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霉酚酸酯作为重症肌无力二线免疫抑制剂的长期前瞻性开放标签研究。

Mycophenolate mofetil as second line immunosuppressant in Myasthenia gravis--a long-term prospective open-label study.

作者信息

Hanisch F, Wendt M, Zierz S

机构信息

Klinik und Poliklinik für Neurologie, Martin-Luther-Universität Halle-Wittenberg, Ernst-Grube Str. 40, 06097 Halle/Saale, Germany.

出版信息

Eur J Med Res. 2009 Aug 12;14(8):364-6. doi: 10.1186/2047-783x-14-8-364.

DOI:10.1186/2047-783x-14-8-364
PMID:19666397
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3352168/
Abstract

BACKGROUND

The preferred immunosuppressive drug for long term treatment of myasthenia gravis (MG) is azathioprine (AZA). Mycophenolate mofetil (MMF) was suggested as an effective and safe second line alternative to AZA.

METHODS

In a prospective open-label study, 11 patients with acetylcholine receptor antibody (AChR-ab) positive MG (n=4 ocular MG, n=7 generalized MG) were treated with MMF which replaced AZA. Reasons for the change of immunosuppressant therapy were side effects (n=9) or unresponsiveness under AZA (n=3).

RESULTS

Mean duration of MMF treatment was 16.9 months (6-46 months). During MMF treatment AZA side effects resolved in 8/9 patients, concomitant therapy could be discontinued in 4 patients and reduced in 5 patients, and 5 patients remitted and 3 remained in remission. One MMF-refractory patient required add-on IVIG therapy and another with ocular MG showed signs of generalization after 20 months of MG treatment. One patient was diagnosed with bronchial carcinoma after 10 months of MMF treatment.

CONCLUSION

Due to its favourable spectrum of side effects compared to AZA MMF might serve as a second-line immunosuppressant in those MG patients who have not tolerated AZA.

摘要

背景

硫唑嘌呤(AZA)是重症肌无力(MG)长期治疗的首选免疫抑制药物。霉酚酸酯(MMF)被认为是一种有效且安全的AZA二线替代药物。

方法

在一项前瞻性开放标签研究中,11例乙酰胆碱受体抗体(AChR-ab)阳性的MG患者(4例眼肌型MG,7例全身型MG)接受了MMF治疗,MMF替代了AZA。免疫抑制治疗改变的原因是副作用(9例)或对AZA无反应(3例)。

结果

MMF治疗的平均持续时间为16.9个月(6 - 46个月)。在MMF治疗期间,9例患者中有8例AZA副作用得到缓解,4例患者可停用伴随治疗,5例患者伴随治疗减少,5例患者病情缓解,3例患者持续缓解。1例MMF难治性患者需要加用静脉注射免疫球蛋白(IVIG)治疗,另1例眼肌型MG患者在MG治疗20个月后出现全身症状。1例患者在MMF治疗10个月后被诊断为支气管癌。

结论

与AZA相比,MMF副作用谱较好,对于那些不能耐受AZA的MG患者,MMF可作为二线免疫抑制剂。

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本文引用的文献

1
A trial of mycophenolate mofetil with prednisone as initial immunotherapy in myasthenia gravis.霉酚酸酯联合泼尼松作为重症肌无力初始免疫治疗的试验。
Neurology. 2008 Aug 5;71(6):394-9. doi: 10.1212/01.wnl.0000312373.67493.7f. Epub 2008 Apr 23.
2
An international, phase III, randomized trial of mycophenolate mofetil in myasthenia gravis.霉酚酸酯治疗重症肌无力的一项国际III期随机试验。
Neurology. 2008 Aug 5;71(6):400-6. doi: 10.1212/01.wnl.0000312374.95186.cc. Epub 2008 Apr 23.
3
Primary CNS lymphoma complicating treatment of myasthenia gravis with mycophenolate mofetil.霉酚酸酯治疗重症肌无力并发原发性中枢神经系统淋巴瘤。
Neurology. 2005 Aug 23;65(4):639-41. doi: 10.1212/01.wnl.0000173031.56429.04.
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Mycophenolate mofetil for myasthenia gravis: a double-blind, placebo-controlled pilot study.霉酚酸酯治疗重症肌无力:一项双盲、安慰剂对照的初步研究。
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Successful treatment of a patient with severe refractory myasthenia gravis using mycophenolate mofetil.使用霉酚酸酯成功治疗一名重度难治性重症肌无力患者。
Neurology. 1998 Sep;51(3):912-3. doi: 10.1212/wnl.51.3.912-a.