Background: Intravenous (IV) edaravone was approved by the US Food and Drug Administration (FDA) in May 2017 for the treatment of amyotrophic lateral sclerosis (ALS) based on a randomized, controlled trial conducted in Japan showing that edaravone slowed the rate of functional loss in ALS. Information on real-world outcomes in patients treated with IV edaravone is limited.
Objective: To describe treatment outcomes in patients treated with IV edaravone collected by a provider of home/alternative-site infusions since its FDA approval.
Approach: A retrospective cohort study was conducted utilizing de-identified data collected from a provider of home/alternative-site infusion and specialty pharmacy services that treat patients with ALS receiving IV edaravone across the United States. Patients with ALS receiving their first IV edaravone dose between September 25, 2017, and December 31, 2020, were selected. Variables collected included demographic information, ALS Functional Rating Scale-Revised (ALSFRS-R), forced vital capacity (FVC) scores, and treatment outcomes. All variables were assessed objectively.
Results: A total of 153 patients were included in the analysis. At the start of treatment, the mean age±SD was 61.4±10.8 years, 66.0% of the patients were male, and 26.1% were covered by commercial insurance plans. Mean ALSFRS-R and FVC scores were 34.3±9.3 and 70.7%±21.6%, respectively. As of March 31, 2021, 105 (68.6%) patients discontinued treatment. The most frequent reason for discontinuation was death or hospice in 45 patients (42.9%), followed by doctor choice in 20 patients (19.0%), patient choice in 19 patients (18.1%), and insurance or care change in 15 patients (14.3%). Of the 48 (31.4%) patients who are still on service, the mean±SD treatment duration (days) was 445.6 ±291.8.
Conclusions: This analysis described treatment outcomes in patients with ALS treated with IV edaravone since its FDA approval. This information may be useful for clinicians who prescribe IV edaravone for their patients with ALS.