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低剂量利妥昔单抗治疗难治性重症肌无力的长期疗效和安全性。

Long-Term Efficacy and Safety of Low-Dose Rituximab in Patients with Refractory Myasthenia Gravis.

机构信息

Department of Neurology, Peking University First Hospital, Beijing, China,

Department of Neurology, Peking University First Hospital, Beijing, China.

出版信息

Eur Neurol. 2023;86(6):387-394. doi: 10.1159/000534336. Epub 2023 Sep 30.

Abstract

INTRODUCTION

Rituximab is a monoclonal chimeric antibody against CD20+ B cells. We aimed to assess the long-term efficacy and safety of CD20+ B cell-guided treatment with low-dose rituximab in refractory myasthenia gravis patients.

METHODS

Patients with refractory myasthenia gravis treated with rituximab for more than 2 years were included. Rituximab was administered when CD20+ B cells were greater than 1%. We analysed the efficacy of rituximab, treatment interval, side effects, prognosis, and treatment course.

RESULTS

A total of 22 patients were included. All patients received 2-12 doses of rituximab, and the median follow-up time was 48.5 months. The scores of the Myasthenia Gravis Activities of Daily Living and Myasthenia Gravis Composite were significantly lower than those at baseline (p < 0.05). MGFA-PIS was significantly improved in 21 (95.45%) patients and 14 (63.64%) patients have reached MGFA-PIS minimal manifestations. The average daily dose of prednisone and pyridostigmine bromide and the proportion of immunosuppressants were significantly lower (p < 0.05). Seven patients suffered from 14 worsenings. Eight patients terminated rituximab due to good efficacy. Most patients tolerated rituximab well, although 1 patient had opportunistic infection and hypogammaglobulinemia, 1 patient had an intracranial mass.

CONCLUSION

Long-term CD20+ B-cell-guided low-dose rituximab showed good efficacy and tolerance in patients with refractory myasthenia gravis.

摘要

简介

利妥昔单抗是一种针对 CD20+B 细胞的嵌合单克隆抗体。我们旨在评估在难治性重症肌无力患者中使用低剂量利妥昔单抗指导的 CD20+B 细胞治疗的长期疗效和安全性。

方法

纳入接受利妥昔单抗治疗超过 2 年的难治性重症肌无力患者。当 CD20+B 细胞大于 1%时,给予利妥昔单抗。我们分析了利妥昔单抗的疗效、治疗间隔、副作用、预后和治疗过程。

结果

共纳入 22 例患者。所有患者均接受了 2-12 剂利妥昔单抗治疗,中位随访时间为 48.5 个月。重症肌无力日常生活活动评分和重症肌无力综合评分均显著低于基线(p < 0.05)。21 例(95.45%)患者的 MGFA-PIS 显著改善,14 例(63.64%)患者达到 MGFA-PIS 最小表现。泼尼松和溴吡斯的明的平均日剂量和免疫抑制剂的比例显著降低(p < 0.05)。7 例患者发生 14 次恶化。8 例患者因疗效好而终止利妥昔单抗治疗。大多数患者对利妥昔单抗耐受良好,尽管 1 例发生机会性感染和低丙种球蛋白血症,1 例发生颅内肿块。

结论

长期 CD20+B 细胞指导的低剂量利妥昔单抗在难治性重症肌无力患者中显示出良好的疗效和耐受性。

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