The Rasch-Built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS) has certain benefits over the Amyotrophic Lateral Sclerosis Functional Rating Scale Revised for Self-Entry (ALSFRS-RSE) instrument for evaluating patients with amyotrophic lateral sclerosis (ALS). These are the findings of a study published in the journal Muscle & Nerve.
Assessing the efficacy of potential therapies in clinical trials relies on accurate quantification of changes of interest among study participants. For ALS, the standard tool for evaluating functional change is the ALSFRS-R which has some limitations, such as not meeting the criteria for unidimensionality.
Researchers conducted a single-center, longitudinal, voluntary, open enrollment study at Mass General Brigham between 2020 and 2021 to assess potential tools to evaluate functional status in ALS. Patients (N=176) with ALS were assessed at baseline and 3, 6, and 12 months using the ROADS and ALSFRS-RSE instruments.
The study population comprised 54.0% men, aged mean 61.6 (standard deviation [SD], 10.0) years, 92.0% were White, symptoms had onset between 3.34 and 275 months previously, 68.8% used an assistive device, and 47.7% used non-invasive ventilation.
Completion of surveys declined over time from 95% and 85% at baseline to 73% and 67% at 3, 54% and 51% at 6, and 48% and 43% at 12 months for the ALSFRS-RSE and ROADS instruments, respectively. At all visits, ALSFRS-RSE took longer to complete (range, 3.9-4.9 min) than ROADS (range, 3.4-3.8 min).
Among the participants who did not complete the 12-month survey, 19% had died. The change in ALSFRS-RSE (hazard ratio [HR], 3.5; P <.001) and ROADS (HR, 1.9; P <.001) scores predicted mortality at 12 months.
Similarly, patients who completed the survey at 12 months had higher ALSFRS-RSE (mean, 31 vs 29) and ROADS (mean, 80 vs 77) scores at baseline, respectively.
The scores of the 2 scales were correlated at baseline (r, 0.90; P <.001) and agreement was high at 3 (71%) and 6 months (74%).
This study may have been limited by the lack of diversity among the study cohort.
The researchers concluded, “Accessible, reliable, and facile metrics, responsive to disease progression, are fundamental to discovery of efficacious ALS therapeutics.”
The ROADS performs at least as well as ALSFRS-RSE, according to the researchers. “Whether it becomes a primary outcome measure in ALS clinical trials first requires treatment trial incorporation to assess performance.”
Disclosure: Multiple authors declared affiliations with industry. Please refer to the original article for a full list of disclosures.
Johnson SA, Burke KM, Scheier ZA, et al. 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. Muscle Nerve. Published online July 29, 2022. doi:10.1002/mus.27691