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Eculizumab Use in Neuromyelitis Optica Spectrum Disorders: Routine Clinical Care Data From a European Cohort
Marius Ringelstein1, Susanna Asseyer1, Gero Lindenblatt1
1From the Department of Neurology (M.R., K.F., S.G.M., O.A.), Medical Faculty; Department of Neurology (M.R.), Center for Neurology and Neuropsychiatry, LVR-Klinikum, Heinrich-Heine-University Düsseldorf; Experimental and Clinical Research Center (S.A., F.P., J.B.-S.), a Cooperation Between the Max Delbrück Center for Molecular Medicine in the Helmholtz Association and Charité Universitätsmedizin; Charité - Universitätsmedizin Berlin (S.A., F.P., J.B.-S.), corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Experimental and Clinical Research Center; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC) (S.A., F.P., J.B.-S.), Berlin; Department of Neurology (G.L.), Johanna Etienne Hospital, Neuss; Center for Translational Neuro- and Behavioral Sciences (R.P., J.S., C.K.), University Medicine Essen, University of Duisburg-Essen; Department of Neurology (L.R., L.K.), University Hospital, Münster; Department of Neurology (K.G., A.B.), School of Medicine and Health, Technical University of Munich, Klinikum Rechts der Isar; Department of Neurology and Institute of Neuroimmunology and MS (INIMS) (V.H.), University Medical Center Hamburg-Eppendorf; Department of General Pediatrics (M.K.), Neonatology and Pediatric Cardiology, University Children's Hospital, Medical Faculty, Heinrich-Heine-University Düsseldorf; Department of Neurology (K.H., R.G., I.A.), St. Josef-Hospital, Ruhr University Bochum; Department of Neurology (C.O., K.R.), Charité-Universitätsmedizin Berlin, corporate member of Freie Universität and Humboldt-Universität zu Berlin; Center of Clinical Neuroscience (T.Z.), Department of Neurology, Dresden; Department of Neurology (A.E.)Halle; Department of Neurology (V.R., F.T.N.), Erlangen; Department of Neurology (K.A., R.L.), Regensburg; Department of Neurology (S.A.L., C.W.), University Hospital, Köln; Department of Neurology (S.J., M.K.-K., B.W.), Molecular Neuroimmunology Group, University of Heidelberg; Department of Neurology (S.W.), Gießen; Department of Neurology (M.Seipelt), Marburg; Department of Neurology (Y.Y.), Frankfurt; Department of Neurology (N.R., U.K.Z.), Rostock, Germany; Department of Neurology (P.S.R.), Medical University of Vienna, Austria; Department of Neurology (M.C.K.), University Hospital, Tübingen; Section of Translational Neuroimmunology (J.W., C.G.), Department of Neurology, Jena University Hospital; Department of Neurology (M.W.H., C.T.), Hannover Medical School; and Department of Neurology (M.Senel), University Hospital, Ulm, Germany.
Background And Objectives:
Attack prevention is crucial in managing neuromyelitis optica spectrum disorders (NMOSDs). Eculizumab (ECU), an inhibitor of the terminal complement cascade, was highly effective in preventing attacks in a phase III trial of aquaporin-4 (AQP4)-IgG seropositive(+) NMOSDs. In this article, we evaluated effectiveness and safety of ECU in routine clinical care.
Methods:
We retrospectively evaluated patients with AQP4-IgG+ NMOSD treated with ECU between December 2014 and April 2022 at 20 German and 1 Austrian university center(s) of the Neuromyelitis Optica Study Group (NEMOS) by chart review. Primary outcomes were effectiveness (assessed using annualized attack rate [AAR], MRI activity, and disability changes [Expanded Disability Status Scale {EDSS}]) and safety (including adverse events, mortality, and attacks after meningococcal vaccinations), analyzed by descriptive statistics.
Results:
Fifty-two patients (87% female, age 55.0 ± 16.3 years) received ECU for 16.2 (interquartile range [IQR] 9.6 - 21.7) months. Forty-five patients (87%) received meningococcal vaccination before starting ECU, 9 with concomitant oral prednisone and 36 without. Seven of the latter (19%) experienced attacks shortly after vaccination (median: 9 days, IQR 6-10 days). No postvaccinal attack occurred in the 9 patients vaccinated while on oral prednisone before starting ECU and in 25 (re-)vaccinated while on ECU. During ECU therapy, 88% of patients were attack-free. The median AAR decreased from 1.0 (range 0-4) in the 2 years preceding ECU to 0 (range 0-0.8; p < 0.001). The EDSS score from start to the last follow-up was stable (median 6.0), and the proportion of patients with new T2-enhancing or gadolinium-enhancing MRI lesions in the brain and spinal cord decreased. Seven patients (13%) experienced serious infections. Five patients (10%; median age 53.7 years) died on ECU treatment (1 from myocardial infarction, 1 from ileus with secondary sepsis, and 3 from systemic infection, including 1 meningococcal sepsis), 4 were older than 60 years and severely disabled at ECU treatment start (EDSS score ≥ 7). The overall discontinuation rate was 19%.
Discussion:
Eculizumab proved to be effective in preventing NMOSD attacks. An increased risk of attacks after meningococcal vaccination before ECU start and potentially fatal systemic infections during ECU-particularly in patients with comorbidities-must be considered. Further research is necessary to explore optimal timing for meningococcal vaccinations.
Classification Of Evidence:
This study provides Class IV evidence that eculizumab reduces annualized attack rates and new MRI lesions in AQP4-IgG+ patients with NMOSD.
