Nair Sruthi S, Jacob Saiju
Department of Neurology, University Hospitals Birmingham, Birmingham, B15 2TH, UK.
Department of Neurology, Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, Kerala, India.
Immunotargets Ther. 2023 Apr 4;12:25-45. doi: 10.2147/ITT.S377056. eCollection 2023.
Myasthenia gravis (MG), a prototype autoimmune neurological disease, had its therapy centred on corticosteroids, non-steroidal broad-spectrum immunotherapy and cholinesterase inhibitors for several decades. Treatment-refractory MG and long-term toxicities of the medications have been major concerns with the conventional therapies. Advances in the immunology and pathogenesis of MG have ushered in an era of newer therapies which are more specific and efficacious. Complement inhibitors and neonatal Fc receptor blockers target disease-specific pathogenic mechanisms linked to myasthenia and have proven their efficacy in pivotal clinical studies. B cell-depleting agents, specifically rituximab, have also emerged as useful for the treatment of severe MG. Many more biologicals are in the pipeline and in diverse stages of development. This review discusses the evidence for the novel therapies and the specific issues related to their clinical use.
重症肌无力(MG)是一种典型的自身免疫性神经疾病,几十年来其治疗主要集中在皮质类固醇、非甾体类广谱免疫疗法和胆碱酯酶抑制剂上。难治性MG以及药物的长期毒性一直是传统疗法的主要关注点。MG免疫学和发病机制的进展开启了一个更新疗法的时代,这些疗法更具特异性且更有效。补体抑制剂和新生儿Fc受体阻滞剂针对与重症肌无力相关的疾病特异性致病机制,并已在关键临床研究中证明了其疗效。B细胞耗竭剂,特别是利妥昔单抗,也已成为治疗重症肌无力的有效药物。还有更多生物制剂正在研发中,处于不同的开发阶段。本综述讨论了这些新疗法的证据以及与其临床应用相关的具体问题。