Dermatology Department, Faculty of Medicine, Cairo University , Cairo , Egypt.
J Dermatolog Treat. 2013 Dec;24(6):422-6. doi: 10.3109/09546634.2013.768327. Epub 2013 May 21.
No effective treatment has been found for epidermolysis bullosa dystrophica (EBD).
To evaluate the efficacy and safety mycophenolate mofetil (MMF) in treating EBD.
This randomized controlled double-blinded study included 35 patients with severe generalized EBD. Patients were randomly divided into two groups: group I (18 patients) received cyclosporine therapy (5 mg/kg/day) and group II (17 patients) received MMF therapy (500-1500 mg/day). Clinical assessment was made weekly for 3 months from the start of the treatment. Patients were assessed by measuring the extent of the disease, the % of improvement, assessing the number of new blister formation and the time of complete healing of new blisters. Side effects were recorded when detected.
The % of improvement in the disease extent was statistically significantly higher (p = 0.009) in group I (mean ± SD: 59.21 ± 22.676) than in group II (mean ± SD: 44.03 ± 25.71). As regards the number of new blisters and the rate of healing of blisters, there was no statistically significant difference between both groups (p = 0.693 and 0.404, respectively). No serious side effects were reported.
MMF seems to be a good therapeutic option for the long-term treatment of EBD, it can be a good alternative for patients who cannot tolerate cyclosporine.
目前尚未找到治疗营养不良型大疱性表皮松解症（EBD）的有效方法。
评估霉酚酸酯（MMF）治疗 EBD 的疗效和安全性。
本随机对照双盲研究纳入了 35 例严重泛发性 EBD 患者。患者随机分为两组：I 组（18 例）接受环孢素治疗（5mg/kg/天），II 组（17 例）接受 MMF 治疗（500-1500mg/天）。从治疗开始后的 3 个月内每周进行临床评估。通过测量疾病程度、改善百分比、评估新水疱形成数量和新水疱完全愈合时间来评估患者。当发现不良反应时记录下来。
I 组（平均±标准差：59.21±22.676）疾病程度改善的百分比明显高于 II 组（平均±标准差：44.03±25.71，p=0.009）。关于新水疱的数量和水疱愈合的速度，两组之间没有统计学上的显著差异（分别为 p=0.693 和 0.404）。未报告严重不良反应。
MMF 似乎是治疗 EBD 的一种较好的长期治疗选择，对于不能耐受环孢素的患者来说，它可能是一种较好的替代选择。
