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吗替麦考酚酯治疗儿童复发性眼肌型重症肌无力的疗效
陈会,刘平,曾向东,蓝明平,胡文广
0
(电子科技大学医学院附属妇女儿童医院,成都市妇女儿童中心医院,成都 611731)
摘要:
目的:探讨吗替麦考酚酯治疗儿童复发性眼肌型重症肌无力(OMG)的临床疗效及安全性。 方法:分析成都市妇女儿童 中心医院儿童神经内科 2017-2021 年收治的 5 例复发性 OMG 患儿应用吗替麦考酚酯治疗的临床资料及随访情况。 结果:5 例 患儿首次发病年龄 14~28 个月,使用吗替麦考酚酯前病程 21~94 个月,均在接受吗替麦考酚酯治疗前接受过激素、激素+溴吡 斯的明单用或联合治疗,均出现多次(≥2 次)复发。 最后一次复发时急性期均接受口服小剂量泼尼松及吗替麦考酚酯联合治 疗方案,病情稳定后,逐渐减停泼尼松,单用吗替麦考酚酯治疗。 随访时间 18 ~ 28 个月,随访期间无复发。 随访未发现明显药 物不良反应,且伴随激素减停,激素相关副作用逐渐缓解。 结论:吗替麦考酚酯是一种对儿童复发性 OMG 安全、有效的治疗药 物,但仍需大样本量的随机对照试验(RCT)进一步证实。
关键词:  眼肌型重症肌无力  复发  吗替麦考酚酯  儿童
DOI:10.13407/j.cnki.jpp.1672-108X.2023.11.012
基金项目:
Efficacy of Mycophenolate Mofetil in the Treatment of Recurrent Ocular Myasthenia Gravis in Children
Chen Hui, Liu Ping, Zeng Xiangdong, Lan Mingping, Hu Wenguang
(The Affiliated Women’ s and Children’ s Hospital, School of Medicine, University of Electronic Science and Technology of China, Chengdu Women’ s and Children’ s Central Hospital, Chengdu 611731, China)
Abstract:
Objective: To probe into the clinical efficacy and safety of mycophenolate mofetil in the treatment of recurrent ocular myasthenia gravis (OMG) in children. Methods: Clinical data and follow-up of 5 patients with OMG treated with mycophenolate mofetil and admitted into the pediatric neurology department of Chengdu Women’ s and Children’ s Central Hospital from 2017 to 2021 were analyzed. Results: Onset age of 5 children was from 14 to 28 months old, and the course of disease was from 21 to 94 months before application of mycophenolate mofetil. All 5 patients received hormones, hormones + pyridine bromide alone or in combination therapy before receiving mycophenolate mofetil therapy, with multiple (⩾2) recurrences. In the last recurrence, all patients received oral low-dose prednisone and mycophenolate mofetil in the acute stage. After stabilization, prednisone was reduced and mycophenolate mofetil alone was administered. Follow-up was as short as 18 months and as long as 28 months, and there was no recurrence. No significant adverse drug reactions were observed during follow-up, and hormone-related side effects were alleviated with drug withdrawal of hormone. Conclusion: For children with recurrent ocular myasthenia gravis, mycophenolate mofetil may be the safe and effective treatment, which still needs to be further confirmed by a large sample of randomized controlled trials (RCT).
Key words:  ocular myasthenia gravis  recurrence  mycophenolate mofetil  children

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