A stem cell transplant works better than medicine to extend the lives of people with scleroderma, an autoimmune disease in which the skin hardens and organs break down, researchers said on Wednesday. The findings in the New England Journal of Medicine point to a new way of treating the incurable disease, which affects some 2.5 million people worldwide, most of them women of childbearing age.
"Scleroderma hardens the skin and connective tissues and in its severe form leads to fatal organ failure, most often the lungs," said lead author Keith Sullivan, professor of medicine and cellular therapy at Duke University. "In these severe cases, conventional drug therapies are not very effective long-term, so new approaches are a priority." The study randomly assigned 36 scleroderma patients in the United States and Canada to receive a stem cell transplant. First they had to undergo high-dose chemotherapy and whole-body radiation to fully wipe out their malfunctioning immune system.
Then they were re-infused with their own blood stem cells that had been removed and treated to eliminate the faulty white blood cells. Another 39 patients were randomly assigned to get 12 monthly intravenous injections of cyclophosphamide, which is the conventional immune suppressing treatment for severe scleroderma.
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