Real-world prognostic role of riluzole use in ALS: a multi-center study from PRECISION-ALS
Rosario Vasta, Fouke Ombelet, Frederik Hobin, Umberto Manera, Ammar Al‐Chalabi, Alejandro Caravaca Puchades, Philippe Corcia, Miriam Galvin, Orla Hardiman, Mark Heverin, Oskar Holmdahl, Caroline Ingre, Nikita Lamaire, Christopher McDermott, Éanna Mac Domhnaill, Harry McDonough, Robert McFarlane, Mohammed Mouzouri, Sarah Opie-Martin, Mónica Povedano Panadés, Stefan Sennfält, Pamela J. Shaw, Carlos Pastor‐Vargas, Leonard H. van den Berg, Ruben P. A. van Eijk, Jan H. Veldink, Daphne N. Weemering, Philip Van Damme, Adriano Chiò
Abstract
Background Amyotrophic Lateral Sclerosis (ALS) remains an incurable disease, with limited treatment options, and riluzole is the most widely available drug. We evaluated survival in a large cohort of patients with ALS, comparing those treated with riluzole to those who were not.Methods Using data from the PRECISION-ALS database, we retrospectively analyzed patients with ALS who were treated with 100 mg of riluzole daily at the time of diagnosis. ALSFRS-R slope from onset to diagnosis (ΔFRS) was calculated. Based on the ΔFRS distribution, we defined fast progressors as patients having a ΔFRS > 1.17, intermediate progressors as those with 1.17 > ΔFRS > 0.31 and slow progressors as those with a ΔFRS < 0.31 points per month. We used Kaplan-Meier curves and Cox proportional hazards model to explore the association of riluzole use with patient survival since diagnosis.Results Out of the 5842 patients with available riluzole data, 4847 (82.9%) received riluzole. The overall survival significantly differed between patients treated and not treated with riluzole (HR 0.70, 95%CI 0.69, 0.79), independently of sex, site of onset, age at onset and diagnostic delay. Patients treated with riluzole exhibited a 7 month longer median survival than those who did not receive riluzole (17.6 months, IQR 9.7, 29.9 vs 10.7 months, IQR 4.3, 23.4; p = 2 × 10−16). The relationship between riluzole use and extended survival varied across ΔFRS strata, being only evident among fast progressors (HR = 0.50, 95% 0.40, 0.63).Conclusions Treatment with riluzole is an independent prognostic factor in ALS. The extended survival related to riluzole use was only evident among fast-progressing patients.