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

Haeussler, Vivien and Trebst, Corinna and Engels, Daniel and Pellkofer, Hanna and Havla, Joachim and Duchow, Ankelien and Schindler, Patrick and Schwake, Carolin and Pakeerathan, Thivya and Fischer, Katinka and Ringelstein, Marius and Lindenblatt, Gero and Huemmert, Martin W. and Tkachenko, Daria and Buetow, Franziska and Giglhuber, Katrin and Flaskamp, Martina and Schiffmann, Insa and Korporal-Kuhnke, Mirjam and Jarius, Sven and Dawin, Eva and Revie, Lisa and Senel, Makbule and Herfurth, Mariella and Walter, Annette and Pompsch, Mosche and Kleiter, Ingo and Angstwurm, Klemens and Kaste, Matthias and Grothe, Matthias and Wickel, Jonathan and Rommer, Paulus Stefan and Sieb, Jorn Peter and Kraemer, Markus and Then Bergh, Florian and Tumani, Hayrettin and Klotz, Luisa and Wildemann, Brigitte and Aktas, Orhan and Ayzenberg, Ilya and Bellmann-Strobl, Judith and Paul, Friedemann and Kuempfel, Tania and Friede, Tim and Berthele, Achim and Stellmann, Jan-Patrick (2025) Real-world multicentre cohort study on choices and effectiveness of immunotherapies in NMOSD and MOGAD. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 96 (6). pp. 582-592. ISSN 0022-3050, 1468-330X

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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.

Item Type: Article
Uncontrolled Keywords: OPTICA SPECTRUM DISORDER; DOUBLE-BLIND; EFFICACY; AZATHIOPRINE; RITUXIMAB; SAFETY; RECOMMENDATIONS; SATRALIZUMAB; DIAGNOSIS; NEUROIMMUNOLOGY
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Lehrstuhl für Neurologie
Depositing User: Dr. Gernot Deinzer
Date Deposited: 07 Apr 2026 11:34
Last Modified: 07 Apr 2026 11:34
URI: https://pred.uni-regensburg.de/id/eprint/65630

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