Babies who are born with severe combined immunodeficiency (SCID) can be successfully treated with a transplant of blood-forming stem cells, according to experts led by Memorial Sloan Kettering’s (MSK’s) Richard J. O’Reilly, M.D., a world-renowned pioneer in the development of transplant protocols. Their review will be published in the July 31, 2014 issue of the New England Journal of Medicine. SCID is a group of inherited disorders that cause the immune system to severely malfunction. When this breakdown occurs, babies no longer have the ability to fight off routine infections because their natural, built-in defense system has been damaged. If undiagnosed or left untreated, SCID is almost always fatal within the first year of life. A review of more than 240 patient cases found transplants to be quite effective, especially when performed early in life. Of those children receiving transplants within three and a half months after birth, 94 percent were alive five years later. The best results, not surprisingly, were seen after transplant from “matched sibling” donors. But among patients who didn’t have a matched sibling, overall five-year survival rates were quite high — 77 to 93 percent — if they were transplanted in the first three and a half months of life. “This confirms that transplants for SCID work well in very young children, but it also shows that any child with this disease can be treated with a high likelihood of a cure with a transplant from a parent or unrelated donor, not just a matched brother or sister,” explained Dr. O’Reilly, Chair of MSK’s Pediatrics Department and Chief of the Pediatric Bone Marrow Transplant Service.
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