Real-world safety and tolerability of intravenous edaravone in patients with amyotrophic lateral sclerosis

This retrospective cohort study describes real-world safety and tolerability outcomes in United States-based edaravone-treated patients with ALS. Amerita Specialty Infusion Services provided IV edaravone to patients with ALS treated with their first dose between 25 September 2017-30 September 2022. Mean ALS Functional Rating Scale-Revised (ALSFRS-R) scores and forced vital capacity (FVC) %–predicted measures were recorded within ± 100 days from care initiation to approximate baseline values. Included patients (<i>n</i> = 243) received/were still receiving IV edaravone/edaravone oral suspension as of 30 September 2022. At initiation, 66.7% were male, mean age ± SD was 61.2 ± 11.2 years, and 61.3% were covered by government insurance. In patients with provider–recorded ALSFRS-R (<i>n</i> = 115) and FVC (<i>n</i> = 84) %–predicted measures within ± 100 days from care initiation, mean ± SD values were 35.1 ± 8.9 and 72.3% ± 21.7%, respectively. Mean ± SD therapy duration was 13.5 ± 11.4 months. Discontinuation reasons included death/hospice (<i>n</i> = 82), patient’s choice (<i>n</i> = 38), doctor’s choice (<i>n</i> = 31), insurance (<i>n</i> = 18), and other (<i>n</i> = 12). Reasons for IV edaravone discontinuation and IV edaravone administration access device were not associated. Treatment discontinuation was primarily related to ALS disease progression/death, rather than safety or tolerability. This study representative of real-world patients with ALS suggests that edaravone showed consistent safety and tolerability profiles with previous studies.