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