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霉酚酸酯治疗儿童重度特应性皮炎:14例患者的经验

Mycophenolate mofetil for severe childhood atopic dermatitis: experience in 14 patients.

作者信息

Heller M, Shin H T, Orlow S J, Schaffer J V

机构信息

Ronald O. Perelman Department of Dermatology, New York University School of Medicine, New York, NY 10016, USA.

出版信息

Br J Dermatol. 2007 Jul;157(1):127-32. doi: 10.1111/j.1365-2133.2007.07947.x. Epub 2007 May 8.

Abstract

BACKGROUND

Reports of successful treatment of atopic dermatitis (AD) with mycophenolate mofetil (MMF) have thus far been limited to adults. Considering that the condition typically develops during childhood and is most active during this period, MMF would represent a valuable addition to the therapeutic armamentarium for paediatric AD.

OBJECTIVES

To evaluate the safety and efficacy of MMF in the treatment of severe childhood AD.

METHODS

A retrospective analysis was performed of all children treated with MMF as systemic monotherapy for severe, recalcitrant AD between August 2003 and August 2006 at New York University Medical Center. Fourteen patients meeting these criteria were identified.

RESULTS

Four patients (29%) achieved complete clearance, four (29%) had > 90% improvement (almost complete), five (35%) had 60-90% improvement and one (7%) failed to respond. Initial responses occurred within 8 weeks (mean 4 weeks), and maximal effects were attained after 8-12 weeks (mean 9 weeks) at MMF doses of 40-50 mg kg(-1) daily in younger children and 30-40 mg kg(-1) daily in adolescents. The medication was well tolerated in all patients, with no infectious complications or development of leucopenia, anaemia, thrombocytopenia or elevated aminotransferases.

CONCLUSIONS

This retrospective case series demonstrates that MMF can be a safe and effective treatment for severe, refractory AD in children. MMF represents a promising therapeutic alternative to traditional systemic immunosuppressive agents with less favourable side-effect profiles, and prospective controlled studies are warranted, further to assess its benefits in paediatric AD.

摘要

背景

迄今为止,关于霉酚酸酯(MMF)成功治疗特应性皮炎(AD)的报道仅限于成人。鉴于该病通常在儿童期发病且在此期间最为活跃,MMF将成为小儿AD治疗手段中的一项有价值的补充。

目的

评估MMF治疗重度儿童AD的安全性和有效性。

方法

对2003年8月至2006年8月在纽约大学医学中心接受MMF作为重度、顽固性AD全身单一疗法治疗的所有儿童进行回顾性分析。确定了14名符合这些标准的患者。

结果

4名患者(29%)实现完全清除,4名(29%)改善超过90%(几乎完全清除),5名(35%)改善60% - 90%,1名(7%)无反应。初始反应在8周内出现(平均4周),在年幼儿童每日MMF剂量为40 - 50 mg·kg⁻¹、青少年每日30 - 40 mg·kg⁻¹的情况下,8 - 12周后(平均9周)达到最大效果。所有患者对该药物耐受性良好,未出现感染并发症,也未发生白细胞减少、贫血、血小板减少或转氨酶升高。

结论

这个回顾性病例系列表明,MMF对儿童重度难治性AD可能是一种安全有效的治疗方法。与副作用较大的传统全身免疫抑制剂相比,MMF是一种有前景的治疗选择,有必要进行前瞻性对照研究,进一步评估其在小儿AD中的益处。

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