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Transplantation Is Highly Effective In Treating Immune Deficiency In Children

Update Date: Jul 31, 2014 01:47 PM EDT
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Babies born with  severe immunodeficiency (SCID) can be successfully treated with a transplant of blood-forming stem cells, a new study has found. 

SCID is a group of inherited disorders that causes immune system to severely malfunction. Post breakdown, new-borns no longer possess the ability to fight off routine infections because of the damaged built-in defense system. 

Researchers reviewed more than 240 patients and found that transplants were quite effective especially when performed early in life. According to the study, children who received transplants within three and a half months after birth, 94 percent were alive five years later. The study found the best results after transplants after a "matched sibling" donors. However, transplants that were not from a matched sibling, overall five-year survival rate was also quite high - 77 to 93 percent. 

"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, in the press release. "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, the press release added. 

"This really illustrates the importance of more widespread screening for SCID so that doctors can intervene immediately," said Dr. O'Reilly. Currently, only 21 states have implemented testing as part of routine newborn screening, including New York, California, and Florida. "By the time symptoms of infection appear, the circumstances for transplant are often less favorable."

The study will be published in the New England Journal of Medicine.

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