Sivadasan Ajith, Bril Vera
Ellen & Martin Prosserman Centre for Neuromuscular Diseases, Toronto General Hospital, University Health Networkand University of Toronto, 5EC-309, TGH, 200 Elizabeth Street, Toronto, ON M5G 2C4, Canada.
Immunotherapy. 2023 Jun;15(8):553-563. doi: 10.2217/imt-2022-0298. Epub 2023 Apr 4.
Treatment of acute exacerbations and refractory myasthenia gravis (MG) remains challenging despite advances in immunotherapy. Frequent use of plasmapheresis and immunoglobulins are associated with adverse events and strain on resources. The neonatal Fc receptor (FcRn) facilitates IgG recycling and FcRn antagonism enhances the degradation of IgG pathogenic autoantibodies without compromising adaptive and innate immunity. Efgartigimod, an FcRN antagonist, has been shown in well-designed clinical trials to improve clinical status and reduce autoantibody levels without significant safety concerns. Efgartigimod has received approvals for use in the United States, Japan and Europe. It is plausible that efgartigimod is effective across different subgroups and varied spectrums of MG severity. Novel strategies involving FcRn modulation and long-term follow-up studies will help provide further insights and expand the therapeutic repertoire.
尽管免疫疗法取得了进展，但急性加重期和难治性重症肌无力（MG）的治疗仍然具有挑战性。频繁使用血浆置换和免疫球蛋白会带来不良事件并造成资源紧张。新生儿Fc受体（FcRn）促进IgG循环，FcRn拮抗作用可增强IgG致病性自身抗体的降解，同时不损害适应性免疫和固有免疫。Efgartigimod是一种FcRN拮抗剂，在精心设计的临床试验中已显示可改善临床状况并降低自身抗体水平，且无重大安全问题。Efgartigimod已在美国、日本和欧洲获得使用批准。Efgartigimod在不同亚组和不同严重程度的MG范围内均有效的说法似乎是合理的。涉及FcRn调节的新策略和长期随访研究将有助于提供进一步的见解并扩大治疗方法。
