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Topical diacerein for epidermolysis bullosa: a randomized controlled pilot study

Journal

ORPHANET JOURNAL OF RARE DISEASES
Volume 8, Issue -, Pages -

Publisher

BIOMED CENTRAL LTD
DOI: 10.1186/1750-1172-8-69

Keywords

Epidermolysis bullosa; Small molecule therapies; Pilot study; Keratin 14; Diacerein; Interleukin-1 beta

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Blistering in epidermolysis bullosa simplex type Dowling-Meara (EBS-DM) is associated with an inflammatory phenotype, which can be disrupted by diacerein in vitro. In this pilot study we hypothesized, that a topical formulation of diacerein 1% reduces blistering. Five patients initially applied diacerein underneath both armpits. Then, each participant received 1% diacerein-cream for one armpit, and placebo for the other (randomized withdrawal). The number of blisters was reduced significantly (left: -78%; right: -66% of baseline) within two weeks and remained significantly below the initial level even during withdrawal in four patients. These findings point to a relevant effect of diacerein and provide important information for a confirmative study.

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