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Journal of the Dermatology Nurses' Association

February 2011, Volume 3 Number 1 , p 50 - 52


  • Allison Sutton


Wagner, J., Ishida-Yamamoto, A., McGrath, J., Hordinsky, M., Keene, D., Woodley, D., et al. (2010). Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. New England Journal of Medicine, 363, 629-639.Recessive dystrophic epidermolysis bullosa (RDEB) is an inherited mechanobullous disease caused by mutations in the gene COL7A1, which encodes type VII collagen. Type VII collagen is a major component of anchoring fibrils, whose function is to tether the epidermal basement membrane to the dermis. This disease is often very severe and characterized by cycles of extensive blistering of mucocutaneous sites followed by ulcerations and scarring. Complications of scarring in the skin include flexion contractures of limbs and pseudosyndactyly or "mitten deformities" of the hands and feet. Complications of scarring in mucosal sites include narrowing of the oral cavity, limitation of tongue movement, loss of teeth, esophageal stricture formation, and narrowing of the airway. Because of these complications as well as the extensive wounds, caloric needs are increased, and the combination leads to malnutrition, anemia, and growth retardation. In the past, these patients died in infancy of sepsis and other complications of extensive blistering. Now, with improved nutritional and wound care, patients can survive into their teens or adulthood. Unfortunately, at that time, aggressive squamous cell carcinomas often occur within the abnormal skin, and many die of metastatic disease.To date, the care of RDEB patients has been palliative and restricted to treatment of individual wounds and systemic complications as they arise. This group hypothesized that allogeneic marrow contains stem cells capable of producing Type VII collagen, and therefore if transferred to patients deficient in this protein, it would ameliorate the manifestations of RDEB. They first studied this in mouse models and then conducted these Phase 1 and Phase 2 clinical trials. Between October 2007 and

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