Efficacy of combined use of mycophenolate mofetil and granulocyte-colony-stimulating factor in the treatment of patients with dystrophic epidermolysis bullosa

[摘要] Back  ground Patients with dystrophic epidermolysis bullosa (DEB) experience various degrees of widespread recurrent skin blistering and erosions that characteristically heal with exuberant scarring and milia formation. DEB may lead to the development of skin cancers. Advances in understanding the pathogenesis of EB in the last decade have led to the development of several therapeutic strategies. Objective  To investigate the effectiveness of the combined use of granulocyte-colony-stimulating factor (GCSF) and mycophenolate mofetil (MMF) in the treatment of DEB and to compare our results with those of the previous studies that used either GCSF alone or MMF alone. Patients and methods  Fifty-one patients with DEB were enrolled into this study. They were clinically assessed for total body blisters and erosions (both count and size) and scored using the Birmingham scoring system. Results  There were significant improvements (P<0.001) with the use of combined treatment which was superior to each treatment alone. Conclusion  Subcutaneous GCSF combined with MMF was shown to be superior to the use of either MMF or GCSF alone, in decreasing the rate of blister formation and in wound healing in patients with DEB.

[学科分类] 皮肤病学

[关键词] DEB;epidermolysis bullosa;granulocyte-colony-stimulating factor;mycophenolate mofetil