Abstract：
In epidermolysis bullosa simplex (EBS), epithelial structural fragility results in blisters and erosions. Diacerein 1% ointment has been shown to reduce this blistering.
To evaluate the efficacy and safety of diacerein 1% ointment in the treatment of EBS.
A double-blind study of 54 patients with EBS were randomized to diacerein 1% or vehicle ointment once daily. The primary endpoint ( ≥60% reduction in body surface area of EBS) and the key secondary endpoint ( ≥2-point reduction in the Investigator’s Global Assessment) were evaluated at 8 weeks.
There was no difference in the proportion of patients achieving either key efficacy endpoint between the diacerein 1% and vehicle groups (P>0.05). No difference in treatment emergent adverse events were noted between the groups. In post hoc analysis stratified by EBS subtypes, an IGA score of 0 or 1 was reported in 6 of 13 patients with severe EBS in the diacerein group (46.2%), compared with 2 of 13 patients with severe EBS in the vehicle group (15.4%); (relative risk= 3.08, 95% CI = 0.71, 13.4).
Although there was no significant difference in outcomes between the groups, further study may elucidate the effects of diacerein on EBS lesions, especially in patients with severe EBS. Teng J, Paller AS, Bruckner AL, et al. Diacerein 1% ointment for the treatment of epidermolysis bullosa simplex: a randomized, controlled trial. J Drugs Dermatol. 2023;22(6):599-604. doi:10.36849/JDD.7108.
To evaluate the efficacy and safety of diacerein 1% ointment in the treatment of EBS.
A double-blind study of 54 patients with EBS were randomized to diacerein 1% or vehicle ointment once daily. The primary endpoint ( ≥60% reduction in body surface area of EBS) and the key secondary endpoint ( ≥2-point reduction in the Investigator’s Global Assessment) were evaluated at 8 weeks.
There was no difference in the proportion of patients achieving either key efficacy endpoint between the diacerein 1% and vehicle groups (P>0.05). No difference in treatment emergent adverse events were noted between the groups. In post hoc analysis stratified by EBS subtypes, an IGA score of 0 or 1 was reported in 6 of 13 patients with severe EBS in the diacerein group (46.2%), compared with 2 of 13 patients with severe EBS in the vehicle group (15.4%); (relative risk= 3.08, 95% CI = 0.71, 13.4).
Although there was no significant difference in outcomes between the groups, further study may elucidate the effects of diacerein on EBS lesions, especially in patients with severe EBS. Teng J, Paller AS, Bruckner AL, et al. Diacerein 1% ointment for the treatment of epidermolysis bullosa simplex: a randomized, controlled trial. J Drugs Dermatol. 2023;22(6):599-604. doi:10.36849/JDD.7108.
摘要：
背景：在单纯大疱性表皮松解症（EBS）中，上皮结构脆性导致水疱和糜烂。已经证明1%的双醋瑞林软膏可以减少这种起泡。
目的：评价1%双醋瑞因软膏治疗EBS的疗效和安全性。
方法：一项针对54例EBS患者的双盲研究随机分为1％双醋瑞因或载体软膏，每天一次。在8周时评估主要终点(≥EBS体表面积减少60%)和关键次要终点(≥在研究者’s全球评估中减少2点)。
结果：双醋瑞因1%组和溶媒组达到任一关键疗效终点的患者比例无差异（P>0.05）。两组之间在治疗紧急不良事件方面没有差异。在按EBS亚型分层的事后分析中，双醋瑞瑞因组13例严重EBS患者中有6例IGA评分为0或1(46.2%),与赋形剂组13例重度EBS患者中的2例(15.4%)相比;(相对危险度=3.08,95%CI=0.71,13.4).
结论：虽然两组间的结果没有显著差异，进一步的研究可能会阐明双醋瑞因对EBS病变的影响，尤其是严重EBS患者。滕杰,帕勒AS,布鲁克纳AL,etal.1％双醋瑞因软膏治疗单纯大疱性表皮松解症：随机，对照试验。J药物Dermatol.2023年；22（6）：599-604。doi:10.36849/JDD.7108.
目的：评价1%双醋瑞因软膏治疗EBS的疗效和安全性。
方法：一项针对54例EBS患者的双盲研究随机分为1％双醋瑞因或载体软膏，每天一次。在8周时评估主要终点(≥EBS体表面积减少60%)和关键次要终点(≥在研究者’s全球评估中减少2点)。
结果：双醋瑞因1%组和溶媒组达到任一关键疗效终点的患者比例无差异（P>0.05）。两组之间在治疗紧急不良事件方面没有差异。在按EBS亚型分层的事后分析中，双醋瑞瑞因组13例严重EBS患者中有6例IGA评分为0或1(46.2%),与赋形剂组13例重度EBS患者中的2例(15.4%)相比;(相对危险度=3.08,95%CI=0.71,13.4).
结论：虽然两组间的结果没有显著差异，进一步的研究可能会阐明双醋瑞因对EBS病变的影响，尤其是严重EBS患者。滕杰,帕勒AS,布鲁克纳AL,etal.1％双醋瑞因软膏治疗单纯大疱性表皮松解症：随机，对照试验。J药物Dermatol.2023年；22（6）：599-604。doi:10.36849/JDD.7108.
