The efficacy of trimethoprim in wound healing of patients with epidermolysis bullosa: A feasibility trial Irene Lara-Corrales, MD, MSc, a Patricia C. Parkin, MD, a Derek Stephens, MS, a Jill Hamilton, MD, MSc, a Gideon Koren, MD, a Miriam Weinstein, MD, a Ronald G. Sibbald, MD, MEd, b and Elena Pope, MD, MSc a Toronto, Ontario, Canada Background: There are no systemic therapies known to facilitate wound healing in patients with recessive dystrophic epidermolysis bullosa (RDEB). Objectives: We sought to assess the feasibility of a trial to examine the efficacy of trimethoprim (TMP) in healing chronic wounds in patients with RDEB and to examine the effect of TMP on lesion counts, quality of life, and emergence of antibiotic resistance. Methods: We conducted a feasibility study using a prospective, randomized, double-blinded, placebo- controlled, crossover design. The study took place between October 2006 and September 2007 in the epidermolysis bullosa clinic at the Hospital for Sick Children in Toronto, Ontario, Canada. Liquid TMP or placebo was given orally or via gastrostomy tube in two divided doses for 2 months; the main outcome measure was a decrease in surface area of selected chronic wounds. Results: Ten subjects with RDEB were enrolled in the study; 7 completed both study arms (4 male, 3 female). Age at enrollment was 14 6 5.4 years. Although all patients showed improved wound healing on TMP, the crossover analysis, TMP versus placebo, approached but did not reach statistical significance (P = .08). While receiving TMP, 6 of 7 patients had more than 50% reduction in chronic wound surface area; while receiving placebo, 2 of 6 patients had more than 50% reduction in wound surface area (P = .03). Secondary outcome measures did not achieve statistical significance. Limitations: Small sample size is a limitation. Conclusions: This proof-of-concept study demonstrates the potential efficacy of TMP in improving wound healing in RDEB, and provides useful information for further prospective studies. ( J Am Acad Dermatol 2012;66:264-70.) Key words: chronic wounds; dystrophic epidermolysis bullosa; randomized controlled trial; trimethoprim; wound healing. E pidermolysis bullosa (EB) is classified into 4 major types: EB simplex (EBS), junctional EB, dystrophic EB (DEB), and mixed. 1 Mutations in the gene COL7A1, encoding collagen VII (the main component of the anchoring fibrils at the dermoepi- dermal junction), characterize DEB. 2 In recessive DEB (RDEB), collagen VII is generally absent, lead- ing to a more severe phenotype, 3 although the same genetic abnormality can be associated with variable clinical features. 4 Patients experience daily blister formation leading to scarring, limb deformities, and chronic wounds. 3 Skin fragility predisposes wounds From the Hospital for Sick Children a and Women’s College Hospital. b Funding sources: None. Conflicts of interest: None declared. Supported in part by DEBRA Canada and the Canadian Dermatol- ogy Foundation. Smith & Nephew provided the VISITRAK and Mo ¨lnlycke Health Care Inc provided the dressings used in the trial. These organizations were not involved in the study design, data analysis, interpretation, or preparation of the manuscript. Accepted for publication January 28, 2010. Reprint requests: Elena Pope, MD, MSc, Hospital for Sick Children, 555 University Ave, Toronto, Ontario M5G1X8 Canada. E-mail: elena.pope@sickkids.ca. Published online December 8, 2011. 0190-9622/$36.00 ª 2010 by the American Academy of Dermatology, Inc. doi:10.1016/j.jaad.2010.01.047 264