Current status and challenges of biologic targeted therapy for myasthenia gravis in China.

IF 1.6 Q2 MEDICINE, GENERAL & INTERNAL Intractable & rare diseases research Pub Date : 2026-02-28 DOI:10.5582/irdr.2025.01061
Shengying Gu, Chongbo Zhao
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Abstract

Myasthenia gravis (MG) is an autoimmune neuromuscular disorder posing substantial disease burden in China, with significant impacts on patient quality of life. While conventional immunosuppressants remain fundamental therapy, biologic targeted agents-including B-cell targeting drugs, complement C5 inhibitors, FcRn antagonists, and IL-6R inhibitors-have brought major advances, especially for refractory and MuSK-antibody-positive MG. Increasing availability in China has demonstrated promising clinical outcomes, yet substantial barriers remain. Challenges include high drug costs, limited insurance access, insufficient multicenter clinical evidence, and disparities in physician adoption. China's research is improving, exemplified by local innovation with telitacicept and active participation in global trials, though most biologics remain imported. To maximize benefits, clinical application should be guided by antibody subtype and immunopathology, with individualized, dynamic regimens balancing efficacy, safety, and affordability. High-quality randomized trials, updated guidelines, broader insurance coverage, and R&D investment are urgently needed to promote individualized and accessible MG care in China.

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中国重症肌无力生物靶向治疗的现状与挑战
重症肌无力(MG)是一种自身免疫性神经肌肉疾病,在中国造成了巨大的疾病负担,严重影响患者的生活质量。虽然传统的免疫抑制剂仍然是基本的治疗方法,但生物靶向药物——包括b细胞靶向药物、补体C5抑制剂、FcRn拮抗剂和IL-6R抑制剂——已经取得了重大进展,特别是对于难治性和musk抗体阳性的MG。在中国,越来越多的可获得性已显示出良好的临床效果,但仍存在实质性障碍。面临的挑战包括高昂的药物成本、有限的保险获取、多中心临床证据不足以及医生采用的差异。中国的研究正在进步,例如telitacicept的本土创新和积极参与全球试验,尽管大多数生物制剂仍然是进口的。为了最大限度地提高疗效,临床应用应以抗体亚型和免疫病理为指导,采用个体化、动态的方案,平衡疗效、安全性和可负担性。中国迫切需要高质量的随机试验、更新的指南、更广泛的保险覆盖和研发投资来促进个体化和可及性MG护理。
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来源期刊
Intractable & rare diseases research
Intractable & rare diseases research MEDICINE, GENERAL & INTERNAL-
CiteScore
2.10
自引率
0.00%
发文量
29
期刊最新文献
Current status and challenges of biologic targeted therapy for myasthenia gravis in China. Foods for special medical purposes for the dietary therapy of rare diseases: Current status and future prospects. Progress in the research and pharmacoeconomic evaluation of drugs and devices for rare diseases in China. Health technology assessment and medical insurance access for rare disease drugs in China: A policy review with quantitative insights from publicly available data. Visualization analysis of the use of traditional Chinese medicine in the diagnosis and treatment of rare diseases in mainland China based on CiteSpace.
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