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

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.

Listener effort quantifies clinically meaningful progression of dysarthria in people living with amyotrophic lateral sclerosis

“…Recent data has demonstrated that the ALSFRS-R Self-Entry (ALSFRS-RSE) is very highly correlated with the ALSFRS-R at baseline (though ALSFRS-RSE is generally 1-3 points higher) and has similar slope of decline of the slope to the ALSFRS-R (4, 5, 43). The adoption of ALSFRS-RSE has allowed fully remote studies to follow the functional progression of people with ALS without the need for trained examiners, thus permitting more frequent sampling and thus smaller standard deviation on the slope of decline compared to traditional ALSFRS-R recording in clinic (44).…”

Wearable device and smartphone data quantify ALS progression and may provide novel outcome measures

“…[12,47] 32 [11,45] 33 [11,47] ALSFRS-R baseline staff-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised, n number, min minimum, max maximum, SD standard deviation. Days in observation period 178 [23,191] 180 [61,189] 179 [23,191] Valid days in observation period* 158 [21,191] 136 [16,183] 146 [16,191] Average number of valid hours on a valid day* 21 [15,24] 12 [10,17] 16 [10,24] ALSFRS-RSE smartphone self-entry Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised, ROADS Rasch-built Overall ALS Disability Scale; valid hours/day --hours/day passing the conditions outlined in Methods; * -different methods used to define valid hour/day between the ActiGraph and Modus groups (see Methods section).…”

“…Other scales are being developed using modern test theory techniques and for self-entry. One such example is the Rasch-built Overall ALS Disability Scale (ROADS) 10,17 . Prior studies have demonstrated the feasibility of both in-clinic and non-clinic ALS PROM collection 15,18 .…”

Wearable device and smartphone data can track ALS disease progression and may serve as novel clinical trial outcome measures

“…Compliance statistics are summarized in Table 2 and were robust for both groups. Days in observation period 178 [23,191] 180 [61,189] 179 [23,191] Valid days in observation period* 158 [21,191] 136 [16,183] 146 [16,191] Average number of valid hours on a valid day* 21 [15,24] 12 [10,17] 16 [10,24] ALSFRS-RSE --smartphone self-entry Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised; ROADS -Rasch-built Overall ALS Disability Scale; valid hour/day --hour/day passing the conditions outlined in Methods section; * --different methods used to de ne valid hour/day across the ActiGraph and Modus groups (see Methods section).…”

“…One such example is the Rasch-built Overall ALS Disability Scale (ROADS). 10,17 Prior studies have demonstrated the feasibility of both in-clinic and non-clinic ALS PROM collection. 15,18 Remote, passive data collection offers another avenue for outcome measure re nement without the need for participants' concentration or effort.…”