Jo-David Fine1, Becky Manes2, Haydar Frangoul2. 1. Division of Dermatology, Department of Medicine, Vanderbilt University School of Medicine, Nashville, Tennessee; Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, Tennessee. Electronic address: Jo-David.Fine@vanderbilt.edu. 2. Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, Tennessee.
Abstract
BACKGROUND: Chronic nonhealing wounds are the norm in patients with inherited epidermolysis bullosa (EB), especially those with dystrophic EB (DEB). A possible benefit in wound healing after subcutaneous treatment with granulocyte colony-stimulating factor (G-CSF) was suggested from an anecdotal report of a patient given this during stem cell mobilization before bone-marrow transplantation. OBJECTIVE: We sought to determine whether benefit in wound healing in DEB skin might result after 6 daily doses of G-CSF and to confirm its safety. METHODS: Patients were assessed for changes in total body blister and erosion counts, surface areas of selected wounds, and specific symptomatology after treatment. RESULTS: Seven patients with DEB (recessive, 6; dominant, 1) were treated daily with subcutaneous G-CSF (10 μg/kg/dose) and reevaluated on day 7. For all patients combined, median reductions of 75.5% in lesional size and 36.6% in blister/erosion counts were observed. When only the 6 responders were considered, there were median reductions of 77.4% and 38.8% of each of these measured parameters, respectively. No adverse side effects were noted. LIMITATIONS: Limitations include small patient number, more than 1 DEB subtype included, and lack of untreated age-matched control subjects. CONCLUSIONS: Subcutaneous G-CSF may be beneficial in promoting wound healing in some patients with DEB when conventional therapies fail.
BACKGROUND: Chronic nonhealing wounds are the norm in patients with inherited epidermolysis bullosa (EB), especially those with dystrophic EB (DEB). A possible benefit in wound healing after subcutaneous treatment with granulocyte colony-stimulating factor (G-CSF) was suggested from an anecdotal report of a patient given this during stem cell mobilization before bone-marrow transplantation. OBJECTIVE: We sought to determine whether benefit in wound healing in DEB skin might result after 6 daily doses of G-CSF and to confirm its safety. METHODS:Patients were assessed for changes in total body blister and erosion counts, surface areas of selected wounds, and specific symptomatology after treatment. RESULTS: Seven patients with DEB (recessive, 6; dominant, 1) were treated daily with subcutaneous G-CSF (10 μg/kg/dose) and reevaluated on day 7. For all patients combined, median reductions of 75.5% in lesional size and 36.6% in blister/erosion counts were observed. When only the 6 responders were considered, there were median reductions of 77.4% and 38.8% of each of these measured parameters, respectively. No adverse side effects were noted. LIMITATIONS: Limitations include small patient number, more than 1 DEB subtype included, and lack of untreated age-matched control subjects. CONCLUSIONS: Subcutaneous G-CSF may be beneficial in promoting wound healing in some patients with DEB when conventional therapies fail.
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