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
Malgorzata Ciepielewska1, Jeffrey Zhang2, Ying Liu2, Melissa Hagan1
1Mitsubishi Tanabe Pharma America, Inc., 2Princeton Pharmatech LLC
Objective:
To estimate time-to-progression milestones in IV edaravone–treated vs non–IV edaravone-treated patients with ALS in a real-world setting.
Background:
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.
Design/Methods:
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.
Results:
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.
Conclusions:
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.
Acknowledgements: p-value communications provided editorial support.