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.

Pathomechanisms in demyelination and astrocytopathy: autoantibodies to AQP4, MOG, GFAP, GRP78 and beyond

“…Eculizumab, satralizumab and inebilizumab are now authorized for the treatment of AQP4-IgG positive NMOSD in several countries. Open-label extension data from the studies of all three approved therapies demonstrated sustained reduction in relapse risk [30][31][32][33].…”

Eculizumab‐related drug reaction in a patient with neuromyelitis optica

“… 1 Eculizumab was FDA‐approved in 2020 for the treatment of neuromyelitis optica (NMO) after it was shown to be effective in reducing relapse frequency in highly clinically active, aquaporin‐4 immunoglobulin G (AQP4‐IgG)‐positive NMO. 2 Commonly reported side effects (>10%) include upper respiratory infections and headache. A life‐threatening desquamating rash and hyperammonemia following the administration of eculizumab for paroxysmal nocturnal hemoglobinuria (PNH) has been reported.…”

“…Eculizumab is a fully humanized monoclonal blocking antibody to complement protein C5 that inhibits cleavage to C5a and C5b, thus preventing terminal complement complex C5b‐9 and formation of the membrane attack complex 1 . Eculizumab was FDA‐approved in 2020 for the treatment of neuromyelitis optica (NMO) after it was shown to be effective in reducing relapse frequency in highly clinically active, aquaporin‐4 immunoglobulin G (AQP4‐IgG)‐positive NMO 2 . Commonly reported side effects (>10%) include upper respiratory infections and headache.…”

Eculizumab Related Drug Reaction in a Patient with Neuromyelitis Optica

“…1 Eculizumab was FDA approved in 2020 for the treatment of neuromyelitis optica (NMO) after it was shown to be effective in reducing relapse frequency in highly clinically active, aquaporin-4 immunoglobulin G (AQP4-IgG)-positive NMO. 2 Commonly reported side effects (>10%) include upper respiratory infections and headache. A life-threatening desquamating rash and hyperammonemia following administration of eculizumab for paroxysmal nocturnal hemoglobinuria (PNH) has been reported.…”