Wingate University School of Pharmacy, Hendersonville, NC, USA.
The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Am J Health Syst Pharm. 2023 May 24;80(11):652-662. doi: 10.1093/ajhp/zxad035.
This review aims to provide an update on current pharmacological agents for the management of generalized myasthenia gravis (MG).
MG is an autoimmune disease characterized by impaired neuromuscular transmission and muscle weakness. Most patients have autoimmune antibodies to the nicotinic acetylcholine receptor, with treatments aimed at eliminating or decreasing levels of autoantibodies. Limitations of current treatments for generalized MG include limited efficacy and serious adverse effects, indicating a continued need for new treatments. Efgartigimod alfa, a biologic newly approved by the Food and Drug Administration, provides a novel treatment option for patients with chronic generalized MG.
While the landscape for treatment of generalized MG has expanded over recent years, there is still an unmet need for patients for whom multiple lines of treatment have failed. The introduction of neonatal Fc receptor antagonists such as efgartigimod alfa may have an immediate impact in patients for whom standard-of-care therapy has failed.
本综述旨在提供目前用于治疗全身性重症肌无力 (MG) 的药理学药物的最新信息。
MG 是一种自身免疫性疾病，其特征是神经肌肉传递受损和肌肉无力。大多数患者具有针对烟碱型乙酰胆碱受体的自身抗体，治疗旨在消除或降低自身抗体水平。目前用于治疗全身性 MG 的方法存在局限性，包括疗效有限和严重不良反应，表明仍需要新的治疗方法。Efgartigimod alfa 是一种新获得美国食品和药物管理局批准的生物制剂，为患有慢性全身性 MG 的患者提供了一种新的治疗选择。
尽管近年来治疗全身性 MG 的方法不断发展，但对于那些已经接受过多线治疗的患者，仍存在未满足的需求。新生儿 Fc 受体拮抗剂（如 efgartigimod alfa）的引入可能会对那些已经接受标准治疗失败的患者产生直接影响。
