Shi Fangyi, Chen Jiaxin, Feng Li, Lai Rong, Zhou Hongyan, Sun Xunsha, Shen Cunzhou, Feng Jiezhen, Feng Huiyu, Wang Haiyan
Department of Neurology, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, China.
Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, Guangzhou, China.
Front Neurol. 2024 Nov 19;15:1486659. doi: 10.3389/fneur.2024.1486659. eCollection 2024.
The prevalence of patients positive for muscle-specific kinase antibody (hereafter, MuSK-Ab) accounts for 5-8% of all myasthenia gravis (MG) cases. Currently, efgartigimod has shown good therapeutic effects in MUSK-Ab-positive MG patients in a phase III clinical trial. However, phase III clinical trials tend to exclude MG patients in exacerbation, and there are only few real-world studies on the efficacy of efgartigimod in MuSK-Ab-positive myasthenic crisis (MC) patients. This retrospective, real-world study aimed to explore the efficacy of efgartigimod in MuSK-Ab-positive MG with exacerbation.
We reviewed the clinical data of four MuSK-Ab-positive patients with exacerbation of MG who received efgartigimod at the First Affiliated Hospital of Sun Yat-sen University, including two patients with MC. All patients were admitted between September 2023 and May 2024. Most patients are simultaneously undergoing rituximab treatment.
Each patient completed one cycle of efgartigimod. After the first administration, four patients showed a clinically meaningful decrease in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score (a reduction of more than 4 points compared to baseline), and all patients showed a decrease in IgG levels after one cycle of efgartigimod. Regarding safety, none of the patients experienced any obvious adverse effects. At the final follow-up, all patients achieved the minimal symptom expression status (an MG-ADL score of 0 or 1) following the first administration of efgartigimod for 8.75 ± 5.56 weeks. This article presents a case involving a patient who exhibited prompt alleviation of symptoms following the administration of a high dose of efgartigimod (20 mg/kg, given on days 1 and 5), without the use of any other fast-acting treatment.
This retrospective real-world study demonstrates the effectiveness and safety of efgartigimod in these four MuSK-Ab-positive, female Asian patients with exacerbation of MG, as well as in patients experiencing MC. It is important to note that efgartigimod should not be viewed as a substitute for foundational immunotherapy; rather, it is intended as a rescue treatment during exacerbations and as an adjunctive therapy in the context of long-term immunotherapy. This non-invasive approach has the potential to become another treatment option for MuSK-Ab-positive MG patients.
肌肉特异性激酶抗体（以下简称MuSK - Ab）阳性患者在所有重症肌无力（MG）病例中的患病率为5 - 8%。目前，艾加莫德在一项III期临床试验中已显示出对MuSK - Ab阳性MG患者具有良好的治疗效果。然而，III期临床试验往往会排除病情加重的MG患者，并且关于艾加莫德在MuSK - Ab阳性重症肌无力危象（MC）患者中的疗效，仅有少数真实世界研究。这项回顾性真实世界研究旨在探讨艾加莫德在病情加重的MuSK - Ab阳性MG患者中的疗效。
我们回顾了中山大学附属第一医院4例病情加重的MuSK - Ab阳性MG患者接受艾加莫德治疗的临床资料，其中包括2例MC患者。所有患者均于2023年9月至2024年5月期间入院。大多数患者同时接受利妥昔单抗治疗。
每位患者均完成了一个周期的艾加莫德治疗。首次给药后，4例患者的重症肌无力日常生活活动（MG - ADL）评分出现了具有临床意义的下降（较基线降低超过4分），并且在一个周期的艾加莫德治疗后，所有患者的IgG水平均有所下降。在安全性方面，所有患者均未出现任何明显的不良反应。在最后一次随访时，所有患者在首次给予艾加莫德治疗8.75 ± 5.56周后均达到了最小症状表现状态（MG - ADL评分为0或1）。本文介绍了1例患者的病例，该患者在给予高剂量艾加莫德（20 mg/kg，于第1天和第5天给药）后症状迅速缓解，且未使用任何其他速效治疗。
这项回顾性真实世界研究证明了艾加莫德在这4例病情加重的MuSK - Ab阳性亚洲女性MG患者以及MC患者中的有效性和安全性。需要注意的是，不应将艾加莫德视为基础免疫治疗的替代品；相反，它旨在作为病情加重期间的抢救治疗以及长期免疫治疗背景下的辅助治疗。这种非侵入性方法有可能成为MuSK - Ab阳性MG患者的另一种治疗选择。
