Department of Neurosurgery and Brain and Nerve Research Laboratory, The First Affiliated Hospital of Soochow University, 188 Shizi Street, Suzhou, 215006, Jiangsu Province, China.
Department of Neurology, The Affiliated Suzhou Hospital of Nanjing Medical University, Suzhou Municipal Hospital, Suzhou, Jiangsu Province, China.
J Neurol. 2024 May;271(5):2298-2308. doi: 10.1007/s00415-024-12247-x. Epub 2024 Mar 3.
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.
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.
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.
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.
重症肌无力(MG)是一种自身免疫性疾病,可导致局部或全身肌肉无力。补体抑制剂和针对新生儿 Fc 受体(FcRn)以阻断 IgG 循环是两种新的、成功的机制。
系统检索了 PubMed、EMBASE、Cochrane 图书馆和 ClinicalTrials.gov,以确定截至 2023 年 5 月 18 日之前发表的相关研究。使用 Review Manager 5.3 软件评估数据。
我们从六项随机对照试验(RCT)中汇总了 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量表(MG-ADL)(MD=-1.69[-2.35, -1.03],P<0.00001)、MG-ADL 应答者(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)、重症肌无力综合量表(MGC)(MD=-2.97[-4.27, -1.67],P<0.00001)、15 项修订版重症肌无力生活质量量表(MGQoL15r)(MD=-2.52[-3.54, -1.50],P<0.00001)方面更有效,且不增加安全性风险。亚组分析表明,efgartigimod 在 MG-ADL 应答者中比安慰剂更有效。Rozanolixizumab 除了 QMG 外比安慰剂更有效,batoclimab 除了 MG-ADL 应答者外比安慰剂更有效。Nipocalizumab 在所有结局中均未显示出令人满意的疗效。除了 rozanolixizumab 外,所有药物的安全性与安慰剂相当。
FcRn 抑制剂在 MG 患者中具有良好的疗效和安全性。其中,efgartigimod 和 nipocalimab 有效且不会增加安全性风险。Rozanolixizumab 虽然疗效优越,但不良反应发生率增加。目前的证据表明 nipocalimab 对 MG 患者无效。