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Transplantation shown to be highly effective in treating immune deficiency in children
Date:7/30/2014

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 Richard J. O'Reilly, MD, a world-renowned pioneer in the development of transplant protocols. Their review will be published in the July 30 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. "Irrespective of the transplant approach used, if the child is transplanted early without infection you will have an extraordinarily good result."

But a critical factor continues to be ensuring that the child has no infection at the time of transplant. "This really illustrates the importance of more wide
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Contact: Courtney DeNicola Nowak
denicolc@mskcc.org
212-639-3573
Memorial Sloan-Kettering Cancer Center
Source:Eurekalert

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