JAMA sub-journal: Stroke drug edaravone in the treatment of "ALS" patients is safe and reliable, but its efficacy is questionable

Previous studies have shown that edaravone has a good effect on patients with early amyotrophic lateral sclerosis (ALS) and can effectively slow down the progression of early amyotrophic lateral sclerosis
.
Therefore, intravenous edaravone is approved as a disease-modifying drug in ALS patients, but evidence of efficacy is limited to the short-term beneficial effects shown in the MCI186-ALS19 study

A multicenter, propensity score-matched cohort study was conducted between June 2017 and March 2020 at 12 ALS academic referral centers associated with the German Motor Neurone Disease Network
.
Of the 1440 patients screened, 738 were included in propensity score matching

Patient characteristics and system safety were assessed in patients who received at least 1 dose of intravenous edaravone
.
The effect of edaravone was assessed in patients who received at least 4 cycles of treatment, matching the propensity score of ALS patients who received standard care alone

RESULTS: A total of 194 patients started intravenous edaravone; 125 (64%) were male, with a median age of 57.
5 years (IQR, 50.
7-63.
8 years)
.
The 116 edaravone-treated patients had no disease progression over a median duration of 13.

During the follow-up period, the difference in ALSSFRS-R score between the two groups

No significant differences were observed in secondary endpoints of survival probability, ventilation time, and change in disease progression

references:

Safety and Effectiveness of Long-term Intravenous Administration of Edaravone for Treatment of Patients With Amyotrophic Lateral Sclerosis.