Telitacicept as an alternative to non-steroidal immunosuppressive therapies in the treatment of myasthenia gravis: a study on clinical efficacy and steroid-sparing effect.

IF 5.7 2区 医学 Q1 IMMUNOLOGY
Frontiers in Immunology Pub Date : 2025-03-24 eCollection Date: 2025-01-01 DOI:10.3389/fimmu.2025.1549034
Zheyu Fang, Yuan Zhang, Yu Zhang, Qiaoyi Zhang, Xi Qu, Shengli Pan, Bingbing Wan, Shiyin Yang, Xu Zhang, Jia Li
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Abstract

Introduction: Myasthenia Gravis (MG) is an autoimmune disorder characterized by impaired neuromuscular junction (NMJ) transmission. Current treatments for MG include steroids and nonsteroidal immunosuppressive therapies (NSISTs). However, approximately 20% of patients show a poor response to these therapies, which are often associated with significant side effects. Telitacicept, a novel recombinant fusion protein targeting the BAFF/APRIL pathway, has shown promise in treating autoimmune diseases, including MG.

Methods: This retrospective study compared the efficacy of telitacicept monotherapy (10 patients) to NSISTs (16 patients) and sequential therapy (6 patients) in managing Myasthenia Gravis (MG) at The First Affiliated Hospital of Wenzhou Medical University (July 2020-November 2024). The primary endpoint was the time to achieve minimal symptom expression (MSE), and secondary endpoint was the change in the mean daily prednisone dosage from baseline to month 4.

Results: Among telitacicept-treated patients, 80% achieved MSE within 4 months, with a significant reduction in mean daily dose of prednisone (from 45.00 mg to 6.25 mg, P < 0.001). In contrast, only 12.5% of the NSISTs group achieved MSE, with no significant change in mean daily dose of prednisone (P = 0.091). The sequential therapy group (efgartigimod followed by telitacicept) maintained stable disease conditions.

Conclusion: Telitacicept is effective in inducing MSE rapidly and offers a steroid-sparing effect, making it a promising alternative to traditional NSISTs with fewer side effects in MG patients.

替利他赛普替代非甾体免疫抑制治疗重症肌无力:临床疗效及甾体节约效应研究
重症肌无力(MG)是一种以神经肌肉连接(NMJ)传递受损为特征的自身免疫性疾病。目前治疗MG的方法包括类固醇和非类固醇免疫抑制疗法(nsts)。然而,大约20%的患者对这些疗法反应不佳,而且往往伴有明显的副作用。Telitacicept是一种靶向BAFF/APRIL通路的新型重组融合蛋白,在治疗自身免疫性疾病(包括MG)方面显示出前景。方法:回顾性比较温州医科大学第一附属医院(2020年7月- 2024年11月)泰利他塞普单药治疗(10例)与nsts治疗(16例)和序贯治疗(6例)治疗重症肌无力(MG)的疗效。主要终点是达到最小症状表达(MSE)的时间,次要终点是从基线到第4个月的平均每日泼尼松剂量的变化。结果:在接受telitacicept治疗的患者中,80%的患者在4个月内达到了MSE,强的松的平均日剂量显著减少(从45.00 mg降至6.25 mg, P < 0.001)。相比之下,只有12.5%的nsts组达到了MSE,而泼尼松的平均日剂量没有显著变化(P = 0.091)。序贯治疗组(依加替莫德加泰利他塞普)维持病情稳定。结论:Telitacicept具有快速诱导MSE的效果,并具有节省激素的作用,是MG患者替代传统nsts的一种有希望的替代方案,副作用更小。
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
CiteScore
9.80
自引率
11.00%
发文量
7153
审稿时长
14 weeks
期刊介绍: Frontiers in Immunology is a leading journal in its field, publishing rigorously peer-reviewed research across basic, translational and clinical immunology. This multidisciplinary open-access journal is at the forefront of disseminating and communicating scientific knowledge and impactful discoveries to researchers, academics, clinicians and the public worldwide. Frontiers in Immunology is the official Journal of the International Union of Immunological Societies (IUIS). Encompassing the entire field of Immunology, this journal welcomes papers that investigate basic mechanisms of immune system development and function, with a particular emphasis given to the description of the clinical and immunological phenotype of human immune disorders, and on the definition of their molecular basis.
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