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

Topic:

Other

Poster Number: M208

Author(s):

Alexander V. Sherman, MS, Massachusetts General Hospital, Jeffrey Zhang, PhD, Princeton Pharmatech, LLC, Ying Liu, PhD, Princeton Pharmatech, LLC, Alex Berger, BS, Massachusetts General Hospital, Kinjal Patel, MHA, MBA, Mitsubishi Tanabe Pharma America, Inc., Malgorzata Ciepielewska, MS, Mitsubishi Tanabe Pharma America, Stephen Apple, MD, Mitsubishi Tanabe Pharma America, Inc., Ximena Arcila-Londono, MD, Henry Ford Hospital, Ghazala Hayat, MD, Washington University, Nicholas Olney, MD, Providence Brain and Spine Institute, James Wymer, MD, University of Florida, Kelly Gwathmey, MD, Department of Neurology, Virginia Commonwealth University School of Medicine, Federica Cerri, Neuromuscular Omnicentre, Christian Lunetta, MD, Istituti Clinici Scientifici Maugeri IRCCS, Neurorehabilitation Unit of Milano, Terry Heiman-Patterson, MD, Temple University, Senda Ajroud-Driss, MD, Northwestern University, David Walk, MD, University of Minnesota

Background: Intravenous (IV) edaravone was approved by the US Food and Drug Administration in 2017 for the treatment of ALS after randomized clinical trial (RCT) results showed slowing of ALS-associated physical functional decline. 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 people with ALS (PALS).

Objectives: To obtain real-world evidence on treatment patterns, clinical outcomes, and patient-reported outcomes (PROs) of IV edaravone–treated PALS in the ALS/MND NHC database. Specifically, this study aimed to: 1) assess the settings in which IV edaravone was initiated, 2) describe real-world characteristics of IV edaravone treatment patterns, 3) establish comparator cohorts of PALS receiving routine care in real-world clinical practice without edaravone, and 4) compare clinical effectiveness and PROs of PALS treated with either IV edaravone or routine care in real-world clinical practice.

Results: This is an ongoing, prospective, observational cohort study of PALS in the ALS/MND NHC database initiating IV edaravone treatment. The index (start) date for this analysis was the date of the first IV edaravone dose. For PALS with no IV edaravone prescription history, the index date was assigned as the date they entered the registry. IV edaravone–treated cases were propensity score matched 1:1 to non–IV edaravone-treated controls.

Cases (n=228) were matched to controls (n=228) on sex, age, body mass index, race; and pre-index non-invasive ventilation, artificial nutrition, riluzole use, disease duration, and hospitalization. Cases and controls were also matched on mean±standard deviation ALS Functional Rating Scale-Revised score (34.6±8.0 and 34.7±7.5, respectively) and forced vital capacity %–predicted measures (72.0%±24.2% and 72.2%±22.9%, respectively) at baseline. All variables had a standardized mean difference <0.1. Additional analyses are ongoing. Conclusions: This ongoing real-world study assesses treatment patterns, clinical outcomes, and PROs of IV edaravone–treated PALS in the ALS/MND NHC database. These data may be useful to inform choices made by clinicians, payers, and other ALS decision-makers.