Department of Neurology, Guigang City People's Hospital, The Eighth Affiliated Hospital of Guangxi Medical University, Guigang, China.
Department of Pharmacy, The Second People's Hospital of Nanning City, The Third Affiliated Hospital of Guangxi Medical University, Nanning, China.
Front Immunol. 2021 Aug 11;12:715036. doi: 10.3389/fimmu.2021.715036. eCollection 2021.
Myasthenia gravis (MG) is an autoimmune disease primarily mediated by acetylcholine receptor antibodies (AChR-Ab), cellular immune dependence, and complement system involvement. Since the AChR on the postsynaptic membrane is destroyed by an immune attack, sufficient endplate potential cannot be generated, resulting in the development of a synaptic transmission disorder at the neuromuscular junction and in muscle weakness. The role of the complement system in MG has been demonstrated in animal models and clinical tests, and it has been determined that complement inhibition in patients with MG can prevent disease induction and reverse its progression. Eculizumab is a humanized monoclonal antibody that inhibits the cleavage of complement protein C5 and prevents autoimmune damage; additionally, it has received subsequent approval by the Federal Drug Administration of the United States for MG treatment. However, various concerns regarding the use of eculizumab persist. In this review, we have discussed the treatment time, cost effectiveness, long-term efficacy, and tolerability of eculizumab for MG treatment. We have also summarized historical information and have presented perspectives on this new therapeutic modality.
重症肌无力（MG）是一种主要由乙酰胆碱受体抗体（AChR-Ab）、细胞免疫依赖性和补体系统参与介导的自身免疫性疾病。由于突触后膜上的 AChR 受到免疫攻击而被破坏，因此无法产生足够的终板电位，导致神经肌肉接头处的突触传递障碍和肌肉无力。补体系统在 MG 中的作用已在动物模型和临床研究中得到证实，并且已经确定在 MG 患者中抑制补体可以预防疾病的诱导和逆转其进展。依库珠单抗是一种人源化单克隆抗体，可抑制补体蛋白 C5 的裂解，防止自身免疫损伤；此外，它已被美国联邦药物管理局批准用于治疗 MG。然而，关于依库珠单抗的使用仍存在各种担忧。在这篇综述中，我们讨论了依库珠单抗治疗 MG 的治疗时间、成本效益、长期疗效和耐受性。我们还总结了历史信息，并对这种新的治疗模式提出了看法。
