title: Systemic granulocyte colony-stimulating factor (G-CSF) enhances wound healing in dystrophic epidermolysis bullosa (DEB): Results of a pilot trial.
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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: 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.
authors.forename: Jo-David
authors.initials: JD
authors.lastname: Fine
authors.affiliation: Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, Tennessee.
authors.forename: Becky
authors.initials: B
authors.lastname: Manes
authors.affiliation: Department of Pediatrics, Vanderbilt University School of Medicine, Nashville, Tennessee.
authors.forename: Haydar
authors.initials: H
authors.lastname: Frangoul
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D016108:Epidermolysis Bullosa Dystrophica / Q000188:drug therapy*; D005260:Female; D016179:Granulocyte Colony-Stimulating Factor / Q000008:administration & dosage*; D006801:Humans; D007223:Infant; D007279:Injections, Subcutaneous; D008297:Male; D010865:Pilot Projects; D011446:Prospective Studies; D014945:Wound Healing / Q000187:drug effects*
chemical_list: D016179:Granulocyte Colony-Stimulating Factor
keywords: dystrophic epidermolysis bullosa; granulocyte colony-stimulating factor; therapy; wound healing