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

Häußler V, Trebst C, Engels D, Pellkofer H, Havla J, Schindler P, Schwake C, Ringelstein M, Hümmert MW, Flaskamp M, Schiffmann I, Korporal-Kuhnke M, Jarius S, Revie L, Senel M, Walter A, Kleiter I, Grothe M, Bergh FT, Tumani H, Klotz L, Wildemann B, Aktas O, Ayzenberg I, Bellmann-Strobl J, Paul F, Kümpfel T, Friede T, Stellmann JP, Albrecht P, Angstwurm K, Asseyer S, Bayas A, Bednarz N, Behnke S, Berthele A, Bittner S, Bütow F, Buttmann M, Dawin E, Dersch R, Duchow A, Etgen T, Finke C, Fischer K, Förster M, Fousse M, Frank B, Freitag F, Gahlen A, Gass A, Gehrig J, Geis C, Giglhuber K, Goereci Y, Gold R, Ayrosa Galvao Ribeiro Gomes AB, Graf J, Groppa S, Gutbrod J, Guthke K, Haarmann A, Hastermann M, Hellwig K, Hemmer B, Herfurth M, Herwerth M, Hoffmann F, Hoffmann O, Hofstadt-Van Oy U, Husseini L, Ingenhoven K, Junghans J, Kaste M, Kern K, Kern P, Kermer P, Kleinschnitz C, Köhler W, Körbel K, Kowarik M, Krämer M, Krumbholz M, Kunzmann V, Kurka N, Ladopoulos T, Landwehr A, Langel S, Lauenstein AS, Laurent S, Lee DH, Lehrieder D, Leypoldt F, Liebetrau M, Lindenblatt G, Linker R, Lohmann L, Lüdemann P, Luessi F, Maiworm M, Marziniak M, Mayer C, Meister S, Melms A, von Mering M, Metz I, Meuth S, Naumann J, Nenkov M, Neuhaus O, Neziraj T, Niederschweiberer M, Niehaus S, Oertel FC, Otto C, Pache F, Pakeerathan T, Paryjas S, Pawlitzki M, Pepping S, Pfeuffer S, Pompsch M, Pretzsch RA, Proebstel AK, Protopapa M, Puhlmann HU, Pul R, Rauer S, Rehfeldt T, Retzlaff N, Riedlinger A, Rommer P, Rostásy K, Rothhammer V, Rückriem L, Ruprecht K, Ruschil C, Saggau C, Schraad M, Schwab M, Schwarz P, Seipelt M, Sieb JP, Soloroza G, Sommer C, Stefanou A, Steinbrecher A, Stephanik H, Steuerwald V, Stoppe M, Stürner K, Süße M, Tarampanis A, Tauber S, Tkachenko D, Tsaktanis T, Tumani H, Wallwitz U, Walz A, Wandinger KP, Weber MS, Weise J, Wickel J, Wiendl H, Winkelmann A, Wohlrab F, Yalachkov Y, Zettl U, Ziemann U, Zipp F (2024)

Publication Type: Journal article

Publication year: 2024

Journal

Article Number: jnnp-2024-334764

DOI: 10.1136/jnnp-2024-334764

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.

Authors with CRIS profile

Involved external institutions

Universitätsmedizin der Johannes Gutenberg-Universität Mainz Germany (DE) Heinrich-Heine-Universität Düsseldorf Germany (DE) Kliniken Maria Hilf GmbH Germany (DE) Universitätsklinikum des Saarlandes (UKS) Germany (DE) Universitätsklinikum Essen Germany (DE) Ruhr-Universität Bochum (RUB) Germany (DE) Universitätsklinikum Mannheim / University Medical Centre Mannheim (Universitätsmedizin Mannheim) Germany (DE) Universitätsklinikum Frankfurt am Main (KGU) Germany (DE) Universitätsklinikum Jena Germany (DE) Klinikum rechts der Isar Germany (DE) Universitätsklinikum Köln Germany (DE) Universitätsspital Basel Switzerland (CH) Universitätsklinikum Düsseldorf Germany (DE) Universitätsklinikum Hamburg-Eppendorf (UKE) Germany (DE) Medizinische Hochschule Hannover (MHH) / Hannover Medical School Germany (DE) Klinikum der Universität München (LMU Klinikum) Germany (DE) Charité - Universitätsmedizin Berlin Germany (DE) Katholisches Klinikum Bochum (St. Josef- und St. Elisabeth-Hospital gGmbH) Germany (DE) Ruprecht-Karls-Universität Heidelberg Germany (DE) Universität Münster (ehem. Westfälische Wilhelms-Universität (WWU)) Germany (DE) Universität Ulm Germany (DE) Klinikum Herford Germany (DE) Marianne-Strauß-Klinik Germany (DE) Universität Greifswald Germany (DE) Universität Leipzig Germany (DE) Universitätsklinikum Göttingen Germany (DE) Universität Regensburg Germany (DE) Universitätsklinikum Leipzig Germany (DE) Knappschaftsklinikum Saar Germany (DE) Caritas-Krankenhaus Bad Mergentheim gemeinnützige GmbH Germany (DE) Universitätsklinikum Freiburg Germany (DE) Kliniken Südostbayern AG Germany (DE) Städtisches Klinikum Görlitz Germany (DE) Julius-Maximilians-Universität Würzburg Germany (DE) Technische Universität München (TUM) Germany (DE) Universitätsspital Zürich (USZ) Switzerland (CH) Klinikum Westfalen Germany (DE) Johanna-Etienne-Krankenhaus Germany (DE) Universitätsklinikum Regensburg Germany (DE) Universitätsklinikum Münster Germany (DE) Agaplesion Ev. Bathildiskrankenhaus Germany (DE) Isar-Amper-Hospital Germany (DE) Universitätsmedizin Rostock Germany (DE) Klinikum Bremen-Nord Germany (DE) SRH Kliniken Landkreis Sigmaringen GmbH Germany (DE) Klinikum Dortmund Germany (DE) Universitätsklinikum Gießen und Marburg (UKGM) Germany (DE) Alfried Krupp Krankenhaus Germany (DE) Dietrich-Bonhoeffer-Klinikum Germany (DE) Asklepios Kliniken Germany (DE) Medizinische Universität Wien Austria (AT) Vestische Caritas-Kliniken GmbH Germany (DE) MediClin Hedon Klinik Germany (DE) Universitätsklinikum Tübingen Germany (DE) Universitätsklinikum Schleswig-Holstein (UKSH) Germany (DE) Universitätsklinikum Würzburg Germany (DE) Katharinenhospital Stuttgart Germany (DE) HELIOS Kliniken Germany (DE) Universitätsklinikum Magdeburg A.ö.R. Germany (DE) Universitätsmedizin Greifswald / Universitätsklinikum Greifswald Germany (DE) Universitätsklinikum Aachen (UKA) Germany (DE) Nordwest-Krankenhaus Sanderbusch Germany (DE) Universitätsklinikum Brandenburg an der Havel Germany (DE) St. Josefs-Hospital Wiesbaden Germany (DE) Schlosspark-Klinik Germany (DE) Universitätsklinikum Ulm Germany (DE)

How to cite

APA:

Häußler, V., Trebst, C., Engels, D., Pellkofer, H., Havla, J., Schindler, P.,... Zipp, F. (2024). Real-world multicentre cohort study on choices and effectiveness of immunotherapies in NMOSD and MOGAD. Journal of Neurology Neurosurgery and Psychiatry. https://doi.org/10.1136/jnnp-2024-334764

MLA:

Häußler, Vivien, et al. "Real-world multicentre cohort study on choices and effectiveness of immunotherapies in NMOSD and MOGAD." Journal of Neurology Neurosurgery and Psychiatry (2024).

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