a Department of Neurology , Beth Israel Deaconess Medical Center , Boston , MA , USA.
b Department of Neurology , Barrow Neurological Institute , Phoenix , AZ , USA.
Amyotroph Lateral Scler Frontotemporal Degener. 2019 Feb;20(1-2):61-67. doi: 10.1080/21678421.2018.1541095. Epub 2018 Nov 28.
To design an ALS clinical study in which patients are remotely recruited, screened, enrolled and then assessed via daily data collection at home by themselves or caregivers.
This observational, natural-history study included two academic medical centers, one providing overall clinical management and the other overseeing computing and web-services design and management. Both healthy and ALS subjects were recruited on the Internet via advertisement on governmental and foundation websites as well as through Facebook and Google paid advertisements. Individuals underwent screening and enrollment remotely, including signing an electronic informed consent form. Participants were then provided self-measurement equipment and instructed on their use through a series of web-based videos. The equipment included a handgrip dynamometer, spirometer with smartphone connection, electrical impedance myography device, and an activity tracker. ALS Functional Rating Scale-Revised data were also collected. Subjects were asked to collect data daily for three months and twice-weekly for the subsequent six months.
One hundred and eleven ALS patients and 30 healthy individuals enrolled in the study from across 41 states (74 men, 62 women). Baseline median ALSFRS-R score was 33. Seventy two percent of the ALS patients sent equipment and 88% of the healthy subjects sent equipment were able to complete a first set of measurements. Expected baseline differences between the ALS patients and healthy participants were identified for all measures.
It is possible to design and institute an at-home based study in ALS patients, using a number of state-of-the-art approaches, including web-based consenting and training and Internet-connected measurement devices.
设计一项 ALS 临床研究，使患者能够远程招募、筛选、入组，并通过在家中自行或由护理人员进行日常数据收集来进行评估。
这项观察性、自然史研究包括两个学术医疗中心，一个提供全面的临床管理，另一个负责监督计算和网络服务的设计和管理。通过政府和基金会网站、Facebook 和 Google 付费广告，在互联网上招募健康和 ALS 受试者。个人通过远程进行筛选和入组，包括签署电子知情同意书。然后，参与者将获得自我测量设备，并通过一系列基于网络的视频接受使用说明。设备包括握力测力计、带智能手机连接的肺活量计、电阻抗肌电图仪和活动追踪器。还收集了 ALS 功能评定量表修订版数据。要求受试者在三个月内每天收集数据，随后的六个月内每两周收集两次。
这项研究从 41 个州招募了 111 名 ALS 患者和 30 名健康个体（74 名男性，62 名女性）。基线时 ALSFRRS-R 评分中位数为 33。72%的 ALS 患者和 88%的健康受试者能够发送设备并完成第一组测量。所有测量都确定了 ALS 患者和健康参与者之间预期的基线差异。
使用多种最先进的方法，包括基于网络的同意和培训以及互联网连接的测量设备，有可能在 ALS 患者中设计和实施一项基于家庭的研究。
