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

• Type of Material: Article
• Authors: Stpehen A Johnson, Massachusetts General Hospital; Katherine M Burke, Massachusetts General Hospital; Zoe A Scheier, Massachusetts General Hospital; Mackenzie A Keegan, Massachusetts General Hospital; Alison P Clark, Massachusetts General Hospital; James Chan, Harvard Medical School; Christina Fournier, Emory University; James D Berry, Massachusetts General Hospital
• Language: English
• Date: 2022-08-15
• Publisher: WILEY
• Publication Version: Final Published Version
• Copyright Statement: © 2022 The Authors. Muscle & Nerve published by Wiley Periodicals LLC.
• License: Creative Commons Attribution-NonCommercial-NoDerivs 4.0 International
• Final Published Version (URL): http://dx.doi.org/10.1002/mus.27691
• Title of Journal or Parent Work: MUSCLE & NERVE
• Volume: 66
• Issue: 4
• Start Page: 495
• End Page: 502
• Grant/Funding Information: U.S. Department of Veterans Affairs, Grant/Award Number: IK2CX001595‐02
• Keywords: ALSFRS-RSE; amyotrophic lateral sclerosis; Science & Technology; Life Sciences & Biomedicine; Clinical Neurology; Neurosciences; ALSFRS-R; ROADS; Neurosciences & Neurology; DESIGN; outcome measure
• Research Categories: Health Sciences, Medicine and Surgery; Biology, Biostatistics

Abstract

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.