Continued Intravenous Edaravone Treatment in Patients with Amyotrophic Lateral Sclerosis (ALS) Increases Overall Survival Compared With No Intravenous Edaravone Treatment: Results from a US Administrative Claims Database
Benjamin Brooks1, James Berry2, Malgorzata Ciepielewska3, Jeffrey Zhang4, Ying Liu4, Gustavo Suarez Zambrano3, Melissa Hagan3
1Clinical Trials Planning LLC, 2Massachusetts General Hospital, 3Mitsubishi Tanabe Pharma America, Inc., 4Princeton Pharmatech LLC
To evaluate overall survival in patients with ALS treated with intravenous (IV) edaravone compared with those who were not treated with IV edaravone.
IV edaravone received approval from the US Food and Drug Administration in May 2017 based on a 33% (P=.0013) slowing of functional loss, as measured by the ALS Functional Rating Scale-Revised compared with placebo at 24 weeks. 
The analysis included patients with ALS who initiated treatment with IV edaravone between 08/08/2017, and 03/31/2020, and enrolled in Optum’s de-identified Clinformatics® Data Mart database. Propensity score matching (1:1) identified IV edaravone–treated patients (cases) and non–edaravone-treated patients (controls) matched for covariates potentially affecting survival: age, race, geographic region, gender, pre-index disease duration (defined as the period between the date of first claim for ALS diagnosis and the first claim for IV edaravone), insurance, history of cardiovascular disease, riluzole prescription, gastrostomy tube placement, artificial nutrition, non-invasive ventilation, and all-cause hospitalization. For cases, the index date was the date of the first claim for IV edaravone. For controls, the index date was the date IV edaravone was available on the market (August 2017). Shared frailty Cox regression analysis was performed to estimate the beneļ¬t of IV edaravone. 
In total, 318 cases were matched to 318 controls. In both groups, 208 patients (65.4%) had a history of riluzole prescription. As of 03/31/2021, there were 155 deaths (48.7%) among the cases and 196 among the controls (61.6%). Median overall survival was 29.5 months and 23.5 months, respectively, and the risk of death was 27% lower among cases vs controls (HR: 0.73; 95% CI: 0.59-0.91; P=0.005).
This real-world analysis demonstrated that continued IV edaravone treatment, in a large predominantly riluzole-treated US cohort, is associated with improved overall survival compared with not using IV edaravone.