Submitted by: Raymond Rosales, MD, PhD
Edited by: Marcus Pai, MD, PhD
Witzel S, Maier A, Steinbach R, et al. Safety and effectiveness of long-term intravenous administration of edaravone for treatment of patients with amyotrophic lateral sclerosis. JAMA Neurol
. 2022;79(2):121-130. [published correction appears in JAMA Neurol. 2022 Jun 13;:null]. doi:10.1001/jamaneurol.2021.4893
This study evaluated the long-term safety and effectiveness of intravenous edaravone therapy for patients with amyotrophic lateral sclerosis (ALS) in a real-world clinical setting. A multicenter, propensity score–matched cohort study was conducted between June 2017 and March 2020 at 12 academic ALS referral centers associated with the German Motor Neuron Disease Network.
Final analyses included 324 patients with ALS comprising 194 patients who started intravenous edaravone treatment (141 received ≥4 consecutive treatment cycles; 130 matched). Disease progression among 116 patients treated for a median of 13.9 months (IQR, 8.9-13.9 months) with edaravone did not differ from 116 patients treated for a median of 11.2 months (IQR, 6.4-20.0 months) with standard therapy (ALSFRS-R points/month, −0.91 [95% CI, −0.69 to −1.07] vs −0.85 [95% CI, −0.66 to −0.99]; P
No significant differences were observed in the secondary end points of survival probability, time to ventilation, and change in disease progression.
Long-term intravenous edaravone therapy for patients with ALS was feasible and mainly well tolerated. It was not associated with any disease-modifying benefit. Intravenous edaravone may not provide a clinically relevant additional benefit compared with standard therapy alone.
Compared to the short-term Japanese ALS study (MCI186-ALS19), subgroup analysis showing benefit in survival, this present long-term German longitudinal ALS study did not show advantage, nor added benefit compared to Riluzole. This long term study was however was generally well tolerated with few adverse events (16%).
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Vu M, Tortorice K, Zacher J, et al. Assessment of use and safety of edaravone for amyotrophic lateral sclerosis in the veterans affairs health care system. JAMA Netw Open
. 2020;3(10):e2014645. Published 2020 Oct 1. doi:10.1001/jamanetworkopen.2020.14645