Improved ALS clinical trials through frequent at-home self-assessment: a proof of concept study
Improved ALS clinical trials through frequent at-home self-assessment: a proof of concept study
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DOI：
10.1002/acn3.51096
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发表时间：
2020-06-09
影响因子：
5.3
通讯作者：
Shefner, Jeremy M.
中科院分区：
文献类型：
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作者：
Rutkove, Seward B.;Narayanaswami, Pushpa;Shefner, Jeremy M.
目的 通过让患者或护理人员在家进行频繁的自我评估，确定改善肌萎缩侧索硬化症 (ALS) 临床试验的潜力。方法和参与者 我们将 ALS 患者纳入一项为期 9 个月的非盲纵向研究，其中患者和护理人员使用几种不同的仪器获取每日数据，包括慢生命容量设备、握力计、基于电阻抗肌电图的健身设备、活动跟踪器、语音应用程序和 ALS 功能评级量表已修订。在两个时间点询问有关可接受性的问题。 结果 共有 113 人参与，其中 61 人（43 名男性，18 名女性，平均年龄 60.1 +/- 9.9 岁）收集了至少 7 天的数据并纳入分析。每日测量可以更准确地评估疾病进展的斜率，从而为假设的临床试验提供更小的样本量估计。例如，通过每日进行慢肺活量测量，计算出的样本量从每月测量的 882 名受试者减少到每研究组 182 名受试者。同样，每周而不是每月执行 ALS 功能评级量表的计算样本量为每组 73 人，而每组为 274 人。参与者普遍认为这些程序是可以接受的，并且对许多人来说，提高了他们对疾病的控制感。 解释 使用标准工具进行频繁的家庭测量有望跟踪进展并减少 ALS 临床试验的样本量要求，同时也为患者所接受。未来对该神经系统疾病和其他神经系统疾病的研究应考虑采用这种数据收集方法。
Objective To determine the potential for improving amyotrophic lateral sclerosis (ALS) clinical trials by having patients or caregivers perform frequent self-assessments at home.Methods and Participants We enrolled ALS patients into a nonblinded, longitudinal 9-month study in which patients and caregivers obtained daily data using several different instruments, including a slow-vital capacity device, a hand grip dynamometer, an electrical impedance myography-based fitness device, an activity tracker, a speech app, and the ALS functional rating scale-revised. Questions as to acceptability were asked at two time points.Results A total of 113 individuals enrolled, with 61 (43 men, 18 women, mean age 60.1 +/- 9.9 years) collecting a minimum of 7 days data and being included in the analysis. Daily measurements resulted in more accurate assessments of the slope of progression of the disease, resulting in smaller sample size estimates for a hypothetical clinical trial. For example, by performing daily slow-vital capacity measurements, calculated sample size was reduced to 182 subjects/study arm from 882/arm for monthly measurements. Similarly, performing the ALS functional rating scale weekly rather than monthly led to a calculated sample size of 73/arm as compared to 274/arm. Participants generally found the procedures acceptable and, for many, improved their sense of control of their disease.Interpretation Frequent at-home measurements using standard tools holds the prospect of tracking progression and reducing sample size requirements for clinical trials in ALS while also being acceptable to the patients. Future studies in this and other neurological disorders should consider adopting this approach to data collection.