Silvestri Nicholas J
Department of Neurology, University at Buffalo Jacobs School of Medicine and Biomedical Sciences, New York, USA.
Muscle Nerve. 2025 Mar;71(3):422-428. doi: 10.1002/mus.28334. Epub 2025 Jan 2.
INTRODUCTION/AIMS: Neonatal Fc receptor (FcRn) inhibitors represent a promising treatment option for patients with generalized myasthenia gravis (gMG); however, data on clinical use are limited. The aim of this report is to describe one center's approach to efgartigimod dosing in patients with gMG.
Medical records of patients with acetylcholine receptor antibody-positive (AChR-Ab+) gMG whose symptoms were not adequately controlled by oral medications and/or intravenous immunoglobulin who received efgartigimod between January 2022 and January 2024 were retrospectively evaluated. The first three efgartigimod cycles (10 mg/kg IV) were initiated at fixed intervals (4 once-weekly infusions, with 4 weeks between cycles). After the third cycle, initiation of subsequent treatment cycles and time between cycles were determined individually by clinical evaluation. Effectiveness was measured by the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale. Adverse events and changes to concomitant therapies were monitored.
Nineteen patients were included and received a mean of 4.4 efgartigimod cycles, including two patients who discontinued after two cycles. All patients exhibited a clinically meaningful improvement in MG-ADL total score from baseline to the end of the last cycle, with a mean improvement of 5.8 points. Seven (37%) patients achieved minimal symptom expression (MSE, MG-ADL 0-1). From baseline to the end of the last cycle, daily prednisone dose was decreased or it was discontinued, while two patients initiated prednisone. Efgartigimod was generally well tolerated.
This approach to efgartigimod dosing resulted in substantial MG-ADL score improvement in these patients with AChR-Ab+ gMG as well as reduced daily dose and/or discontinuation of concomitant corticosteroids.
引言/目的：新生儿Fc受体（FcRn）抑制剂是全身型重症肌无力（gMG）患者一种有前景的治疗选择；然而，临床应用数据有限。本报告的目的是描述一个中心对gMG患者使用艾加莫德的给药方法。
回顾性评估2022年1月至2024年1月期间接受艾加莫德治疗的乙酰胆碱受体抗体阳性（AChR-Ab+）gMG患者的病历，这些患者的症状未通过口服药物和/或静脉注射免疫球蛋白得到充分控制。前三个艾加莫德疗程（10mg/kg静脉注射）以固定间隔开始（4次每周一次输注，疗程之间间隔4周）。第三个疗程后，后续治疗疗程的开始和疗程之间的时间由临床评估单独确定。疗效通过重症肌无力日常生活活动（MG-ADL）量表衡量。监测不良事件和伴随治疗的变化。
纳入19例患者，平均接受4.4个艾加莫德疗程，包括2例在两个疗程后停药的患者。所有患者从基线到最后一个疗程结束时MG-ADL总分均有临床意义的改善，平均改善5.8分。7例（37%）患者达到最小症状表现（MSE，MG-ADL 0-1）。从基线到最后一个疗程结束，每日泼尼松剂量减少或停用，同时有2例患者开始使用泼尼松。艾加莫德总体耐受性良好。
这种艾加莫德给药方法使这些AChR-Ab+ gMG患者的MG-ADL评分有显著改善，同时减少了每日剂量和/或停用了伴随的皮质类固醇。
