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Severe combined immunodeficiency (SCID) is a heterogeneous primary condition that is often lethal in the absence of aggressive immune replacement therapy, curative hematopoietic-cell transplantation, or both. A multicenter collaboration reports on 240 children with SCID who were diagnosed at a median age of 139 days and underwent transplantation at a median of 180 days. The children had an overall 5-year survival of 74%, and 45 required a second transplant (56% survival).
The survival rate in infants transplanted at 3.5 months or younger was 94%, similar to that among older infants with no history of infection (90%) and among those with infections that were cured before transplantation (82%). Matched donor siblings offered the highest surviv…