Abstract

<h3>Objective:</h3> To describe real-world treatment outcomes in patients with ALS treated with IV edaravone collected by a provider of home/alternative-site infusions. <h3>Background:</h3> Intravenous (IV) edaravone was US Food and Drug Administration approved in May 2017 after it was shown to slow the rate of physical functional decline in patients with amyotrophic lateral sclerosis (ALS). Information on real-world use of IV edaravone is limited. <h3>Design/Methods:</h3> A retrospective cohort study was conducted utilizing de-identified data collected from Amerita Specialty Infusion Services, a US-based provider of home/alternative-site infusions that has treated and collected data from patients with ALS receiving IV edaravone since the time of commercial availability. Patients with ALS who received their first IV edaravone dose between September 25, 2017, and December 31, 2020, were included. Patient status was confirmed through March 31, 2021. Variables collected included patient demographics, revised ALS Functional Rating Scale (ALSFRS-R) and forced vital capacity (FVC) scores, and treatment outcomes. <h3>Results:</h3> Patients (N=153) receiving IV edaravone were included in this analysis; 66.0% were male, mean±SD age was 61.4±10.8 years, and 27.5% were covered by commercial insurance. In patients who had healthcare provider-recorded ALSFRS-R (n=61) and FVC (n=38) assessed ±100 days from start of care, mean±SD scores were 33.7±9.3 and 66%±24%, respectively. At the end of the study period, 105 (68.6%) patients had discontinued treatment. The most frequent reason for discontinuation was death/hospice (42.9%), doctor choice (19.0%), patient choice (18.1%), and insurance/care change (14.3%). Mean±SD treatment duration for patients who discontinued was 314±260 days. As of March 31, 2021, 48 (31.4%) patients were still receiving treatment with IV edaravone and their mean±SD treatment duration was 446±292 days. <h3>Conclusions:</h3> This analysis describes treatment outcomes in IV edaravone-treated patients with ALS in a real-world setting. This information may be useful to clinicians who prescribe IV edaravone for patients with ALS. <b>Disclosure:</b> Mrs. Da Silva has nothing to disclose. Malgorzata Ciepielewska has received personal compensation for serving as an employee of Mitsubishi Tanable Pharma America . Mr. Do has nothing to disclose. Miss Anderson has nothing to disclose. Mrs. Martin has received personal compensation for serving as an employee of Amerita Specialty Infusion. Dr. Ortlip has nothing to disclose.

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