Early stem cell transplantation improves survival in children with SCID

NEW YORK (Reuters Health) – Stem cell transplantation performed during the neonatal period can dramatically improve the survival of children with severe combined immunodeficiency (SCID), according to a report published in the February 1st issue of Blood.

Typically, the posttransplantation survival rate for children with SCID approaches 80%. The current findings indicate, however, that if the transplantation is performed in the first 28 days of life, a survival rate of 95% can be achieved.

Dr. Laurie A. Myers, from Duke University Medical Center in Durham, North Carolina, and colleagues compared the outcomes of 21 SCID infants who underwent transplantation during the neonatal period with those of 96 similar infants who received transplants later. The transplantations occurred over a 19.2-year period.

All but one of the early transplant recipients were alive at the time of the current study, the authors note. In contrast, only 71 of the later transplant recipients were alive at evaluation.

Higher lymphocyte responses to phytohemagglutinin were noted in the early recipient group compared with the later recipient group. Early recipients also demonstrated higher numbers of CD3+ and CD45RA+ T cells and had T-cell antigen receptor excision circles that peaked earlier and with higher values than those of later recipients.

The current findings suggest that routine testing for SCID should be incorporated in newborn screening protocols, the investigators note. Until such measures are implemented, physicians should pay close attention to the absolute lymphocyte count in infants presenting with fever, chronic diarrhea, or failure to thrive, they emphasize.

"Early diagnosis of SCID would allow for transplantation to be performed not only before a potentially fatal opportunistic infection develops but under conditions ideal for thymopoiesis," Dr. Myers' team concludes.

Blood 2002;99:872-878.

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