Longer Milestone-Free Time in Patients With Amyotrophic Lateral Sclerosis Treated With IV Edaravone vs Not Treated With IV Edaravone: Results From a US Administrative Claims Analysis (S14.007)

Abstract

<h3>Objective:</h3> To estimate time-to-progression milestones in IV edaravone–treated vs non–IV edaravone-treated patients with ALS in a real-world setting. <h3>Background:</h3> Intravenous (IV) edaravone was US Food and Drug Administration-approved in May 2017 based on a slowing of functional decline in patients with amyotrophic lateral sclerosis (ALS). Information on real-world outcomes in patients with ALS treated with IV edaravone is limited. <h3>Design/Methods:</h3> This retrospective observational analysis included patients with ALS who were continuously enrolled in Optum’s de-identified Clinformatics® Data Mart database between August 8, 2017, and September 30, 2021. Propensity score matching (1:1) identified IV edaravone–treated patients (cases) and non–IV edaravone-treated patients (controls) matched for covariates: age, race, geographic region, gender, pre-index disease duration (period between the first claim for ALS diagnosis and the first claim for IV edaravone [cases] or August 2017 [controls]), insurance, cardiovascular disease history, riluzole prescription, gastrostomy tube placement, artificial nutrition, noninvasive ventilation, and all-cause hospitalization. Milestones were defined according to the Healthcare Common Procedure Coding System and included the use of canes/walkers/wheelchairs (M1); artificial nutrition (M2); noninvasive ventilation (M3); invasive ventilation/speech-generating devices (M4); and hospice (M5). Difference between cases and controls in cause-specific restricted mean time lost (RMTL) was estimated to examine IV edaravone benefit. <h3>Results:</h3> Cases (n=360) were matched to controls (n=360); mean age in years (SD) was 62.9 (10.2) and 62.8 (10.2) in cases and controls, respectively. In the post-index period, differences in RMTL (95% CI) in months indicate longer milestone-free time in cases than controls: 2.50 (0.93–4.07), 4.30 (2.88–5.72), 2.92 (1.56–4.28), 4.12 (3.00–5.23), and 2.33 (1.31–3.34) for M1, M2, M3, M4, and M5, respectively. <h3>Conclusions:</h3> This analysis describes the time to milestones in patients with ALS treated with IV edaravone in a real-world setting. This information may be useful to payers and clinicians in evaluating use of IV edaravone. <b>Disclosure:</b> Malgorzata Ciepielewska has received personal compensation for serving as an employee of Mitsubishi Tanable Pharma America . Jeffrey Zhang has received personal compensation for serving as an employee of Princeton Pharmatech. Jeffrey Zhang has received stock or an ownership interest from CalciMedica. Ying Liu has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for MPTA. Dr. Hagan has received personal compensation for serving as an employee of Mitsubishi Tanabe Pharma America.