Safety and efficacy of edaravone compared to historical controls in patients with amyotrophic lateral sclerosis from North-Eastern Italy

To test efficacy and tolerability of edaravone in patients with amyotrophic lateral sclerosis (ALS) originating from North-Eastern Italy. We compared 3-month and 6-month changes of ALSFRS-R score, FVC value, and MRC score of 31 consecutive patients with ALS who were treated with edaravone to those of 50 historical ALS patients who were not treated with edaravone. No significant difference for any functional measures was found between the two groups at each time point as compared to baseline. In treated patients, we also observed creatinine values to significantly decrease at 3 and 6 months (p = 0.0078 and 0.030, respectively) and ALSAQ5 score to significantly increase (i.e. worse quality of life) at 3 and 6 months (p = 0.0005 and 0.0078, respectively). Yet, we observed an overall safety of the medication over the 6-month period of observation. Our retrospective study suggests no benefit of edaravone on ALS in populations of Caucasian ancestry. •We conducted a retrospective study on edaravone in a Caucasian ALS population.•No benefit on ALS progression was observed at 3 and 6 months.•Creatinine values significantly decreased in treated patients at 3 and 6 months.•Quality of life significantly worsened in treated patients at 3 and 6 months.•Edaravone was overall well tolerated.