Marrow transplant improves rare epidermolysis bullosa

Minneapolis - Results of a small study show that bone-marrow transplantation may help fight the rare, genetic skin disease recessive dystrophic epidermolysis bullosa. But, due to risk factors, the treatment is probably appropriate only for the most severe cases, MedPage Today reports.

Researchers from the University of Minnesota found that among seven young patients with the disease, the six who received allogeneic stem-cell transplantation from bone marrow displayed improved wound healing and a reduction in blister formation.

One of the children with the incurable, often fatal, disease died during preparation for the transplant. Of the remaining transplant patients, one experienced a severe, regimen-related cutaneous toxicity, and another died 183 days after the procedure from graft rejection and infection.

The authors wrote, “Despite the potential benefits of marrow transplantation, it is a high-risk therapeutic approach that could shorten the expected survival of patients with recessive dystrophic epidermolysis bullosa, particularly those with less severe clinical manifestations.”

They add, however, that the study’s results warrant further research into stem cell-based approaches for patients with the severest forms of the disease. The study appeared in the Aug. 12 issue of the New England Journal of Medicine.