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Long-term safety and efficacy of Eculizumab in Aquaporin-4 IgG-positive NMOSD

Wingerchuk, D.M., Fujihara, K., Palace, J., Berthele, A., Levy, M., Kim, H.J., Nakashima, I., Oreja-Guevara, C., Wang, K.-C., Miller, L., Shang, S., Sabatella, G., Yountz, M., Pittock, S.J., Villa, A., Garcea, O., Manin, A., Melamud, L., Aguirre, F., Fernandez, V., Munoz, D.J., Amor, J., Bocchiardo, C., Obregon, C.D.D., Laffue, A., Paez, M.F., Perez, R.M., Rocchi, V., Teijeiro, L., De Virgiliis, M., Cordoba, M., Ingolotti, M., Lupinacci, A., Ballario, C., Chiesa, A., Gomez, H., Mainella, C., Lattini, H., Barnett, M., Barton, J., Beadnall, H., Garber, J., Hardy, T., Pollard, J., Trewin, B., Shuey, N., Bryson, A., French, A., Laing, J., Law, L.Y., Plummer, C., Sanders, L., Sedal, L., Winkel, A., Neal, A., Habek, M., Adamec, I., Barun, B., Crnosija, L., Gabelic, T., Pitha, J., Nytrova, P., Novakova, I., Tyblova, M., Krasulova, E., Pavlickova, J., Petersen, T., Rasmussen, P., Stilund, M., Svendsen, K., Diem, R., Platten, M., Berberich, A., Jaschonek, H., Wildemann, B., Biberacher, V.M., Brinkhoff, K., Golkowski, D., Kowarik, M., Lehmann-Horn, K., Pongratz, V., Zettl, U., Klinke, J., Loebermann, M., Meister, S., Rimmele, F., Winkelmann, A., Lau, A.Y.L., Au, L.W.C., Fan, S.Y.F., Ip, V.H.L., Ma, S.H., Ma, K.Y.K., Mok, C.T.V., Patti, F., Messina, S., Proietto, M., Filla, A., Morra, V.B., Costabile, T., Nozzolillo, A., Sacc, Sacca and Tackley, George ORCID: 2021. Long-term safety and efficacy of Eculizumab in Aquaporin-4 IgG-positive NMOSD. Annals of Neurology 89 (6) , pp. 1088-1098. 10.1002/ana.26049

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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 = 1,200 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 weeks (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 in 100 PY and 8.6 in 100 PY, respectively. Serious infection rates were 10.2 in 100 PY in eculizumab-treated patients versus 15.1 in 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 of 119 patients) 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. ANN NEUROL 2021;89:1088–1098

Item Type: Article
Date Type: Publication
Status: Published
Schools: Psychology
Cardiff University Brain Research Imaging Centre (CUBRIC)
Additional Information: This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License. G. Tackley contributed as a member of the PREVENT Study Group .
Publisher: Wiley
ISSN: 0364-5134
Date of First Compliant Deposit: 25 April 2022
Date of Acceptance: 11 February 2021
Last Modified: 10 Nov 2022 11:04

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