背景:重症肌无力(MG)是一种自身免疫性疾病,可引起局部或全身肌肉无力。补体抑制剂和靶向新生儿Fc受体(FcRn)以阻断IgG循环是两种新颖且成功的机制。
方法:PubMed,EMBASE,Cochrane图书馆,和ClinicalTrials.gov进行了系统搜索,以确定2023年5月18日之前发表的相关研究。使用ReviewManager5.3软件评估数据。
结果:我们汇集了来自6项随机对照试验(RCT)的532名参与者。与安慰剂相比,FcRn抑制剂在重症肌无力日常生活活动(MG-ADL)中更有效(MD=-1.69[-2.35,-1.03],P<0.00001),MG-ADL响应者(RR=2.01[1.62,2.48],P<0.00001),定量重症肌无力(QMG)(MD=-2.45[-4.35,-0.55],P=0.01),重症肌无力复合物(MGC)(MD=-2.97[-4.27,-1.67],P<0.00001),重症肌无力生活质量(MGQoL15r)的15项修订版(MD=-2.52[-3.54,-1.50],P<0.00001),不会增加安全风险。亚组分析显示,在MG-ADL应答者中,efgartigimod比安慰剂更有效。Rozanolixizumab比安慰剂更有效,除了QMG,除MG-ADL反应者外,batoclimab比安慰剂更有效。Nipocalizumab在所有结果中均未显示出令人满意的疗效。除了rozanolixizumab,所有药物的安全性均低于安慰剂.
结论:FcRn抑制剂对MG患者具有良好的疗效和安全性。其中,efgartigimod和nipocalimab均有效,且不增加安全风险.Rozanolixizumab,尽管其疗效优越,导致不良事件发生率增加。目前的证据并不表明nipocalimab对MG患者有效。
BACKGROUND: Myasthenia gravis (MG) is an autoimmune disease that causes local or generalized muscle weakness. Complement inhibitors and targeting of the neonatal Fc receptor (FcRn) to block IgG cycling are two novel and successful mechanisms.
METHODS: PubMed, EMBASE, the Cochrane Library, and ClinicalTrials.gov were systematically searched to identify relevant studies published before May 18, 2023.
Review Manager 5.3 software was used to assess the data.
RESULTS: We pooled 532 participants from six randomized controlled trials (RCTs). Compared to the placebo, the FcRn inhibitors were more efficacy in Myasthenia Gravis Activities of Daily Living (MG-ADL) (MD = - 1.69 [- 2.35, - 1.03], P < 0.00001), MG-ADL responder (RR = 2.01 [1.62, 2.48], P < 0.00001), Quantitative Myasthenia Gravis (QMG) (MD = - 2.45 [- 4.35, - 0.55], P = 0.01), Myasthenia Gravis Composite (MGC) (MD = - 2.97 [- 4.27, - 1.67], P < 0.00001), 15-item revised version of the Myasthenia Gravis Quality of Life (MGQoL15r) (MD = - 2.52 [- 3.54, - 1.50], P < 0.00001), without increasing the risk of safety. The subgroup analysis showed that efgartigimod was more effective than placebo in MG-ADL responders. Rozanolixizumab was more effective than the placebo except in QMG, and batoclimab was more effective than the placebo except in MG-ADL responder. Nipocalizumab did not show satisfactory efficacy in all outcomes. With the exception of rozanolixizumab, all drugs showed non-inferior safety profiles to placebo.
CONCLUSIONS: FcRn inhibitors have good efficacy and safety in patients with MG. Among them, efgartigimod and nipocalimab were effective without causing an increased safety risk. Rozanolixizumab, despite its superior efficacy, caused an increased incidence of adverse events. Current evidence does not suggest that nipocalimab is effective in patients with MG.