Ofatumumab treatment in patients with neuromyelitis optica spectrum disorder: a retrospective multicenter cohort study.

IF 4.6 2区医学 Q1 CLINICAL NEUROLOGY

Journal of Neurology Pub Date : 2025-09-26 DOI:10.1007/s00415-025-13408-2

Xiaoxia Yang, Zhen Jia, Xuegan Lian, Rui Zhang, Bin Li, Daishi Tian, Xiuju Gao, Shougang Guo, Baojie Wang, Hongbo Liu, Youming Long, Limei Wang, Jingfen Zhang, Qun Xue, Zhihou Liang, Yongkai Han, Huiyu Feng, Lin Huang, Pin Wang, Naiyuan Shao, Fu-Dong Shi, Chao Zhang

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引用次数: 0

Abstract

Background: Ofatumumab is a fully human anti-CD20 monoclonal antibody that selectively and highly depletes B cells. However, limited data on ofatumumab treatment are available in patients with neuromyelitis optica spectrum disorders (NMOSD). In this study, we aimed to evaluate the efficacy and safety of subcutaneous ofatumumab in patients with NMOSD.

Methods: We conducted a retrospective multicenter cohort study of patients with NMOSD who received ofatumumab treatment at 15 tertiary hospitals in China. The primary outcome was the annualized relapse rate (ARR). The secondary outcomes included disability measures (Expanded Disability Status Scale score, EDSS; the Aminoff-Logue Disability Scale, ALS), changes in aquaporin-4 IgG (AQP4-IgG) titers, and safety profiles during ofatumumab treatment.

Results: A total of 112 patients (88% female, median age 44.0 years with interquartile range [IQR 29.5-57.5]) received ofatumumab treatment for a median of 1.7 years (IQR: 1.1-2.0). The median ARR decreased significantly from 2.0 (IQR 0.7-10.0) before ofatumumab to 0 (IQR 0.0-0.0; p < 0.001) after ofatumumab. Twenty-two patients (20%) experienced 25 relapses, with 20 (80%) occurring within the first year of initiating ofatumumab treatment and 19 (76%) classified as minor. The EDSS score from start to the last follow-up also improved significantly (median: pre-treatment 3.5, IQR 2.0-6.5, post-treatment: 2.0, IQR1.0-3.5, p < 0.001). Among 94 patients, 71 (76%) showed reduced AQP4-IgG titers at last follow-up. Injection-related reactions were reported in 13 (12%) of 112 patients. Twenty-four infections occurred in 20 patients (18%) during the ofatumumab treatment, with 92% (22/24) being grade 1 or 2 (CTCAE version 5.0). Only 2 patients (2%) experienced pneumonia requiring hospitalization and recovered after antibiotic treatment (grade 3). Hypogammaglobulinemia was recorded in 14% (13/95) of patients and was not associated with infection.

Conclusions: Subcutaneous ofatumumab treatment significantly reduces the relapse risk, limits worsening of disability, and reduces AQP4-IgG titers in NMOSD. Moreover, the safety profiles were generally acceptable. Further research is necessary to explore the sustained clinical response of ofatumumab in NMOSD.

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Ofatumumab治疗视神经脊髓炎谱系障碍患者：一项回顾性多中心队列研究

背景：Ofatumumab是一种全人源抗cd20单克隆抗体，选择性和高度消耗B细胞。然而，ofatumumab治疗视神经脊髓炎谱系障碍（NMOSD）患者的数据有限。在这项研究中，我们旨在评估皮下ofatumumab治疗NMOSD患者的有效性和安全性。方法：我们对中国15家三级医院接受ofatumumab治疗的NMOSD患者进行了一项回顾性多中心队列研究。主要终点为年复发率（ARR）。次要结果包括残疾测量（扩展残疾状态量表评分，EDSS； Aminoff-Logue残疾量表，ALS），水通道蛋白-4 IgG （AQP4-IgG）滴度的变化，以及阿图单抗治疗期间的安全性。结果：共有112例患者（88%为女性，中位年龄44.0岁，四分位数间差[IQR: 29.5-57.5]）接受了ofatumumab治疗，中位时间为1.7年（IQR: 1.1-2.0）。中位ARR从ofatumumab治疗前的2.0 （IQR 0.7-10.0）显著下降至0 (IQR 0.0-0.0)； p结论：皮下ofatumumab治疗可显著降低NMOSD患者的复发风险，限制残疾恶化，并降低AQP4-IgG滴度。此外，安全概况大体上是可以接受的。需要进一步的研究来探索ofatumumab在NMOSD中的持续临床反应。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Neurology 医学-临床神经学

CiteScore

10.00

自引率

5.00%

发文量

558

审稿时长

1 months

期刊介绍： The Journal of Neurology is an international peer-reviewed journal which provides a source for publishing original communications and reviews on clinical neurology covering the whole field. In addition, Letters to the Editors serve as a forum for clinical cases and the exchange of ideas which highlight important new findings. A section on Neurological progress serves to summarise the major findings in certain fields of neurology. Commentaries on new developments in clinical neuroscience, which may be commissioned or submitted, are published as editorials. Every neurologist interested in the current diagnosis and treatment of neurological disorders needs access to the information contained in this valuable journal.