The randomized open clinical trial on a novel free radical scavenger edaravone in amyotrophic lateral sclerosis

Objective To observe the efficacy and safety of a novel free radical scavenger, edaravone, in the treatment of amyotrophic lateral sclerosis (ALS). Methods Fifty patients with amyotrophic lateral sclerosis were randomly divided and assigned to edaravone group and control group. The control group was given a routine treatment of ALS, and the edaravone group was treated with edaravone (30 mg/d, iv) in addition to the routine treatment for 14 days. ALS-functional rating scale (ALS-FRS) in the two groups were assessed before treatment and 3, 6, 9, 12, 15 and 18 months after treatment. The adverse reaction associated with the edaravone treatment was also observed. Results The absolute changes of ALS-FRS score in two groups increased after treatment, and the differences within each group were significant (P 0.05), but the changes of ALS-FRS score between two groups were not significant (P = 0.081). The survival analysis showed that median survival time was 14.80 months in edaravone group and 15.60 months in control group. The cumulative survival rate at 3, 6, 9, 12, 15 and 18 months was 96%, 96%, 80%, 76%, 60% and 60% respectively in edaravone group, and 92%, 88%, 84%, 80%, 80%, and 80% respectively in control group. Log-rank test showed that there were no significant differences between two groups (P = 0.178). No obvious adverse reactions were found in edaravone treatment. Conclusion Edaravone is safe in the treatment of amyotrophic lateral sclerosis. Further observation should be done by increasing sample or dosage to verify the efficacy.