多周期艾加莫德在实现重症肌无力最小症状表现方面的疗效：一项多中心比较研究。

Efficacy of multi-cycle Efgartigimod in achieving minimal symptom expression in myasthenia gravis: A comparative multi-center study.

作者信息

Chen Jiaxin, Zhu Xiaohuan, Zhou Haihong, Huang Wen, Wu Wei, Chen Chao, Zhao Zhongyan, Zhang Zhengxue, Zhao Yuanqi, Wang Shuangqing, Zhou Yanxia, Hong Liang, Mo Jinping, Lin Xiaowei, Gu Mei, Zhou Jing, Feng Huiyu

机构信息

Department of Neurology, The First Affiliated Hospital of Sun Yat-sen University, Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, Guangzhou, Guangdong 510080, China.

Department of Neurology, Neuromedical Center, The University of Hong Kong- Shenzhen Hospital, Shenzhen 518000, China.

出版信息

Int Immunopharmacol. 2025 May 8;154:114603. doi: 10.1016/j.intimp.2025.114603. Epub 2025 Apr 4.

DOI:10.1016/j.intimp.2025.114603

PMID:40186904

Abstract

OBJECTIVES

Efgartigimod has been approved as an effective and safe treatment for myasthenia gravis (MG). However, real-world experience on multi-cycle efgartigimod treatment and its comparison with Standard of Care (SoC) remain limited. This study aimed to evaluate minimal symptom expression (MSE) as the treatment goal and compared the proportion and time to achieving it between two groups.

METHODS

Patients receiving multi-cycle efgartigimod and contemporaneous counterparts treated with SoC were included. The rate of MSE achievement were compared using Kaplan-Meier analysis and Cox regression. Subgroup analysis focused on efgartigimod group, observing involved muscles and oral medication for further insights.

RESULTS

A total of 76 and 124 MG patients were enrolled in the efgartigimod and SoC groups, respectively. Efgartigimod group demonstrated a higher rate (73.3 % vs. 22.6 %, p < 0.001) and shorter time [0.7 (0.5, 3.1) vs. 3.3 (3, 6.1), months, p < 0.001] to achieving MSE compared to SoC group. Kaplan-Meier analysis revealed efgartigimod group had a higher MSE probability with a median time of 2.27 (95 %CI, 0.70, 4.39) months. MG Patients had a 9.69 fold (95 %CI, 5.54, 16.92) greater chance of achieving MSE compared to SoC group, remaining significant at 9.44 fold (95 %CI, 5.36, 16.60) after adjusting for ADL scores. After treatment, respiratory and bulbar symptoms improved significantly, with average scores from 0.57 ± 0.87 to zero, and 2.62 ± 2.56 to 0.37 ± 0.96. Additionally, the daily dosage of corticosteroid dropped from 20(10, 25) mg to 10(10,20) mg, with only 7 (9.2 %) patients requiring over 20 mg/day.

CONCLUSION

Multi-cycle efgartigimod treatment achieves early MSE more effectively than SoC, serving as a fast-acting therapy for MG.

摘要

目的

艾加莫德已被批准作为治疗重症肌无力（MG）的一种安全有效的药物。然而，关于艾加莫德多周期治疗的真实世界经验及其与标准治疗（SoC）的比较仍然有限。本研究旨在评估将最小症状表现（MSE）作为治疗目标，并比较两组实现该目标的比例和时间。

方法

纳入接受多周期艾加莫德治疗的患者以及同期接受SoC治疗的患者。使用Kaplan-Meier分析和Cox回归比较MSE实现率。亚组分析聚焦于艾加莫德组，观察受累肌肉和口服药物以获取更多见解。

结果

艾加莫德组和SoC组分别纳入了76例和124例MG患者。与SoC组相比，艾加莫德组实现MSE的比例更高（73.3%对22.6%，p<0.001），时间更短[0.7（0.5，3.1）对3.3（3，6.1）个月，p<0.001]。Kaplan-Meier分析显示，艾加莫德组实现MSE的概率更高，中位时间为2.27（95%CI，0.70，4.39）个月。与SoC组相比，MG患者实现MSE的可能性高9.69倍（95%CI，5.54，16.92），在调整日常生活活动（ADL）评分后仍显著高9.44倍（95%CI，5.36，16.60）。治疗后，呼吸和延髓症状显著改善，平均评分从0.57±0.87降至零，从2.62±2.56降至0.37±0.96。此外，皮质类固醇的每日剂量从20（10，25）mg降至10（10，20）mg，只有7例（9.2%）患者需要超过20mg/天。