Transplantation Outcomes for Severe Combined Immunodeficiency, 2000–2009

An analysis of outcomes from 2000 through 2009 at 25 centers treating children with severe combined immunodeficiency (SCID) showed that early transplantation of hematopoietic cells in uninfected children had the best outcome, regardless of donor type. Severe combined immunodeficiency (SCID) is a genetically heterogeneous and lethal disorder of infancy. It is characterized by severe T-cell lymphocytopenia and a lack of antigen-specific T-cell and B-cell immune responses. 1 Allogeneic hematopoietic-cell transplantation with the use of bone marrow from an HLA-identical sibling 2 or an unrelated donor, 3 T-cell–depleted marrow or peripheral-blood stem cells from a haploidentical, related donor, 4 – 7 or umbilical-cord blood 8 – 10 can fully correct the T-cell deficiency and, less consistently, the B-cell deficiency in patients with SCID. 2 – 12 Expanded donor availability and advances in supportive care and treatment of infections have improved long-term outcomes after hematopoietic-cell transplantation. 10 , . . .