BACKGROUND: Amyotrophic lateral sclerosis (ALS) is an incurable and progressive neurodegenerative disease. Historically, ALS pharmacotherapy has been limited. Patient options for and access to treatment have improved over time with the availability of generic riluzole in 2013 and intravenous (IV) edaravone in 2017.
OBJECTIVES: To understand the demographic and clinical characteristics of patients with ALS in the United States (US) with and without IV edaravone treatment.
METHODS: This retrospective, observational study used a US administrative claims database to identify patients with ALS with and without IV edaravone treatment between August 8, 2017 (first date of US market availability) and March 31, 2022. Baseline characteristics over a 6-month period were compared between patients who did and did not receive IV edaravone treatment.
RESULTS: This study identified 208 IV edaravone users and 1,021 non-IV edaravone users. Demographic characteristics, including age, sex, and region of residence, were similar between groups. IV edaravone users had a shorter mean pre-index disease duration than non-IV edaravone users (0.1 months vs 8.5 months, p<0.01) and a higher proportion of historical riluzole use (77% vs 30%, p<0.01). IV edaravone users were equally likely to have experienced baseline respiratory symptoms when compared to non-IV edaravone users but were more likely to have experienced nerve symptoms (23% vs 16%, p<0.05), limb symptoms (60% vs 48%, p<0.01), and bulbar symptoms (37% vs 27%, p<0.01). Except for invasive ventilation (1% vs 6%, p<0.01), IV edaravone users were at similar stages of disease progression as non-IV edaravone users with mobility aids, noninvasive ventilation, invasive ventilation, speech-generating devices, and hospice care serving as proxies for progression. CONCLUSIONS: In this real-world analysis, IV edaravone users appeared to be more symptomatic than non-IV edaravone users during the baseline period. Considering the shorter pre-index disease duration and the higher proportion of historical riluzole use among IV edaravone users, these findings suggest that patients with ALS experiencing more symptoms are managed earlier and more aggressively.