肌萎缩侧索硬化症功能评定量表修订版（ALSFRS-RSE）和基于 Rasch 构建的总体肌萎缩侧索硬化症残疾量表（ROADS）作为肌萎缩侧索硬化症患者的结局测量指标的纵向比较。
Longitudinal comparison of the self-entry Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-RSE) and Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) as outcome measures in people with amyotrophic lateral sclerosis.
机构信息
Neurological Clinical Research Institute and Sean M. Healey & AMG Center for ALS, Massachusetts General Hospital, Boston, Massachusetts, USA.
Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston, Massachusetts, USA.
出版信息
Muscle Nerve. 2022 Oct;66(4):495-502. doi: 10.1002/mus.27691. Epub 2022 Aug 15.
INTRODUCTION/AIMS: Improved functional outcome measures in amyotrophic lateral sclerosis (ALS) would aid ALS trial design and help hasten drug discovery. We evaluate the longitudinal performance of the Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) compared to the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised for Self-Entry (ALSFRS-RSE) as patient reported outcomes of functional status in people with ALS.
METHODS
Participants completed the ROADS and the ALSFRS-RSE questionnaires at baseline, 3-, 6-, and 12- mo using Research Electronic Data Capture as part of a prospective, longitudinal, remote, online survey study of fatigue in ALS from 9/2020 to 12/2021. The scales were compared cross-sectionally (at baseline) and longitudinally. Correlation coefficients, coefficients of variation, and descriptive statistics were assessed.
RESULTS
A total of 182 adults with ALS consented to the study. This volunteer sample was comprised of predominantly White, non-Hispanic, non-smoking participants. Consented participant survey completion was approximately 90% at baseline and greater than 40% at 12 mo. The ALSFRS-RSE and the ROADS had high, significant agreement at 3 and 6 mo by Cohen's kappa ≥71% (p < 0.001); the number of functional increases or plateaus on the two scales were not significantly different; and the coefficient of variation of functional decline was similar at the 6-month mark, though higher for the ROADS at 3 mo and lower at 12 mo.
DISCUSSION
Although the ROADS performed similarly to the ALSFRS-RSE in an observational cohort, it has psychometric advantages, such as Rasch-modeling and unidimensionality. It merits further investigation as a patient reported outcome of overall disability and efficacy outcome measure in ALS trials.
简介/目的：在肌萎缩侧索硬化症（ALS）中，改善功能结果测量将有助于 ALS 试验设计，并有助于加速药物发现。我们评估了 Rasch 构建的整体肌萎缩侧索硬化症残疾量表（ROADS）与肌萎缩侧索硬化症功能评定量表修订版（ALSFRS-RSE）作为 ALS 患者报告的功能状态结果的纵向表现。
方法
参与者在 2020 年 9 月至 2021 年 12 月期间，使用 Research Electronic Data Capture 作为前瞻性、纵向、远程、在线 ALS 疲劳研究的一部分，在基线、3、6 和 12 个月时完成 ROADS 和 ALSFRS-RSE 问卷。这两个量表进行了横断面（基线）和纵向比较。评估了相关系数、变异系数和描述性统计数据。
结果
共有 182 名成年人同意参加这项研究。这个志愿者样本主要由白人、非西班牙裔、不吸烟的参与者组成。基线时，同意参加调查的参与者的调查完成率约为 90%，12 个月时超过 40%。ALSFRS-RSE 和 ROADS 在 3 和 6 个月时具有高度显著的一致性，Cohen's kappa ≥71%（p<0.001）；两种量表的功能增加或平台数量没有显著差异；两种量表的功能下降变异系数在 6 个月时相似，但在 3 个月时 ROADS 的变异系数较高，而在 12 个月时较低。
讨论
尽管 ROADS 在观察性队列中与 ALSFRS-RSE 表现相似，但它具有 Rasch 模型和维度性等心理计量学优势。它值得进一步研究，作为 ALS 试验中患者报告的整体残疾和疗效结果测量的工具。
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