Background: Many interventions have been described for inherited epidermolysis bullosa (EB), but it is unclear which are beneficial.
Aims: A systematic review of randomized controlled trials (RCTs) was performed to inform practice and highlight research gaps.
Methods: 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.
Results: 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).
Conclusions: 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.