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Hematopoietic-Cell Transplantation Provides Excellent Outcome in SCID

July 30, 2014

Hematopoietic-Cell Transplantation Provides Excellent Outcome in SCID

  1. F. Bruder Stapleton, MD

Five-year survival was 94% in infants receiving transplants before age 3.5 months.

  1. F. Bruder Stapleton, MD

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 survival (97%). Older age and active infection at the time of transplantation were strongly associated with lower graft survival. Patients infected at the time of transplantation had better survival with reduced-intensity pretransplant conditioning. Among survivors, reduced conditioning was associated with improved CD3+ T-cell reconstitution, freedom from immunoglobulin infusions, and IgA recovery. The genetic subtype of SCID did not affect survival.

Comment

With newborn screening, SCID can now be diagnosed before the first infection. These results clearly show that early transplantation prior to any infection has a very high success rate. However, convincing families to approve transplantation for a perfectly normal-appearing newborn with SCID is a challenging, but important task.

  • Disclosures for F. Bruder Stapleton, MD at time of publication Grant / research support NIH-NCI Editorial boards UpToDate

Citation(s):

Reader Comments (1)

sedigheh rafiei tabatabaei Pediatric Subspecialty

In my country, we don't have newborn screening. So, SCID usually can be diagnosed after the first severe or several infections in the first child of a family. Therefore transplantation in infants 3.5 months or younger age is impossible.

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