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
Department of Neurology and Institute of Neuroimmunology and MS (INIMS), University Medical Center Hamburg-Eppendorf, Hamburg, Germany
Department of Neurology, Hannover Medical School, Hannover, Germany.
J Neurol Neurosurg Psychiatry. 2025 May 14;96(6):582-592. doi: 10.1136/jnnp-2024-334764.
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.
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.
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.
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.
视神经脊髓炎谱系障碍（NMOSD）或髓鞘少突胶质细胞糖蛋白抗体相关疾病（MOGAD）的复发发作可导致严重残疾。我们旨在分析NMOSD和MOGAD患者免疫疗法的实际应用情况，重点关注治疗策略的变化、对发作率（ARR）的影响以及发作的危险因素。
这项基于纵向注册的队列研究纳入了493例患者（320例水通道蛋白4免疫球蛋白G（AQP4-IgG）血清阳性的NMOSD患者（65%）、44例AQP4-IgG血清阴性的NMOSD患者（9%）和129例MOGAD患者（26%）），他们接受了来自德国19个和奥地利1个中心的视神经脊髓炎研究组（NEMOS）注册登记的1247次治疗。我们分析了未经调整的ARR，并进行了生存分析和Cox比例风险回归，以评估随时间推移后续发作的疗效和危险因素。
利妥昔单抗和硫唑嘌呤是NMOSD以及MOGAD中使用最广泛的免疫疗法，在过去十年中其分布有所变化。免疫疗法在NMOSD中显示出显著的治疗效果，但在MOGAD中效果不太明显。发作的危险因素包括年龄较小和在相同治疗下既往发作过。不同免疫疗法的疗效各不相同，硫唑嘌呤、利妥昔单抗和依库珠单抗在AQP4-IgG血清阳性的NMOSD中显示出显著的风险降低。
本研究提供了有关NMOSD和MOGAD中免疫疗法不断演变的治疗格局和有效性的见解。既定的非标签疗法继续发挥重要作用，特别是对于病情稳定的患者，同时有新出现的证据支持新批准的疗法。未来需要开展研究以完善治疗算法并解决MOGAD管理中持续存在的不确定性。
Zotero 插件
