BACKGROUND: Intravenous (IV) edaravone received approval from the US Food and Drug Administration based on a 33% (P=.0013) slowing of functional loss compared with placebo, as measured by the Amyotrophic Lateral Sclerosis (ALS) Functional Rating Scale-Revised. Real-world studies in people with ALS might help further evaluate effectiveness.
OBJECTIVES: To estimate time to progression milestones in IV edaravone-treated patients with ALS vs IV edaravone-naïve patients in a real-world setting.
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, insurance, cardiovascular disease history, riluzole prescription, gastrostomy tube placement, artificial nutrition, non-invasive ventilation, and all-cause hospitalization. For cases, the index date was the first IV edaravone claim date; for controls, it was the date IV edaravone was available on the market (August 2017). 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 (M4); speech-generating devices (M5); and hospice (M6). Difference between IV edaravone-treated cases and non-IV edaravone-matched controls in cause-specific restricted mean time lost (RMTL) was estimated to examine the beneﬁt of IV edaravone while considering mortality as the competing risk.
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) for 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), 0.92 (0.25-1.58), 3.37 (2.27-4.46), and 2.33 (1.31-3.34) for M1, M2, M3, M4, M5, and M6, respectively. Median treatment duration (months [interquartile range]) for cases was 9.11 (3.45, 16.08).
CONCLUSIONS: This real-world analysis describes the time to milestones in patients with ALS treated with IV edaravone. This information may be useful to payers and clinicians in evaluating IV edaravone use.