Abstract：
Remote study visits (RSVs) are emerging as important tools for clinical research. We tested the feasibility of using RSVs to evaluate patients with myotonic dystrophy type 1 (DM1), including remote quantitative assessment of muscle function, and we assessed correlations of remote assessments with patient-reported function.
Twenty three subjects with DM1 were consented remotely. Toolkits containing a tablet computer, grip dynamometer, and spirometer were shipped to participants. The tablets were loaded with software for video-conferencing and questionnaires about functional impairment, patient experience with technology, and willingness to participate in future remote studies. Grip strength, forced vital capacity, peak cough flow, timed-up-and-go (TUG), and grip myotonia (hand opening time) were determined during RSVs. We assessed correlations of remote assessments with patient-reported outcomes of muscle function and with CTG repeat size.
All 23 subjects completed RSVs. 95% of participants were able to complete all components of the remote study. All toolkit components were returned upon completion. Grip strength and TUG demonstrated moderate to strong correlations with self-reported inventories of upper and lower extremity impairment, respectively (ρ = 0.7 and ρ = -0.52). A total of 91% of subjects expressed interest in participating in future RSVs.
Results of this study support the feasibility of using portable devices and video-conferencing for remote collection of patient-reported outcomes and quantitative assessment of muscle function in DM1.
Twenty three subjects with DM1 were consented remotely. Toolkits containing a tablet computer, grip dynamometer, and spirometer were shipped to participants. The tablets were loaded with software for video-conferencing and questionnaires about functional impairment, patient experience with technology, and willingness to participate in future remote studies. Grip strength, forced vital capacity, peak cough flow, timed-up-and-go (TUG), and grip myotonia (hand opening time) were determined during RSVs. We assessed correlations of remote assessments with patient-reported outcomes of muscle function and with CTG repeat size.
All 23 subjects completed RSVs. 95% of participants were able to complete all components of the remote study. All toolkit components were returned upon completion. Grip strength and TUG demonstrated moderate to strong correlations with self-reported inventories of upper and lower extremity impairment, respectively (ρ = 0.7 and ρ = -0.52). A total of 91% of subjects expressed interest in participating in future RSVs.
Results of this study support the feasibility of using portable devices and video-conferencing for remote collection of patient-reported outcomes and quantitative assessment of muscle function in DM1.
摘要：
远程研究访问（RSV）正在成为临床研究的重要工具。我们测试了使用RSV评估强直性肌营养不良1型（DM1）患者的可行性，包括肌肉功能的远程定量评估，我们评估了远程评估与患者报告功能的相关性。
23名DM1受试者远程同意。包含平板电脑的工具包，握力计,和肺活量计被运送给参与者。这些平板电脑装载了用于视频会议的软件和关于功能障碍的问卷，患者对技术的体验，并愿意参与未来的远程研究。握力,强制肺活量,咳嗽流量峰值,定时启动(TUG)，和握力肌强直(手打开时间)在RSV期间测定。我们评估了远程评估与患者报告的肌肉功能结果以及CTG重复大小的相关性。
所有23名受试者均完成RSV。95%的参与者能够完成远程研究的所有组成部分。所有工具包组件都在完成后返回。握力和TUG与自我报告的上肢和下肢减值库存表现出中等至强烈的相关性，分别为(ρ=0.7和ρ=-0.52)。总共91%的受试者表示有兴趣参与未来的RSV。
本研究结果支持使用便携式设备和视频会议远程收集患者报告的结果和定量评估DM1肌肉功能的可行性。
23名DM1受试者远程同意。包含平板电脑的工具包，握力计,和肺活量计被运送给参与者。这些平板电脑装载了用于视频会议的软件和关于功能障碍的问卷，患者对技术的体验，并愿意参与未来的远程研究。握力,强制肺活量,咳嗽流量峰值,定时启动(TUG)，和握力肌强直(手打开时间)在RSV期间测定。我们评估了远程评估与患者报告的肌肉功能结果以及CTG重复大小的相关性。
所有23名受试者均完成RSV。95%的参与者能够完成远程研究的所有组成部分。所有工具包组件都在完成后返回。握力和TUG与自我报告的上肢和下肢减值库存表现出中等至强烈的相关性，分别为(ρ=0.7和ρ=-0.52)。总共91%的受试者表示有兴趣参与未来的RSV。
本研究结果支持使用便携式设备和视频会议远程收集患者报告的结果和定量评估DM1肌肉功能的可行性。
