Background: Epidermolysis bullosa simplex (EBS) is a rare genetic, blistering skin disease for which there is no cure. Treatments that address the pathophysiology of EBS are needed.
Objective: Compare the impact of 1% diacerein cream with placebo in reducing the number of blisters in EBS.
Methods: In a randomized, placebo-controlled, phase 2/3 trial we used a 1% diacerein topical formulation to treat defined skin areas in 17 patients. In a 2-period crossover trial, patients were randomized to either placebo or diacerein for a 4-week treatment and a 3-month follow-up in period 1. After a washout, patients were crossed over during period 2. The prespecified primary end point was the proportion of patients with a reduction of number of blisters by more than 40% from baseline in selected areas over the treatment episode.
Results: Of the patients receiving diacerein, 86% in episode 1 and 37.5% in episode 2 met the primary end point (vs 14% and 17% with placebo, respectively). This effect was still significant after the follow-up. Changes in absolute blister numbers were significant for the diacerein group only. No adverse effects were observed.
Limitations: Low patient numbers and no invasive data acquisition because of clinical burden in children.
Conclusion: This trial provides evidence of the impact of 1% diacerein cream in the treatment of EBS.
Keywords: diacerein; epidermolysis bullosa; keratin; rare genodermatosis.
Copyright © 2018 American Academy of Dermatology, Inc. Published by Elsevier Inc. All rights reserved.