• J. Neurol. Neurosurg. Psychiatr. · Dec 2024

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

    • Vivien Häußler, Corinna Trebst, Daniel Engels, Hanna Pellkofer, Joachim Havla, Ankelien Duchow, Patrick Schindler, Carolin Schwake, Thivya Pakeerathan, Katinka Fischer, Marius Ringelstein, Gero Lindenblatt, Martin W Hümmert, Daria Tkachenko, Franziska Bütow, Katrin Giglhuber, Martina Flaskamp, Insa Schiffmann, Mirjam Korporal-Kuhnke, Sven Jarius, Eva Dawin, Lisa Revie, Makbule Senel, Mariella Herfurth, Annette Walter, Mosche Pompsch, Ingo Kleiter, Klemens Angstwurm, Matthias Kaste, Matthias Grothe, Jonathan Wickel, Paulus Stefan Rommer, Jörn Peter Sieb, Markus Krämer, Florian Then Bergh, Hayrettin Tumani, Luisa Klotz, Brigitte Wildemann, Orhan Aktas, Ilya Ayzenberg, Judith Bellmann-Strobl, Friedemann Paul, Tania Kümpfel, Tim Friede, Achim Berthele, Jan-Patrick Stellmann, and Neuromyelitis optica study group (NEMOS).
    • Department of Neurology and Institute of Neuroimmunology and MS (INIMS), University Medical Center Hamburg-Eppendorf, Hamburg, Germany v.haeussler@uke.de.
    • J. Neurol. Neurosurg. Psychiatr. 2024 Dec 27.

    BackgroundRecurrent 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.MethodsThis 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.ResultsRituximab 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.ConclusionsThis 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)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.

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