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 ± 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.
RCT Entities:
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 ± 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.
Authors: Amy S Paller; John Browning; Milos Nikolic; Christine Bodemer; Dedee F Murrell; Willistine Lenon; Eva Krusinska; Allen Reha; Hjalmar Lagast; Jay A Barth Journal: Orphanet J Rare Dis Date: 2020-06-23 Impact factor: 4.123
Authors: Johannes S Kern; Agnes Schwieger-Briel; Sandra Löwe; Mark Sumeray; Charles Davis; Anna E Martinez Journal: Trials Date: 2019-06-11 Impact factor: 2.279
Authors: Vanessa Lys Simas Yamakawa Boeira; Erica Sales Souza; Bruno de Oliveira Rocha; Pedro Dantas Oliveira; Maria de Fátima Santos Paim de Oliveira; Vitória Regina Pedreira de Almeida Rêgo; Ivonise Follador Journal: An Bras Dermatol Date: 2013 Mar-Apr Impact factor: 1.896