重症肌无力靶向治疗进展

Progress in Targeted Therapy for Myasthenia Gravis

摘要: 重症肌无力(myasthenia gravis, MG)是一种慢性的、波动的、补体和抗体介导的自身免疫性疾病，针对骨骼肌突触后神经肌肉连接的疾病。临床上主要表现为全身或局部肌肉无力、容易疲劳等症状。乙酰胆碱酯酶和皮质类固醇仍然是一线治疗，静脉注射免疫球蛋白(IVIG)和血浆置换(PE)为急性加重期的推荐疗法，特别是重症肌无力危象。然而，MG的治疗需要长期的免疫抑制，传统药物有多样选择性，但需长时间才能起作用，并有相应的不良反应，大约10%~15%的MG患者对传统疗法无效。过去十年，已经出现了几种新的生物制剂，包括补体抑制剂、新生儿Fc受体(FcRn)抑制剂、B细胞耗竭剂等，它们具有靶向性的免疫治疗，副作用少，起效快等特点。本文就几种生物制剂的疗效、安全性等进行综述。

Abstract: Myasthenia gravis (MG) is a chronic, fluctuating, complement and antibody mediated autoimmune disease that targets the postsynaptic neuromuscular connections in skeletal muscles. The main clinical manifestations are systemic or local muscle weakness, and easy fatigue. Acetylcholinesterase and corticosteroids remain first-line treatments, while intravenous immunoglobulin (IVIG) and plasma exchange (PE) are recommended therapies for acute exacerbation, especially in the crisis of myasthenia gravis. However, the treatment of MG requires long-term immunosuppression, and traditional drugs have diverse selectivity but require a long time to take effect, with corresponding adverse reactions. About 10%~15% of MG patients are ineffective with traditional therapies. In the past decade, several new biological agents have emerged, including B-cell depleting agents, complement inhibitors, and neonatal Fc receptor (FcRn) inhibitors, which have the characteristics of targeted immunotherapy, low side effects, and fast onset. This article provides a review of the efficacy, safety, and other aspects of several biological agents.