A systematic review of randomized controlled trials of treatments for inherited forms of epidermolysis bullosa

Many interventions have been described for inherited epidermolysis bullosa (EB), but it is unclear which are beneficial. A systematic review of randomized controlled trials (RCTs) was performed to inform practice and highlight research gaps. The Cochrane Central Register of Controlled Trials, MEDLINE, EMBASE and the Cochrane Skin Group specialist library, from inception until 1 April 2007, were searched. Primary outcomes were healing of lesions or prevention of new lesions. Trials were assessed for quality of reporting and data were extracted. Five randomized double-blind placebo-controlled crossover studies were identified (n = 102). Two studies assessed oral tetracyclines in EB simplex (EBS). In one study (n = 12), 4/6 patients improved and 2/6 deteriorated on a dose of 1500 mg of tetracycline daily; only two patients completed the study. In the second study (n = 21), 6/18 and 7/18 improved on oxytetracycline 1 g and placebo, respectively. Two RCTs assessed topical interventions for EBS: aluminium chloride hexahydrate solution 20% (n = 23) and bufexamac cream 5% (n = 8). Neither showed a benefit over placebo. One RCT of 36 patients with recessive dystrophic EB compared phenytoin with placebo and failed to show any difference in mean lesion counts (difference = 0, 95% CI -11 to 4). There is no reliable trial evidence for interventions in inherited EB. In future, it may be that gene treatment becomes the best treatment approach for these diseases.