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J Am Acad Dermatol. 2018 May;78(5):892-901.e7. doi: 10.1016/j.jaad.2018.01.019. Epub 2018 Feb 2.

Diacerein orphan drug development for epidermolysis bullosa simplex: A phase 2/3 randomized, placebo-controlled, double-blind clinical trial.

Author information

1
EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria. Electronic address: v.wally@salk.at.
2
INSERM UMR 1163, Laboratory of Genetic Skin Diseases, Imagine Institute, Paris, France; University Paris Descartes-Sorbonne Paris Cité, Paris, France; Department of Genetics, Necker Hospital, AP-HP, Paris, France.
3
Department of Pediatric Dermatology, Children's Hospital, Brno, Czech Republic.
4
EB House Austria, Research Program for Molecular Therapy of Genodermatoses, Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.
5
Department of Laboratory Medicine, Paracelsus Medical University, Salzburg, Austria.
6
Landesapotheke Salzburg, Department of Production, Hospital Pharmacy, Salzburg, Austria.
7
Institute for Inborn Errors of Metabolism, Paracelsus Medical University, Salzburg, Austria; Department of Pediatrics, Paracelsus Medical University, Salzburg, Austria.
8
Research Office Biostatistics, Paracelsus Medical University, Salzburg, Austria.
9
Department of Dermatology, University Hospital Salzburg, Paracelsus Medical University, Salzburg, Austria.

Abstract

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

PMID:
29410318
DOI:
10.1016/j.jaad.2018.01.019
[Indexed for MEDLINE]
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