Diacerein 1% Ointment for the Treatment of Epidermolysis Bullosa Simplex: A Randomized, Controlled Trial

Background: In epidermolysis bullosa simplex (EBS), epithelial structural fragility results in blisters and erosions. Diacerein 1 % ointment has been shown to reduce this blistering. Objective: To evaluate the efficacy and safety of diacerein 1 % ointment in the treatment of EBS. Methods: 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. Results: 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). Conclusions: 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.