Bone Marrow Transplant May Treat Blistering Disease
Treated epidermolysis bullosa patients show increased deposition of type VII collagen
WEDNESDAY, Aug. 11 (HealthDay News) -- Allogeneic bone marrow transplantation may help children with recessive dystrophic epidermolysis bullosa by increasing the deposition of type VII collagen (C7), the lack of which characterizes the disease, according to research published in the Aug. 12 issue of the New England Journal of Medicine.
John E. Wagner, M.D., of the University of Minnesota in Minneapolis, and colleagues used chemotherapy and allogeneic stem-cell transplantation to treat seven children with recessive dystrophic epidermolysis bullosa. They assessed for C7 expression on the hypothesis that the stem cells of allogeneic marrow may be able to ameliorate manifestations of the disease.
Of the six surviving patients (one died before receiving the stem-cell transplant), the researchers found that all experienced better wound healing and less blister formation 30 to 130 days post-transplantation, although one had severe regimen-related cutaneous toxicity. Five patients had observable C7 deposition at the dermal-epidermal junction, and five patients were still living 130 to 799 days after the procedure. One patient died 183 days after transplantation due to graft rejection and infection. All six showed substantial amounts of donor cells without detectable anti-C7 antibodies.
"Increased C7 deposition and a sustained presence of donor cells were found in the skin of children with recessive dystrophic epidermolysis bullosa after allogeneic bone marrow transplantation. Further studies are needed to assess the long-term risks and benefits of such therapy in patients with this disorder," the authors write.