(081) Real-World Survival Effectiveness of Edaravone in Amyotrophic Lateral Sclerosis: A Propensity Score Weighted, Registry-based, Canada-wide Cohort Study
Executive Director, Canadian Neuromuscular Disease Registry Department of Clinical Neurosciences, Hotchkiss Brain Institute, University of Calgary, Canada
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease without a cure and limited treatment options. Edaravone, a free radical scavenger, was shown to slow disease progression in a select group of patients with ALS over a 6-month period; however, the effect on survival was not investigated in randomized trials.
Objectives: To describe real-world data on survival effectiveness after at least 6 months of treatment with edaravone.
Methods: This retrospective cohort study included patients with ALS across Canada with symptom onset up to three years. The interventional arm included patients who had been exposed to edaravone for a minimum of 6 months between 2017 and 2022. The control arm included patients that had no exposure to edaravone and were matched according to disease duration, ALSFRS-R score, forced vital capacity (% predicted), symptoms onset, age, gender, intervention use, and comorbidities. For each edaravone user, there are approximately three matched controls. The primary outcome of tracheostomy-free survival was compared between the exposed and the controls using Kaplan Meier curves, which used inverse propensity weights to account for the differences in age, sex, ALS-disease progression rate, disease duration, pulmonary vital capacity, bulbar ALS-onset, and presence of frontotemporal dementia or C9ORF72 mutation between the two groups.
Results: 182 patients were enrolled in the edaravone arm, and 860 patients in the control arm. Mean ages ± SD were 60 ± 11 and 63 ± 12 years, respectively. Mean ± SD time from onset to edaravone initiation was 18 ± 10 months. Tracheostomy-free survival will be calculated.
Conclusions: This study will provide evidence for edaravone effectiveness on tracheostomy-free survival in patients with ALS. These findings will help inform decisions regarding the risks and benefits of this novel therapy for patients with ALS.
