Treatment of dystrophic epidermolysis bullosa with bone marrow non‐hematopoeitic stem cells: a randomized controlled trial

Patients with dystrophic epidermolysis bullosa (DEB) have mutations in type VII collagen gene. Type VII collagen is synthesized by keratinocytes and fibroblasts. Based on the ability of bone marrow non‐hematopoeitic stem cells (NHBMSC) to develop into fibroblasts, we decided to investigate the use of NHBMSC in the treatment of recessive DEB (RDEB). This study included fourteen patients with RDEB; the first seven of them were given cyclosporine after the infusion of NHBMSC. As cyclosporine has been used for the treatment of RDEB we decided not to use cyclosporine for the second group of seven patients. Skin biopsies from the lesions were studied by electron microscopy before and after treatment. The number of new blisters decreased significantly after treatment in both groups (p = 0.003 and 0.004 respectively) and the rate of healing of new blisters became significantly faster after treatment in both groups (p < 0.001) with no significant difference between the two groups. Electron microscopic examination revealed increased number of anchoring fibrils after treatment in both groups. No major side effects were reported during the 1‐year follow‐up period. Our findings highlight the efficacy as well as the safety of NHBMSC in the treatment of RDEB.

[1]  J. Reichelt,et al.  Current and Future Perspectives of Stem Cell Therapy in Dermatology , 2017, Annals of dermatology.

[2]  B. Cohen,et al.  Raising Awareness Among Healthcare Providers about Epidermolysis Bullosa and Advancing Toward a Cure. , 2017, The Journal of clinical and aesthetic dermatology.

[3]  S. Yuk,et al.  Functional characterization of mesenchymal stem cells labeled with a novel PVP-coated superparamagnetic iron oxide. , 2009, Contrast media & molecular imaging.

[4]  E. Volpi,et al.  FISH glossary: an overview of the fluorescence in situ hybridization technique. , 2008, BioTechniques.

[5]  Y. Kaneda,et al.  Stem Cells , Tissue Engineering and Hematopoietic Elements Bone Marrow Cell Transfer into Fetal Circulation Can Ameliorate Genetic Skin Diseases by Providing Fibroblasts to the Skin and Inducing Immune Tolerance , 2010 .

[6]  Wei Li,et al.  Intravenously injected human fibroblasts home to skin wounds, deliver type VII collagen, and promote wound healing. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

[7]  J. Uitto,et al.  Epidermolysis bullosa. II. Type VII collagen mutations and phenotype–genotype correlations in the dystrophic subtypes , 2006, Journal of Medical Genetics.

[8]  Xiaobing Fu,et al.  Enhanced wound‐healing quality with bone marrow mesenchymal stem cells autografting after skin injury , 2006, Wound repair and regeneration : official publication of the Wound Healing Society [and] the European Tissue Repair Society.

[9]  D. Keene,et al.  Normal and gene-corrected dystrophic epidermolysis bullosa fibroblasts alone can produce type VII collagen at the basement membrane zone. , 2003, The Journal of investigative dermatology.

[10]  P. Quesenberry,et al.  Participation of bone marrow derived cells in cutaneous wound healing , 2003, Journal of cellular physiology.

[11]  G. Krueger.  Fibroblasts and dermal gene therapy: a minireview. , 2000, Human gene therapy.

[12]  E. del-Río.  Prevention of blisters in dystrophic recessive epidermolysis bullosa with cyclosporine. , 1993, Journal of the American Academy of Dermatology.

[13]  A. Meyer,et al.  Wound coverage with cultured autologous keratinocytes: use after burn wound excision, including biopsy followup. , 1988, The Journal of trauma.