Treatment patterns and survival benefit of edaravone-treated people with amyotrophic lateral sclerosis in the ALS/MND natural history consortium
Recommended Citation
Sherman AV, Zhang J, Liu Y, Berger A, Patel K, Ciepielewska M, Arcila-Londono X, Cerri F, Ajroud-Driss S, Forsman K, Gwathmey KG, Hayat G, Olney N, Regan T, Lunetta C, Heiman-Patterson T, Wymer J, Walkand D. Treatment patterns and survival benefit of edaravone-treated people with amyotrophic lateral sclerosis in the ALS/MND natural history consortium. Amyotroph Lateral Scler Frontotemporal Degener 2024; 25:7-8.
Document Type
Conference Proceeding
Publication Date
11-7-2024
Publication Title
Amyotroph Lateral Scler Frontotemporal Degener
Keywords
edaravone, riluzole, adult, aged, amyotrophic lateral sclerosis, Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised, artificial feeding, body mass, clinical outcome, conference abstract, controlled study, drug efficacy, drug therapy, Food and Drug Administration, forced vital capacity, functional disease, human, longitudinal study, major clinical study, male, mean survival time, motor neuron disease, noninvasive ventilation, propensity score, randomized controlled trial, survival, therapy
Abstract
Background: Riluzole was US Food and Drug Administration (FDA)-approved in 1995 and is considered the standard of Platform Communications Improving Clinical Management 7 care for the treatment of people with amyotrophic lateral sclerosis (PALS) due to the 60- to 90-day survival advantage it has demonstrated. RadicavaVR (edaravone) IV (intravenous) was FDA-approved in 2017 after randomized clinical trial (RCT) results showed slowing of ALS-associated physical functional decline. Radicava ORSVR (edaravone) oral suspension was FDA approved in 2022. While RCTs are the gold standard to evaluate drug effectiveness and safety, real-world data can complement their findings. The ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC) is a clinic-based registry that captures longitudinal clinical information from PALS. Objectives: To obtain real-world evidence on treatment patterns, clinical outcomes, and survival of Mitsubishi Tanabe Pharma America (MTPA) edaravone-treated PALS in the ALS/ MND NHC database. Methods: This is a study of PALS utilizing data from the ALS/ MND NHC database initiating edaravone treatment. The index date for this analysis was the dose date of the first ALS treatment. Patients receiving edaravone ± riluzole were propensity score matched 1:1 to those receiving riluzole only. Survival (mortality) between treatment arms was estimated using the Kaplan-Meier model. Differences in restricted mean survival time (RMST) were adjusted for potential confounding. Results: Patients receiving edaravone ± riluzole (n=176) were matched to those receiving riluzole only (n=176) on sex, age, body mass index, race; and pre-index non-invasive ventilation, artificial nutrition, and disease duration; baseline mean ± SD ALS Functional Rating Scale-Revised score (39.5 ± 4.8 and 39.3 ± 4.8, respectively) and baseline forced vital capacity %-predicted (79.3%±23.5 and 79.4%±21.4%, respectively). Matched variables had a standardized mean difference 0.1. After baseline covariate adjustment, RMST analyses over 50 months suggested a survival benefit for patients receiving edaravone ± riluzole (30.5 months) vs. riluzole only (27.2 months), which is an RMST difference between groups of 3.2 months (p<0.03). Discussion: This ongoing real-world study of edaravone- treated PALS in the ALS/MND NHC database suggests an additional survival benefit of 3.2 months with edaravone ± riluzole compared to riluzole only treatment. These data may be useful to inform choices made by clinicians, payers, and other ALS decision-makers.
Volume
25
First Page
7
Last Page
8
