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利妥昔单抗治疗重症肌无力:现状如何?

Rituximab in Myasthenia Gravis - Where do we stand?

机构信息

Division of Neurology, Dept of Medicine, University of Alberta Hospital, Edmonton, Canada.

出版信息

Expert Opin Biol Ther. 2021 Aug;21(8):1013-1023. doi: 10.1080/14712598.2021.1889509. Epub 2021 Apr 2.

Abstract

: Myasthenia gravis (MG) is an antibody-mediated disease with diverse serology and clinical presentation. Currently, MG is managed by untargeted immunomodulatory agents. About 15% patients are refractory to these therapies. Several novel and targeted treatments are on the horizon. Rituximab, a monoclonal antibody, is reported to be highly effective with widespread oof-label usage in MG, particularly in patients with antibody against muscle-specific kinase or refractory disease. However, a recent trial showed negative results. Compared to conventional oral immunosuppressive therapies used in MG, Rituximab has several benefits. Regular hematological monitoring is not required though serious side effects can occur. Current status of Rituximab in MG and newer immunosuppressants is discussed.: Biologic features, clinical effectiveness, safety profile, and newer preparations of Rituximab.: Rituximab provides a promising option for management of MG, particularly in patients with muscle-specific kinase antibodies or those with refractory disease. Several knowledge gaps remain due to scarcity of data from randomized controlled studies. Despite lack of regulatory approval Rituximab has found widespread usage in MG. Large, well-designed studies are needed to assess the comparative efficacy of Rituximab and its optimal regimen in MG.

摘要

重症肌无力(MG)是一种抗体介导的疾病,具有多种血清学和临床表现。目前,MG 通过非靶向免疫调节剂进行治疗。大约 15%的患者对这些治疗方法有抗药性。几种新型的靶向治疗方法正在出现。利妥昔单抗是一种单克隆抗体,在 MG 中具有广泛的超适应证使用,尤其是在针对肌肉特异性激酶或难治性疾病的抗体患者中,效果显著。然而,最近的一项试验结果为阴性。与 MG 中常规的口服免疫抑制疗法相比,利妥昔单抗具有许多优势。尽管可能会出现严重的副作用,但不需要常规进行血液学监测。本文讨论了利妥昔单抗在 MG 中的现状和更新的免疫抑制剂。:生物特征、临床疗效、安全性概况和利妥昔单抗的新制剂。:利妥昔单抗为 MG 的治疗提供了一种有前途的选择,特别是对于肌肉特异性激酶抗体阳性或难治性疾病的患者。由于缺乏随机对照研究的数据,仍存在一些知识空白。尽管缺乏监管批准,但利妥昔单抗在 MG 中的应用已广泛普及。需要进行大型、精心设计的研究来评估利妥昔单抗及其在 MG 中的最佳方案的相对疗效。

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