San Pyae Phyo, Jacob Saiju
Institute of Immunology and Immunotherapy, University of Birmingham, Birmingham, United Kingdom.
Department of Neurology, Queen Elizabeth Hospital Birmingham, University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom.
Front Neurol. 2023 Oct 5;14:1277596. doi: 10.3389/fneur.2023.1277596. eCollection 2023.
Myasthenia gravis is a prototypic neuroimmune disorder with autoantibodies targeting the acetylcholine receptor complex at the neuromuscular junction. Patients present with mainly ocular muscle weakness and tend to have a generalized muscle weakness later in the clinical course. The weakness can be severe and fatal when bulbar muscles are heavily involved. Acetylcholine receptor antibodies are present in the majority of patients and are of IgG1 and IgG3 subtypes which can activate the complement system. The complement involvement plays a major role in the neuromuscular junction damage and the supporting evidence in the literature is described in this article. Complement therapies were initially studied and approved for paroxysmal nocturnal hemoglobinuria and in the past decade, those have also been studied in myasthenia gravis. The currently available randomized control trial and real-world data on the efficacy and safety of the approved and investigational complement therapies are summarized in this review.
重症肌无力是一种典型的神经免疫疾病,其自身抗体靶向神经肌肉接头处的乙酰胆碱受体复合物。患者主要表现为眼肌无力,在临床病程后期往往会出现全身肌无力。当延髓肌肉严重受累时,肌无力可能会很严重甚至致命。大多数患者体内存在乙酰胆碱受体抗体,且为IgG1和IgG3亚型,可激活补体系统。补体参与在神经肌肉接头损伤中起主要作用,本文将描述文献中的支持证据。补体疗法最初是针对阵发性夜间血红蛋白尿进行研究并获批的,在过去十年中,也对其在重症肌无力中的应用进行了研究。本综述总结了目前关于已获批和正在研究的补体疗法的疗效和安全性的随机对照试验及真实世界数据。