Retrospective review of respiratory function in the Amyotrophic Lateral Sclerosis(ALS) veteran population with and without Edaravone therapy. (S54.009)

Objective: The objective of this study is to determine if there is more rapid respiratory decline in patients receiving edaravone therapy compared to patients not receiving edaravone in a veteran population. Background: The initial phase 3 study of edaravone did not show a significant difference in the decline in the ALS-FRS-R scores in treated patients compared to placebo.. However, a post-hoc analysis and a subsequent phase-3 study revealed that patients in the early stages of ALS with mild symptoms and preserved respiratory function did show a slower progression than did the full study population. The FDA approval of the drug was based on this small study in a population that was followed over a 24 week period, leaving very little time for recordable adverse events to manifest. We hypothesize that although administration of edaravone in the early stages of ALS may be beneficial, there is a more rapid respiratory decline in patients that continue to receive edaravone once respiratory function begins to decline. Design/Methods: This study will involve a restrospective review of ALS-FRS-R scores and pulmonary function tests in ALS cases seen at the Boston VAMC since 2016. Patients with definite/probable ALS will be included. Patients with tracheostomy or other identified cause for respiratory decline will be excluded. ALS-FRS-R scores and pulmonary function tests will be compared using t-test (with p Results: Preliminary review of a subset of this population show there is a more rapid decline in respiratory function in patients receiving edaravone (full dataset review is ongoing). Conclusions: Edaravone should be used with caution in ALS patients once respiratory decline begins. (Data collection and analysis is ongoing and is anticipated to be complete by time of meeting). Disclosure: Dr. Thakore-James has nothing to disclose. Dr. Salajegheh has nothing to disclose. Dr. James has nothing to disclose.