Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension

Sean J Pittock; Kazuo Fujihara; Jacqueline Palace; Achim Berthele; Ho Jin Kim; Celia Oreja-Guevara; Ichiro Nakashima; Michael Levy; Shulian Shang; Marcus Yountz; Larisa Miller; Róisín Armstrong; Dean M Wingerchuk; PREVENT Study Group

doi:10.1177/13524585211038291

Eculizumab monotherapy for NMOSD: Data from PREVENT and its open-label extension

Mult Scler. 2022 Mar;28(3):480-486. doi: 10.1177/13524585211038291. Epub 2021 Sep 9.

Authors

Affiliations

¹ Department of Neurology, Mayo Clinic, Rochester, MN, USA.
² Department of Neurology, Tohoku University Graduate School of Medicine, Sendai, Japan/Department of Multiple Sclerosis Therapeutics, Fukushima Medical University, Fukushima City, Japan/Multiple Sclerosis and Neuromyelitis Optica Center, Southern TOHOKU Research Institute for Neuroscience (STRINS), Koriyama, Japan.
³ Nuffield Department of Clinical Neurosciences, John Radcliffe Hospital, Oxford, UK.
⁴ Department of Neurology, School of Medicine, Klinikum rechts der Isar of the Technical University of Munich, Munich, Germany.
⁵ Department of Neurology, Research Institute and Hospital, National Cancer Center, Goyang, South Korea.
⁶ Department of Neurology, Hospital Clinico Universitario San Carlos, Madrid, Spain/Department of Medicine, Universidad Complutense de Madrid, Madrid, Spain/IdISSC, Madrid, Spain.
⁷ Department of Neurology, Tohoku University Graduate School of Medicine, Sendai, Japan/Division of Neurology, Tohoku Medical and Pharmaceutical University, Sendai, Japan.
⁸ Department of Neurology, Johns Hopkins University, Baltimore, MD, USA/Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA.
⁹ Alexion Pharmaceuticals, Boston, MA, USA.
¹⁰ Alexion Pharmaceuticals, Boston, MA, USA/Rallybio, New Haven, CT, USA.
¹¹ Department of Neurology, Mayo Clinic, Scottsdale, AZ, USA.

Abstract

During PREVENT (a phase 3, randomized, double-blind, placebo-controlled, time-to-event study) and its open-label extension (interim analysis), 33 adults with aquaporin-4 immunoglobulin G-positive neuromyelitis optica spectrum disorder (AQP4-IgG + NMOSD) received eculizumab monotherapy for a median of 2.8 years (range, 14 weeks-5.2 years). At 192 weeks (~4 years), 96% of these patients were free from adjudicated relapses (Kaplan-Meier analysis; 95% confidence interval, 75.7-99.4). During PREVENT, 95% (20/21) of patients receiving eculizumab monotherapy had no disability worsening. Eculizumab monotherapy provides effective long-term relapse prevention, relieving the chronic immunosuppression burden in patients with AQP4-IgG + NMOSD. ClinicalTrials.gov; PREVENT: NCT01892345; open-label extension: NCT02003144.

Keywords: Clinical trial; neuromyelitis optica (NMO).

Publication types

Clinical Trial, Phase III
Randomized Controlled Trial
Research Support, Non-U.S. Gov't

MeSH terms

Adult
Antibodies, Monoclonal, Humanized* / therapeutic use
Aquaporin 4
Autoantibodies
Humans
Immunoglobulin G
Neuromyelitis Optica* / drug therapy

Substances

Antibodies, Monoclonal, Humanized
Aquaporin 4
Autoantibodies
Immunoglobulin G
eculizumab

Associated data

ClinicalTrials.gov/NCT01892345
ClinicalTrials.gov/NCT02003144