Real-world multicentre cohort study on choices and effectiveness of immunotherapies in NMOSD and MOGAD.

Hayrettin Tumani, Tania Kümpfel, Joachim Havla, Jan-Patrick Stellmann, Corinna Trebst, Brigitte Wildemann, Martina Flaskamp, Achim Berthele, Florian Then Bergh, Ankelien Duchow, Judith Bellmann-Strobl, Tim Friede, Katrin Giglhuber, Friedemann Paul, Gero Lindenblatt, Katinka Fischer, Lisa Revie, Daniel Engels, Matthias Kaste, Jörn Peter Sieb, Hanna Pellkofer, Insa Schiffmann, Mariella Herfurth, Makbule Senel, Daria Tkachenko, Thivya Pakeerathan, Mosche Pompsch, Carolin Schwake, Matthias Grothe, Annette Walter, Jonathan Wickel, Paulus Stefan Rommer, Patrick Schindler, Martin W Hümmert, Eva Dawin, Franziska Bütow, Luisa Klotz, Markus Krämer, Ilya Ayzenberg, Marius Ringelstein, Klemens Angstwurm, Sven Jarius, Orhan Aktas, Vivien Häußler, Ingo Kleiter, Mirjam Korporal-Kuhnke

Journal: Journal of neurology, neurosurgery, and psychiatry 2025;96(6):582-592

PMID: 39730197

Abstract

BACKGROUND

Recurrent attacks in neuromyelitis optica spectrum disorders (NMOSDs) or myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) can lead to severe disability. We aimed to analyse the real-world use of immunotherapies in patients with NMOSD and MOGAD, focusing on changes in treatment strategies, effects on attack rates (ARR) and risk factors for attacks.

METHODS

This longitudinal registry-based cohort study included 493 patients (320 with aquaporin-4 immunoglobulin G (AQP4-IgG) seropositive NMOSD (65%), 44 with AQP4-IgG seronegative NMOSD (9%) and 129 MOGAD (26%)) with 1247 treatments from 19 German and one Austrian centre from the registry of the neuromyelitis optica study group (NEMOS). We analysed unadjusted ARR and implemented survival analyses and Cox proportional hazard regression to assess efficiency and risk factors for subsequent attacks over time.

RESULTS

Rituximab and azathioprine are the most widely used immunotherapies in NMOSD as well as in MOGAD, with changes in distribution over the last decade. Immunotherapy demonstrated significant therapeutic effects in NMOSD but less pronounced effects in MOGAD. Risk factors for attacks included younger age and prior attacks under the same therapy. Efficacy varied among the different immunotherapies, with azathioprine, rituximab and eculizumab showing significant risk reductions in AQP4-IgG seropositive NMOSD.

CONCLUSIONS

This study provides insights into the evolving treatment landscape and effectiveness of immunotherapies in NMOSD and MOGAD. Established off-label therapies continue to play an important role, especially for patients with stable disease, with emerging evidence supporting newly approved therapies. Future studies are needed to refine treatment algorithms and address the ongoing uncertainties in MOGAD management.

© Author(s) (or their employer(s)) 2025. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ Group.

Address: Department of Neurology and Institute of Neuroimmunology and MS (INIMS), University Medical Center Hamburg-Eppendorf, Hamburg, Germany [email protected].; Department of Neurology, Hannover Medical School, Hannover, Germany.; Institute of Clinical Neuroimmunology, LMU Hospital, Ludwig-Maximilians University Munich, Munich, Germany.; Neuroscience Clinical Research Center, Charité, Universitätsmedizin Berlin, Berlin, Germany.; Experimental and Clinical Research Center, Charité, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany.; Max Delbrück Center for Molecular Medicine in the Helmholtz Association (MDC), Berlin, Germany.; Department of Neurology, St. Josef Hospital, Ruhr-University Bochum, Bochum, Germany.; Department of Neurology, Medical Faculty, Heinrich Heine University Düsseldorf, Düsseldorf, Germany.; Department of Neurology, Medical Faculty, Heinrich Heine University Düsseldorf, Düsseldorf, Germany.; Department of Neurology, Heinrich Heine University, Centre for Neurology and Neuropsychiatry, LVR-Klinikum, Düsseldorf, Germany.; Department of Neurology, Johanna Etienne Hospital, Neuss, Germany.; Department of Neurology, School of Medicine and Health, Technical University Munich, Klinikum rechts der Isar, Munich, Germany.; Department of Neurology and Institute of Neuroimmunology and MS (INIMS), University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; Molecular Neuroimmunology Group, Department of Neurology, University of Heidelberg, Heidelberg, Germany.; Department of Neurology with Institute of translational Neurology, University of Münster, Munster, Germany.; Department of Neurology, University of Ulm, Ulm, Germany.; Department of Neurology, University of Leipzig, Leipzig, Germany.; Department of Neurology, Herford Hospital, Herford, Germany.; Department of Neurology, Alfried Krupp Hospital, Essen, Germany.; Marianne-Strauß-Klinik, Behandlungszentrum Kempfenhausen für Multiple Sklerose Kranke, Berg, Germany.; Department of Neurology, University of Regensburg, Regensburg, Germany.; Department of Neurology, Nordwestkrankenhaus Sanderbusch, Sande, Germany.; Department of Neurology, University of Greifswald, Greifswald, Germany.; Section of Translational Neuroimmunology, Department of Neurology, Jena University Hospital, Jena, Germany.; Department of Neurology, Medical University of Vienna, Vienna, Austria.; Department of Neurology, Hanseklinikum Stralsund, Stralsund, Germany.; Department of Medical Statistics, University Medical Center Göttingen, Göttingen, Germany.; Department of Neurology and Institute of Neuroimmunology and MS (INIMS), University Medical Center Hamburg-Eppendorf, Hamburg, Germany.; APHM, Hopital de la Timone, CEMEREM, Marseille, France.; Aix Marseille Univ, CNRS, CRMBM, Marseille, France.
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