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, the PREVENT Study Group
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.