Department of Neurology, Neurological Institute, Cleveland Clinic, Cleveland, OH, USA.
Neuromuscular Center, Neurological Institute, Cleveland Clinic, Cleveland, OH, USA.
Expert Opin Pharmacother. 2024 Mar;25(4):395-408. doi: 10.1080/14656566.2024.2332610. Epub 2024 Mar 25.
Myasthenia gravis (MG) is an autoimmune condition targeting the neuromuscular junction, which manifests with neuromuscular symptoms of varying severity and significant morbidity. The mainstay of treatment in MG is mitigation of the immune cascade with steroids and non-steroidal immunosuppressive therapies. The therapeutic strategies in MG are transitioning from broad and indiscriminate immunosuppression to novel agents targeting key steps in MG pathogenesis, including T cell activation, B cell proliferation, complement activation, maintenance of pathogenic antibody production, and proinflammatory cytokine production.
In this review, an overview of the pathogenesis of MG and traditional MG therapies is presented, followed by a discussion of the novel MG drugs that have been evaluated in phase 3 clinical trials with an emphasis on those which have received regulatory approval.
Novel MG therapeutics belonging to the classes of complement inhibitors, neonatal Fc receptor (FcRn) inhibitors and B cell depletors, as well as the other emerging MG drugs in the pipeline constitute promising treatment strategies with potentially better efficacy and safety compared to the conventional MG treatments. However, further long-term research is needed in order to optimize the implementation of these new treatment options for the appropriate patient populations.
重症肌无力(MG)是一种针对神经肌肉接头的自身免疫性疾病,表现为严重程度不同的神经肌肉症状和高发病率。MG 的主要治疗方法是用类固醇和非甾体类免疫抑制疗法来减轻免疫级联反应。MG 的治疗策略正在从广泛而无差别的免疫抑制转向针对 MG 发病机制关键步骤的新型药物,包括 T 细胞激活、B 细胞增殖、补体激活、维持致病性抗体产生和促炎细胞因子产生。
本文综述了 MG 的发病机制和传统 MG 治疗方法,随后讨论了已在 3 期临床试验中评估的新型 MG 药物,重点介绍了已获得监管批准的药物。
属于补体抑制剂、新生儿 Fc 受体(FcRn)抑制剂和 B 细胞耗竭剂类别的新型 MG 治疗药物,以及在研的其他新兴 MG 药物,与传统 MG 治疗相比,具有潜在更好的疗效和安全性,是有前途的治疗策略。然而,需要进一步进行长期研究,以优化这些新的治疗选择在适当患者人群中的应用。