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

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.

• Authors: 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
• Journal: Multiple sclerosis (Houndmills, Basingstoke, England) (Mult Scler) Vol. 28 Issue 3 Pg. 480-486 (03 2022) ISSN: 1477-0970 [Electronic] England
• PMID: 34498507 (Publication Type: Clinical Trial, Phase III, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Antibodies, Monoclonal, Humanized
• Aquaporin 4
• Autoantibodies
• Immunoglobulin G
• eculizumab

Topics

• Adult
• Antibodies, Monoclonal, Humanized (therapeutic use)
• Aquaporin 4
• Autoantibodies
• Humans
• Immunoglobulin G
• Neuromyelitis Optica (drug therapy)