RESUMEN
UNLABELLED: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genodermatosis with severe blistering. No curative treatment is available. Scientific data indicated that epigallocatechin-3-gallate (EGCG), a green tea extract, might improve the phenotype of RDEB patients. In a multicentre, randomized, crossover, double-blind, placebo-controlled clinical trial, we evaluated a 4-month oral EGCG treatment regimen in 17 RDEB patients. We found that EGCG treatment was not more effective than placebo in modified intention to treat and per protocol analysis (n = 16; p = 0.78 and n = 10; p = 1 respectively). Tolerance was good. Specific organizational and technical difficulties of controlled randomized double-blind trials in EB patients are discussed. TRIAL REGISTRATION: US National Institutes of Health Clinical Trial Register ( NCT00951964 ).
Asunto(s)
Catequina/análogos & derivados , Epidermólisis Ampollosa Distrófica/tratamiento farmacológico , Catequina/uso terapéutico , Estudios Cruzados , Método Doble Ciego , Epidermólisis Ampollosa Distrófica/enzimología , Humanos , Metaloendopeptidasas/metabolismo , Fenoles/uso terapéutico , Estados UnidosRESUMEN
We have employed a successful therapy for epidermolysis bullosa with high-dose oral tocopherol acetate. Two siblings with dominant dystrophic epidermolysis bullosa of the Cockayne-Touraine type were reported. Both siblings suffered from tense blisters and erosions healing with scars and transient milia on the extremities. Electron microscopic study of the blisters revealed a cleavage beneath the lamina densa in both siblings. High-dose oral tocopherol acetate therapy was administered to them with satisfactory clinical reduction of the blister formation.