Department of Neurology, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jing Wu Road, Huaiyin District, Jinan 250021, Shandong, China.
Department of Neurology, Shandong Provincial Hospital Affiliated to Shandong First Medical University, Jing Wu Road, Huaiyin District, Jinan 250021, Shandong, China.
J Neuroimmunol. 2022 Jan 15;362:577767. doi: 10.1016/j.jneuroim.2021.577767. Epub 2021 Nov 18.
Eculizumab is a recombinant humanized monoclonal antibody that targets the complement protein C5, inhibiting its cleavage into C5a and C5b and ultimately preventing the formation of C5b-9 membrane attack complex (MACs), thereby protecting the neuromuscular junction from the damage of complement activation. In 2017, eculizumab became the second FDA-approved medication for AchR-positive generalized myasthenia gravis (gMG) patients based on the successful results of a randomized, double-blinded, placebo-controlled, phase 2, phase 3 study (the REGAIN trial) and its open-label extension study. Despite the efficacy of eculizumab in treating AchR antibody-positive refractory gMG was demonstrated in the REGAIN study, there is few information on its efficacy in other subgroup of MG patients including seronegative MG, thymoma-associated MG and MG crisis. This narrative review summarizes current clinical studies of eculizumab in these refractory gMG patients, with a focus on the therapeutic efficacy and tolerability in different subgroup of MG.
依库珠单抗是一种针对补体蛋白 C5 的重组人源化单克隆抗体，可抑制 C5 的裂解为 C5a 和 C5b，从而阻止 C5b-9 膜攻击复合物（MAC）的形成，进而保护神经肌肉接头免受补体激活的损害。2017 年，基于一项随机、双盲、安慰剂对照、2 期和 3 期研究（REGAIN 试验）及其开放标签扩展研究的成功结果，依库珠单抗成为美国食品药品监督管理局（FDA）批准的第二种用于治疗乙酰胆碱受体阳性全身型重症肌无力（gMG）的药物。尽管 REGAIN 研究表明依库珠单抗治疗乙酰胆碱受体抗体阳性难治性 gMG 有效，但关于其在其他 MG 患者亚组中的疗效的信息较少，包括血清阴性 MG、胸腺瘤相关 MG 和 MG 危象。本综述总结了依库珠单抗在这些难治性 gMG 患者中的临床研究现状，重点关注不同 MG 亚组患者的治疗效果和耐受性。
