Factors Contributing to Positive Outcomes for Infants with Severe Combined Immunodeficiency
Bethesda, MD - Early transplantation of blood-forming stem cells is a highly effective treatment for infants with severe combined immunodeficiency (SCID), a group of rare, life-threatening inherited immune system disorders, a study funded by the National Institutes of Health suggests.
Approximately three-quarters of SCID infants who received transplants survived for at least five years. Infants who received transplants within the first 3.5 months of life had the best outcomes. Researchers from the Primary Immune Deficiency Treatment Consortium (PIDTC), funded by NIH’s National Institute of Allergy and Infectious Diseases (NIAID), report their findings in the July 31 issue of the New England Journal of Medicine.
SCID is caused by defects in genes involved in the development and function of infection-fighting T and B cells. Infants with SCID appear healthy at birth but are highly susceptible to infections. If untreated, SCID is fatal, usually within the first year of life. Development of a newborn screening test has made it possible to detect SCID before symptoms appear. The test was added to the U.S. Department of Health and Human Services’ Recommended Uniform Screening Panel for newborns in 2010, but to date, only 21 states have implemented newborn screening for SCID. Continue>
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