Transplantation of hematopoietic stem cells in human severe combined immunodeficiency: longterm outcomes.


Journal Article (Review)

Severe combined immunodeficiency (SCID) is a syndrome of diverse genetic cause characterized by profound deficiencies of T- and B-cell function and, in some types, also of NK cells and function. Mutations in thirteen different genes have been found to cause this condition, which is uniformly fatal in the first 2 years of life unless immune reconstitution can be accomplished. In the 42 years since the first bone marrow transplant was given in 1968, the standard treatment for all forms of SCID has been allogeneic bone marrow transplantation. Both HLA-identical unfractionated and T-cell-depleted HLA-haploidentical bone marrow transplants have been very successful in effecting immune reconstitution, especially if performed in the first 3.5 months of life and without pre-transplant chemotherapy. This paper summarizes the longterm outcome, according to molecular type, of 166 consecutive SCID infants given non-conditioned related donor bone marrow transplants at this institution over the past 28.3 years and reviews published reports of longterm outcomes of transplants in SCID performed at other centers.

Full Text

Duke Authors

Cited Authors

  • Buckley, RH

Published Date

  • April 2011

Published In

Volume / Issue

  • 49 / 1-3

Start / End Page

  • 25 - 43

PubMed ID

  • 21116871

Pubmed Central ID

  • 21116871

Electronic International Standard Serial Number (EISSN)

  • 1559-0755

Digital Object Identifier (DOI)

  • 10.1007/s12026-010-8191-9


  • eng

Conference Location

  • United States