Assessment of IV Edaravone Use in the Management of Amyotrophic Lateral Sclerosis

Background: Edaravone has been shown to slow functional degeneration of amyotrophic lateral sclerosis (ALS). The primary objective of this study was to assess ALS disease progression in veterans on IV edaravone compared with veterans who received standard of care. Methods: This retrospective case-control study was conducted at a large, academic US Department of Veteran Affairs medical center. The primary endpoint was the change in baseline Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) scores after 6 months of IV edaravone compared with standard-of-care ALS management. The secondary outcomes included change in ALSFRS-R scores, percent forced vital capacity (%FVC) and speech intelligibility stage (SIS) 3 to 24 months after initiation of therapy, duration of edaravone completed (months), time to death (months), and safety outcomes. Results: Twenty-one edaravone and 42 standard-of-care patients were evaluated. No difference was noted in ALSFRS-R at 6 months between the edaravone and standard-of-care groups ( P = .84). Additionally, no difference was noted in change from baseline %FVC, change from baseline SIS, and time to death between the 2 groups ( P > .05). No safety events were reported in either group. Conclusions: No difference was noted in the rate of ALS disease progression between patients who received IV edaravone vs standard of care.