Systemic granulocyte colony-stimulating factor (G-CSF) enhances wound healing in dystrophic epidermolysis bullosa (DEB): Results of a pilot trial

J Am Acad Dermatol. 2015 Jul;73(1):56-61. doi: 10.1016/j.jaad.2015.04.015. Epub 2015 May 5.

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.

Keywords: dystrophic epidermolysis bullosa; granulocyte colony-stimulating factor; therapy; wound healing.

Publication types

  • Evaluation Study

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Epidermolysis Bullosa Dystrophica / drug therapy*
  • Female
  • Granulocyte Colony-Stimulating Factor / administration & dosage*
  • Humans
  • Infant
  • Injections, Subcutaneous
  • Male
  • Pilot Projects
  • Prospective Studies
  • Wound Healing / drug effects*

Substances

  • Granulocyte Colony-Stimulating Factor