TY - JOUR
T1 - Real-world multicentre cohort study on choices and effectiveness of immunotherapies in NMOSD and MOGAD
AU - Neuromyelitis Optica Study Group (NEMOS)
AU - Häußler, Vivien
AU - Trebst, Corinna
AU - Engels, Daniel
AU - Pellkofer, Hanna
AU - Havla, Joachim
AU - Duchow, Ankelien
AU - Schindler, Patrick
AU - Schwake, Carolin
AU - Pakeerathan, Thivya
AU - Fischer, Katinka
AU - Ringelstein, Marius
AU - Lindenblatt, Gero
AU - Hümmert, Martin W.
AU - Tkachenko, Daria
AU - Bütow, Franziska
AU - Giglhuber, Katrin
AU - Flaskamp, Martina
AU - Schiffmann, Insa
AU - Korporal-Kuhnke, Mirjam
AU - Jarius, Sven
AU - Dawin, Eva
AU - Revie, Lisa
AU - Senel, Makbule
AU - Herfurth, Mariella
AU - Walter, Annette
AU - Pompsch, Mosche
AU - Kleiter, Ingo
AU - Angstwurm, Klemens
AU - Kaste, Matthias
AU - Grothe, Matthias
AU - Wickel, Jonathan
AU - Rommer, Paulus Stefan
AU - Sieb, Jörn Peter
AU - Krämer, Markus
AU - Then Bergh, Florian
AU - Tumani, Hayrettin
AU - Klotz, Luisa
AU - Wildemann, Brigitte
AU - Aktas, Orhan
AU - Ayzenberg, Ilya
AU - Bellmann-Strobl, Judith
AU - Paul, Friedemann
AU - Kümpfel, Tania
AU - Friede, Tim
AU - Berthele, Achim
AU - Stellmann, Jan Patrick
AU - Albrecht, Philipp
AU - Asseyer, Susanna
AU - Bayas, Antonios
AU - Hemmer, Bernhard
N1 - Publisher Copyright:
© 2025 Author(s) (or their employer(s)). Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ Group.
PY - 2025/6/1
Y1 - 2025/6/1
N2 - 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.
AB - 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.
KW - NEUROIMMUNOLOGY
UR - https://www.scopus.com/pages/publications/85214555783
U2 - 10.1136/jnnp-2024-334764
DO - 10.1136/jnnp-2024-334764
M3 - Article
C2 - 39730197
AN - SCOPUS:85214555783
SN - 0022-3050
VL - 96
SP - 582
EP - 592
JO - Journal of Neurology, Neurosurgery and Psychiatry
JF - Journal of Neurology, Neurosurgery and Psychiatry
IS - 6
ER -