Long-term Safety and Efficacy of Eculizumab in Aquaporin-4 IgG-Positive NMOSD

Dean M Wingerchuk, Kazuo Fujihara, Jacqueline Palace, Achim Berthele, Michael Levy, Ho Jin Kim, Ichiro Nakashima, Celia Oreja-Guevara, Kai-Chen Wang, Larisa Miller, Shulian Shang, Guido Sabatella, Marcus Yountz, Sean J Pittock
Annals of Neurology 2021 February 14

OBJECTIVE: During PREVENT (NCT01892345), eculizumab significantly reduced relapse risk versus placebo in patients with aquaporin-4 immunoglobulin G-positive neuromyelitis optica spectrum disorder (AQP4-IgG+ NMOSD). We report an interim analysis of PREVENT's ongoing open-label extension (OLE; NCT02003144) evaluating eculizumab's long-term safety and efficacy.

METHODS: Patients who completed PREVENT could enroll in the OLE to receive eculizumab (maintenance dose, 1200 mg/2 weeks, after a blinded induction phase). Safety and efficacy data from PREVENT and its OLE (interim data cut, July 31, 2019) were combined for this analysis.

RESULTS: Across PREVENT and the OLE, 137 patients received eculizumab and were monitored for a median (range) of 133.3 (5.1-276.9) weeks, for a combined total of 362.3 patient-years (PY). Treatment-related adverse event (AE) and serious adverse event (SAE) rates were 183.5/100 PY and 8.6/100 PY, respectively. Serious infection rates were 10.2/100 PY in eculizumab-treated patients versus 15.1/100 PY in the PREVENT placebo group. No patient developed a meningococcal infection. At 192 weeks (3.7 years), 94.4% (95% confidence interval [CI], 88.6-97.3) of patients remained adjudicated relapse-free. The adjudicated annualized relapse rate was 0.025 (95% CI, 0.013-0.048) in all eculizumab-treated patients versus 0.350 (95% CI, 0.199-0.616) in the PREVENT placebo group. During the OLE, 37% of patients (44/119) stopped or decreased background immunosuppressive therapy use.

INTERPRETATION: This analysis demonstrates that eculizumab's long-term safety profile in NMOSD is consistent with its established profile across other indications. This analysis also demonstrated the sustained ability of long-term eculizumab treatment to reduce relapse risk in patients with AQP4-IgG+ NMOSD. This article is protected by copyright. All rights reserved.

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