Topical diacerein for epidermolysis bullosa: a randomized controlled pilot study

Orphanet Journal of Rare Diseases

Johann W Bauer³  Nora Eiler³  Anja Diem³  Michael Dornauer³  Thomas K Felder¹  Peter Hofbauer²  Martin Wolkersdorfer²  Wolfgang Hitzl⁴  Angelika Moder⁵  Florian Lagler⁵  Sophie Kitzmueller³  Verena Wally³ 

[1] Department of Laboratory Medicine, Paracelsus Medical University, Salzburg, Austria;Department of Production, Hospital Pharmacy, Landesapotheke, Salzburg, Austria;Division of Experimental Dermatology and EB House Austria, Department of Dermatology, Paracelsus Medical University Salzburg, Muellner Hauptstraße 48, Salzburg 5020, Austria;Paracelsus Medical University, Research Office, Biostatistics, Salzburg, Austria;Institute for Inborn Errors of Metabolism and Department of Pediatrics, Paracelsus Medical University, Salzburg, Austria

关键词: Interleukin-1ß;    Diacerein;    Keratin 14;    Pilot study;    Small molecule therapies;    Epidermolysis bullosa;   

Others  :  864001
DOI  :  10.1186/1750-1172-8-69

 received in 2013-03-04, accepted in 2013-05-02,  发布年份 2013

【 摘 要 】

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.

【 授权许可】

   
2013 Wally et al.; licensee BioMed Central Ltd.

【 图 表 】

【 参考文献 】

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