Survival Benefits and Treatment Patterns of Edaravone–Treated People With Amyotrophic Lateral Sclerosis in the ALS/MND Natural History Consortium

Topic:

Other

Poster Number: P293

Author(s):

Alexander Sherman, MS, Massachusetts General Hospital, Boston, MA, USA, Jeffrey Zhang, PhD, Princeton Pharmatech, LLC, Princeton, NJ, Ying Liu, PhD, Princeton Pharmatech, LLC, Princeton, NJ, Alex Berger, BS, Massachusetts General Hospital, Boston, MA, USA, Kinjal Patel, MHA, MBA, Mitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA, Malgorzata Ciepielewska, Mitsubishi Tanabe Pharma America, Inc., Jersey City, NJ, Ximena Arcila-Londono, MD, Henry Ford Health, Detroit MI, USA, Federica Cerri, Neuromuscular Omnicentre, Milano, Italy, Senda Ajroud-Driss, MD, Northwestern university, Kelly G. Gwathmey, MD, Department of Neurology, Virginia Commonwealth University, Richmond, VA, USA, Ghazala Hayat, MD, St. Louis University, St Louis, MO, USA, Nicholas Olney, MD, Providence Brain and Spine Institute, Portland, OR, USA, Tyler Regan, Providence Brain and Spine Institute, Portland, OR, USA, Christian Lunetta, MD, Istituti Clinici Scientifici Maugeri IRCCS, Neurorehabilitation Unit of Milano, Milan, Italy, Terry Heiman-Patterson, MD, Temple University, Philadelphia, PA, USA, JAMES WYMER, Md, University of Florida, David Walk, MD, University of Minnesota, Minneapolis, MN, USA, Stephen Apple, MD, Mitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA

BACKGROUND: Currently, there are 3 United States (US) Food and Drug Administration (FDA)-approved treatments for amyotrophic lateral sclerosis (ALS), including riluzole, Mitsubishi Tanabe Pharma America (MTPA) intravenous (IV) and oral edaravone (Radicava® IV and Radicava ORS® oral suspension), and tofersen (for patients with superoxide dismutase 1 mutations). While clinical trials are the gold standard for analyzing a drug’s effectiveness, 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 people with ALS (PALS).

OBJECTIVES: To obtain real-world evidence on survival, treatment patterns and clinical outcomes, of MTPA edaravone–treated PALS in the ALS/MND NHC database.

RESULTS: The ALS/MND NHC database was used to investigate PALS initiating MTPA edaravone treatment, with an index date of the dose date of the first ALS treatment. Patients receiving MTPA edaravone ± riluzole were propensity score matched 1:1 to those receiving riluzole only. Survival (mortality) was estimated using the Kaplan-Meier model. Differences in restricted mean survival time (RMST) were adjusted for potential confounding. Patients receiving MTPA edaravone ± riluzole (n=169) were matched to those receiving riluzole only (n=169) on sex, age, body mass index, and race; pre-index non-invasive ventilation, artificial nutrition, and disease duration; baseline mean±SD ALS Functional Rating Scale-Revised score (37.7±6.3 and 37.6±5.9, respectively) and baseline forced vital capacity %–predicted (78.3%±24.0% and 78.3%±22.5%, respectively). Matched variables had a standardized mean difference ≤0.1. After baseline covariate adjustment, RMST analyses over 48 months suggested a survival benefit for patients receiving MTPA edaravone ± riluzole (29.3 months) vs riluzole only (26.5 months), which is an RMST difference between groups of 3.2 months (P<0.03). CONCLUSION: This real-world study of MTPA edaravone–treated PALS in the ALS/MND NHC database suggests an additional survival benefit of 3.2 months with MTPA edaravone ± riluzole vs riluzole only treatment. Sponsorship: This study was sponsored by Mitsubishi Tanabe Pharma America, Inc.