes in the timed supine-to-stand test. EIM was well-tolerated throughout the study. Interpretation This study supports that EIM 100 kHz resistance is sensitive to DMD progression in both ambulatory and non-ambulatory boys. Given the technology's ease of use and broad age range of utility it should be employed as an exploratory endpoint in future clinical therapeutic trials in DMD. Trial Registration: Clincialtrials.gov registration #NCT02340923