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他克莫司治疗难治性重症肌无力的疗效与安全性:36例患者报告

[Therapeutic efficacy and safety of tacrolimus for intractable myasthenia gravis: a report of 36 patients].

作者信息

Feng Hui-yu, Liu Wei-bin, Qiu Li, Huang Xin, Huang Ru-xun

机构信息

Department of Neurology, Sun Yat-sen University, Guangzhou, China.

出版信息

Zhonghua Yi Xue Za Zhi. 2011 Dec 6;91(45):3190-2.

Abstract

OBJECTIVE

To examine the efficacies and adverse events of low-dose tacrolimus in intractable myasthenia gravis (MG) patients during a long-term follow-up.

METHODS

Tacrolimus was administered at 0.1 mg×kg(-1)×d(-1) to 36 generalized or ocular MG patients at our department from November 2008 to December 2010. The efficacies of tacrolimus were assessed by the myasthenia gravis activities of daily living (MG-ADL) profile and the classification of Myasthenia Gravis Foundation of America (MGFA). And the adverse events of tacrolimus were monitored in each patient.

RESULTS

(1) All patients were followed up for 7 - 23 months. Adverse events occurred in 6 patients (16.67%). (2) The myasthenic symptoms improved up to the levels of MG-ADL and MGFA in 24 patients (66.67%). There was notable statistical significance in the comparison of clinical status at pre- and post-treatment (P = 0.000). (3) The efficacies in patients with generalized MG were better than those with ocular MG (P = 0.032). (4) The average blood trough levels of tacrolimus were lower than the recommended maintenance range from other countries in 24 effective patients.

CONCLUSION

The administration of tacrolimus induces symptomatic improvements in MG patients especially in generalized type. And the adverse events should be closely monitored.

摘要

目的

在长期随访中研究低剂量他克莫司治疗难治性重症肌无力(MG)患者的疗效及不良事件。

方法

2008年11月至2010年12月,对我科36例全身型或眼肌型MG患者给予他克莫司0.1mg×kg⁻¹×d⁻¹治疗。通过重症肌无力日常生活活动(MG-ADL)量表及美国重症肌无力基金会(MGFA)分类评估他克莫司的疗效。对每位患者监测他克莫司的不良事件。

结果

(1)所有患者随访7 - 23个月。6例患者(16.67%)出现不良事件。(2)24例患者(66.67%)肌无力症状改善至MG-ADL及MGFA水平。治疗前后临床状态比较有显著统计学意义(P = 0.000)。(3)全身型MG患者的疗效优于眼肌型MG患者(P = 0.032)。(4)24例有效患者他克莫司的平均血药谷浓度低于其他国家推荐的维持范围。

结论

他克莫司治疗可使MG患者尤其是全身型患者症状改善。应密切监测不良事件。

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