文献类型：期刊文献

中文题名：艾加莫德治疗全身型重症肌无力有效性及安全性的Meta分析

英文题名：Efficacy and safety of efgartigimod in treatment of generalized myasthenia gravis:a meta-analysis

作者：袁曼[1,2];阚鸿姣[1,2];吴倩[3];陈云娜[2];赵开杰[1,2];孙秀亭[1,2];张晓燕[2]

第一作者：袁曼

机构：[1]中国人民解放军联勤保障部队第九四〇医院神经内科,甘肃兰州730050;[2]甘肃中医药大学第一临床学院,甘肃兰州730000;[3]山东省菏泽市立医院神经内科,山东菏泽274009

第一机构：中国人民解放军联勤保障部队第九四〇医院神经内科,甘肃兰州730050

年份：2025

卷号：44

期号：5

起止页码：368

中文期刊名：中国新药与临床杂志

外文期刊名：Chinese Journal of New Drugs and Clinical Remedies

收录：;北大核心:【北大核心2023】；

基金：甘肃省科技厅自然科学基金项目(21JR11RA003)。

语种：中文

中文关键词：艾加莫德;重症肌无力;Meta分析;疗效;安全性

外文关键词：efgartigimod;myasthenia gravis;meta-analysis;efficacy;safety

摘要：目的系统评价静脉注射艾加莫德治疗全身型重症肌无力(gMG)的临床疗效和安全性。方法在中国知网、万方、维普、中国临床注册中心、Clinical Trials.gov、PubMed、Cochrane Library、Embase、Web of Science等数据库中检索关于静脉注射艾加莫德治疗gMG的相关文献,检索时限为数据库建立至2024年8月,采用RevMan 5.3、StataMP 18.0软件进行Meta分析。结果在单臂研究中,乙酰胆碱受体抗体阳性gMG患者MG日常生活质量评分(MG-ADL)(MD=-4.25,95%CI:-5.32~-3.19,P<0.00001)及MG定量(QMG)评分(MD=-4.50,95%CI:-5.46~-3.53,P<0.00001)较基线显著改善,QMG改善率、最小症状表达(MSE)发生率分别为86%(95%CI:69%~97%)、32%(95%CI:13%~55%);gMG患者MG-ADL改善率及MG-ADL应答率分别为83%(95%CI:71%~93%)、68%(95%CI:50%~84%),不良反应发生率为29%(95%CI:4%~65%,P=0.01)。在随机对照研究中,gMG患者MG-ADL改善率(RR=1.83,95%CI:1.43~2.36,P<0.0001)及MG-ADL应答率(RR=2.37,95%CI:1.61~3.47,P<0.0001)明显高于安慰剂组,未见明显不良事件发生(RR=0.91,95%CI:0.77~1.06)。结论静脉注射艾加莫德可显著改善gMG患者的MG-ADL和QMG评分,从而有效缓解临床症状并减轻疾病负担。尽管在临床试验中未观察到显著不良事件发生,但在实际临床应用中仍需对潜在的不良反应保持警惕。
AIM To systematically review the clinical efficacy and safety of efgartigimod administered intravenously in patients with generalized myasthenia gravis(gMG).METHODS The clinical trials published at CNKI,Wanfang,VIP,Chinese Clinical trial Register,Clinical Trials.gov,PubMed,and the Cochrane Library,Embase,Web of Science from their establishment to August 2024 were searched,and the data were analysed using Review Manager 5.3 and Stata MP 18.0 software.RESULTS In the single-arm studies,clinically significant improvements in the myasthenia gravis-activities of daily living(MG-ADL)score(MD=-4.25,95%CI:-5.32 to-3.19,P<0.00001)and the quantitative myasthenia gravis(QMG)score(MD=-4.50,95%CI:-5.46 to-3.53,P<0.00001)were demonstrated in patients with acetylcholine receptor antibodypositive gMG.The rates of QMG improvement and minimal symptom expression(MSE)were 86%(95%CI:69%to 97%)and 32%(95%CI:13%to 55%),respectively.The incidence rate of MG-ADL improvement was 83%(95%CI:71%to 93%),and the proportion of MG-ADL responders was 68%(95%CI:50%to 84%).The incidence of adverse events was 29%(95%CI:4%to 65%,P=0.01).In randomized controlled trials,the incidence rate of MG-ADL improvement(RR=1.83,95%CI:1.43 to 2.36,P<0.0001)and the proportion of MG-ADL responders(RR=2.37,95%CI:1.61 to 3.47,P<0.0001)in gMG patients were significantly higher compared to the placebo group.No significant increase in adverse events was observed(RR=0.91,95%CI:0.77 to 1.06).CONCLUSION Intravenous administration of efgartigimod significantly enhances the MG-ADL and QMG scores in patients with gMG,thereby effectively alleviating clinical symptoms and reducing disease burden.While no significant increase in adverse events was observed in clinical trials,vigilance for potential adverse reactions remains important in real-world clinical practice.