Myasthenia Gravis Treatment: From Old Drugs to Innovative Therapies with a Glimpse into the Future.

IF 7.4 2区医学 Q1 CLINICAL NEUROLOGY

CNS drugs Pub Date : 2024-01-01 Epub Date: 2024-01-11 DOI:10.1007/s40263-023-01059-8

Salvatore Crisafulli, Brigida Boccanegra, Massimo Carollo, Emanuela Bottani, Paola Mantuano, Gianluca Trifirò, Annamaria De Luca

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Abstract

Myasthenia gravis (MG) is a rare autoimmune disease that causes debilitating muscle weakness due to impaired neuromuscular transmission. Since most (about 80-90%) MG patients present autoantibodies against the acetylcholine receptor, standard medical therapy consists of symptomatic treatment with acetylcholinesterase inhibitors (e.g., pyridostigmine). In addition, considering the autoimmune basis of MG, standard therapy includes immunomodulating agents, such as corticosteroids, azathioprine, cyclosporine A, and cyclophosphamide. New strategies have been proposed for the treatment of MG and include complement blockade (i.e., eculizumab, ravulizumab, and zilucoplan) and neonatal Fc receptor antagonism (i.e., efgartigimod and rozanolixizumab). The aim of this review is to provide a detailed overview of the pre- and post-marketing evidence on the five pharmacological treatments most recently approved for the treatment of MG, by identifying both preclinical and clinical studies registered in clinicaltrials.gov. A description of the molecules currently under evaluation for the treatment of MG is also provided.

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肌无力治疗：从旧药到创新疗法，展望未来。

重症肌无力（MG）是一种罕见的自身免疫性疾病，会因神经肌肉传递受损而导致肌肉无力。由于大多数（约 80-90%）重症肌无力患者会出现针对乙酰胆碱受体的自身抗体，因此标准的药物治疗包括使用乙酰胆碱酯酶抑制剂（如吡啶斯的明）进行对症治疗。此外，考虑到 MG 的自身免疫基础，标准疗法还包括免疫调节剂，如皮质类固醇、硫唑嘌呤、环孢素 A 和环磷酰胺。目前已提出治疗 MG 的新策略，包括补体阻断（即 eculizumab、ravulizumab 和 zilucoplan）和新生儿 Fc 受体拮抗（即 efgartigimod 和 rozanolixizumab）。本综述旨在通过识别在 clinicaltrials.gov 上注册的临床前和临床研究，详细概述最近获批用于治疗 MG 的五种药物治疗的上市前和上市后证据。本文还介绍了目前正在评估的用于治疗 MG 的分子。

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

CNS drugs 医学-精神病学

CiteScore

12.00

自引率

3.30%

发文量

审稿时长

6-12 weeks

期刊介绍： CNS Drugs promotes rational pharmacotherapy within the disciplines of clinical psychiatry and neurology. The Journal includes: - Overviews of contentious or emerging issues. - Comprehensive narrative reviews that provide an authoritative source of information on pharmacological approaches to managing neurological and psychiatric illnesses. - Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. - Adis Drug Reviews of the properties and place in therapy of both newer and established drugs in neurology and psychiatry. - Original research articles reporting the results of well-designed studies with a strong link to clinical practice, such as clinical pharmacodynamic and pharmacokinetic studies, clinical trials, meta-analyses, outcomes research, and pharmacoeconomic and pharmacoepidemiological studies. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in CNS Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.