Transplantation highly effective in immunodeficient children

31 July 2014

Experts at the Memorial Sloan Kettering's have said that a stem cell transplant could help treat babies born with severe combined immunodeficiency (SCID).

Dr Richard O'Reilly, a world-renowned pioneer in the development of transplant protocols, has found that babies with SCID - a group of disorders resulting in severe malfunction of the immune system - can receive a transplant of blood-forming stem cells.

The review, published in the New England Journal of Medicine, saw 240 babies with SCID undergo transplant surgery in the first 3.5 months after birth, and 94 per cent lived for at least five years. The condition means that infants lose the ability to fight off routine infections and, if undiagnosed or untreated, it is almost always fatal within the first year of life.

It found that the earlier the transplants were given, the more effective they were, and the best results were delivered from "matched sibling" donors. However, when this preferred option wasn't available, overall five-year survival rates were still quite high at 77 to 93 per cent.

One critical factor remains that the baby must be free of infection at the time of transplant, which illustrates the importance of widespread screening for SCID, according to Dr O'Reilly.

Posted by Philip Briggs


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