El-Darouti Mohammad, Fawzy Marwa, Amin Iman, Abdel Hay Rania, Hegazy Rehab, Gabr Hala, El Maadawi Zeinab
Department of Dermatology, Faculty of Medicine, Cairo University, Cairo, Egypt.
Department of Clinical Pathology, Faculty of Medicine, Cairo University, Cairo, Egypt.
Dermatol Ther. 2016 Mar-Apr;29(2):96-100. doi: 10.1111/dth.12305. Epub 2015 Oct 5.
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.
营养不良性大疱性表皮松解症（DEB）患者的VII型胶原蛋白基因存在突变。VII型胶原蛋白由角质形成细胞和成纤维细胞合成。基于骨髓非造血干细胞（NHBMSC）发育为成纤维细胞的能力，我们决定研究NHBMSC在隐性DEB（RDEB）治疗中的应用。本研究纳入了14例RDEB患者；其中前7例在输注NHBMSC后给予环孢素。由于环孢素已用于RDEB的治疗，我们决定对第二组7例患者不使用环孢素。在治疗前后，通过电子显微镜对病变部位的皮肤活检样本进行研究。两组治疗后新水疱数量均显著减少（分别为p = 0.003和0.004），且两组治疗后新水疱的愈合速度均显著加快（p < 0.001），两组之间无显著差异。电子显微镜检查显示两组治疗后锚定原纤维数量增加。在1年的随访期内未报告重大副作用。我们的研究结果突出了NHBMSC在RDEB治疗中的有效性和安全性。
