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重症肌无力的常规和新兴治疗方法及争议。

Conventional and emerging treatments and controversies in myasthenia gravis.

机构信息

Dipartimento di Neuroscienze, Università Cattolica del Sacro Cuore, Rome, Italy.

Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.

出版信息

Expert Rev Neurother. 2023 May;23(5):445-456. doi: 10.1080/14737175.2023.2207739. Epub 2023 May 3.

Abstract

INTRODUCTION

Myasthenia gravis (MG) is caused by IgG antibodies against different proteins at the neuromuscular junction. Anti-acetylcholine receptor (AChR) Abs are detected in the great majority of patients. MG management consists of long-term immunotherapy, based on steroids and immunosuppressants, short-term treatments and therapeutic thymectomy. Targeted immunotherapies that reduce B cell survival, inhibit complement activation, and decrease serum IgG concentration have been evaluated in trials and have entered clinical practice.

AREAS COVERED

Herein, the efficacy and safety data of conventional and novel therapeutic options are reviewed and their indications in the disease subtypes are discussed.

EXPERT OPINION

Even though conventional treatment is generally effective, 10-15% of patients have refractory disease and there are safety concerns related to long-term immunosuppression. Novel therapeutic options offer several advantages but also have limitations. Safety data based on long-term treatment are not yet available for some of these agents. The mechanisms of action of new drugs and the immunopathogenesis of different MG subtypes must be considered in therapy decision making. Integrating new agents in the treatment scenario of MG can significantly improve disease management.

摘要

简介

重症肌无力(MG)是由神经肌肉接头处不同蛋白的 IgG 抗体引起的。大多数患者都检测到抗乙酰胆碱受体(AChR)抗体。MG 的治疗包括长期免疫治疗,基于类固醇和免疫抑制剂,以及短期治疗和胸腺切除术。已在临床试验中评估了靶向免疫疗法,这些疗法可降低 B 细胞的存活率、抑制补体的激活,并降低血清 IgG 浓度,已进入临床实践。

涵盖领域

本文回顾了传统和新型治疗选择的疗效和安全性数据,并讨论了它们在疾病亚型中的适应证。

专家意见

尽管常规治疗通常有效,但仍有 10-15%的患者存在难治性疾病,而且长期免疫抑制存在安全性问题。新型治疗选择具有多种优势,但也存在局限性。其中一些药物的长期治疗安全性数据尚不可用。在治疗决策中,必须考虑新型药物的作用机制和不同 MG 亚型的免疫发病机制。将新药物整合到 MG 的治疗方案中可以显著改善疾病管理。

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