Experience of Using Edaravone in a US-based ALS Center
Anem Kohli1, Sandeep Rana1
1Allegheny Health Network, Allegheny General Hospital
Objective:
We elected to study the effect of edaravone in a US-based population since the original study data were derived from a Japanese patient population.
Background:
Edaravone clinical trials were conducted in Japan in a placebo controlled study of ALS patients in the early stages of disease and shown to be effective in slowing disease progression over a 6-month period. Based on these results, the US FDA approved the drug for clinical use in the summer of 2017.
Design/Methods:

We retrospectively studied the data of all patients that were started on edaravone at our institution after it became available in the US. We collected the data on 16 consecutive patients.

Results:
The average age of patients in the study was 58.8 years; 81% were male, 75% were limb onset, and 25% were bulbar onset. There were two African Americans; the remained of patients were Caucasian.  All were in the early stages of disease, i.e., scoring over 2 in all categories of the ALS FRS. Of the 16 patients, 4 discontinued edaravone, the remainder were on the medication for the entire 6 months.  Overall, the patients tolerated the medication well and none discontinued its use due to adverse effects. For those patients completing the study, the mean decrease of the ALS FRS score over the 6-month period was 6; the mean decrease of FVC was 13.4%.
Conclusions:
Edaravone is well tolerated in a regional US-based population, with only mild side adverse having been reported. The efficacy of the medication in this small sample appears reasonably comparable to the results from the original Japanese study. We would recommend that edaravone be offered to ALS patients in the early stages of disease and that US clinics continue to monitor and analyze outcomes data, as therapeutic responses across diverse populations of patients can be difficult to predict.