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利妥昔单抗治疗全身型重症肌无力:临床实践经验

[Rituximab for the treatment of generalised myasthenia gravis: experience in clinical practice].

作者信息

Martínez-Monte E, Gascón-Giménez F, Domínguez-Morán J A, Láinez-Andres J M

机构信息

Hospital Clínico Universitario de Valencia, Valencia, España.

出版信息

Rev Neurol. 2021 Nov 16;73(12):416-420. doi: 10.33588/rn.7312.2021166.

Abstract

INTRODUCTION AND AIMS

Rituximab (RTX) is an anti-CD20 monoclonal antibody that has been used in cases of refractory myasthenia gravis (MG). The aim of this work is to analyse the efficacy and safety of RTX in MG in real clinical practice in a tertiary hospital.

PATIENTS AND METHODS

A retrospective study was conducted with patients with MG treated with RTX in our centre from March 2014 to September 2020. Demographic and serological data, together with information about previous immunomodulatory treatment, clinical response and adverse effects are collected.

RESULTS

Twenty patients with MG - 100% generalised: 70% late-onset MG (LOMG) and 30% early-onset MG (EOMG) - were given RTX (mean age: 66.8 years; 70% male). A total of 90% are seropositive, 16 of them with positive anti-acetylcholine receptor antibodies and two with positive muscle-specific tyrosine kinase (anti-MuSK) antibodies. All had failed previous treatments: 100% with steroids, 100% with intravenous immunoglobulins and/or plasmapheresis, 55% with other immunosuppressants (25% with one previous immunosuppressant, 10% with two, 15% with three and 5% with four) and 35% with thymectomy. After RTX, 75% of patients showed a clinical response (12 patients with complete remission and the possibility of steroid withdrawal without recurrence; and three patients with partial remission and the possible reduction of steroid dosage) and 25% therapeutic failure; in all these cases RTX was withdrawn. All the anti-MuSK+ patients (100%) and 92.8% of the LOMG patients responded to RTX, while 66% of EOMG patients failed. Only three patients reported adverse effects, all of which were mild and did not require RTX withdrawal.

CONCLUSION

In our experience, rituximab is a safe and effective treatment in aggressive generalised MG with anti-MuSK or late-onset MG (LOMG).

摘要

引言与目的

利妥昔单抗(RTX)是一种抗CD20单克隆抗体,已用于难治性重症肌无力(MG)病例。本研究旨在分析在一家三级医院的实际临床实践中RTX治疗MG的疗效和安全性。

患者与方法

对2014年3月至2020年9月在我院接受RTX治疗的MG患者进行回顾性研究。收集人口统计学和血清学数据,以及既往免疫调节治疗、临床反应和不良反应的信息。

结果

20例MG患者——100%为全身性:70%为晚发型MG(LOMG),30%为早发型MG(EOMG)——接受了RTX治疗(平均年龄:66.8岁;70%为男性)。90%的患者血清学呈阳性,其中16例抗乙酰胆碱受体抗体阳性,2例肌肉特异性酪氨酸激酶(抗MuSK)抗体阳性。所有患者既往治疗均失败:100%使用过类固醇,100%使用过静脉注射免疫球蛋白和/或血浆置换,55%使用过其他免疫抑制剂(25%使用过一种既往免疫抑制剂,10%使用过两种,15%使用过三种,5%使用过四种),35%接受过胸腺切除术。RTX治疗后,75%的患者有临床反应(12例完全缓解,有可能停用类固醇且无复发;3例部分缓解,有可能减少类固醇剂量),25%治疗失败;在所有这些情况下均停用了RTX。所有抗MuSK阳性患者(100%)和92.8%的LOMG患者对RTX有反应,而66%的EOMG患者治疗失败。只有3例患者报告了不良反应,均为轻度,无需停用RTX。

结论

根据我们的经验,利妥昔单抗在侵袭性全身性抗MuSK MG或晚发型MG(LOMG)中是一种安全有效的治疗方法。

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