Cord blood transplants for SCID: better B-cell engraftment?

J Pediatr Hematol Oncol. 2013 Jan;35(1):e14-8. doi: 10.1097/MPH.0b013e31824e15b8.

Abstract

Hematopoietic stem-cell transplantation is the treatment of choice for severe combined immunodeficiency (SCID). Despite successful T-cell engraftment in transplanted patients, B-cell function is not always achieved; up to 58% of patients require immunoglobulin therapy after receiving haploidentical transplants. We report 2 half-sibling males with X-linked γ-chain SCID treated with different sources of stem cells. Sibling 1 was transplanted with T-cell-depleted haploidentical maternal bone marrow and sibling 2 was transplanted with 7/8 human leukocyte antigen-matched unrelated umbilical cord blood. Both patients received pretransplant conditioning and posttransplant graft-versus-host-disease prophylaxis. B-cell engraftment and function was achieved in sibling 1 but not in sibling 2. This disparate result is consistent with a review of 19 other SCID children who received cord blood transplants. B-cell function, as indicated by no need for immunoglobulin therapy, was restored in 42% of patients given haploidentical transplants and in 68% of patients given matched unrelated donor transplants compared with 80% of patients given cord blood transplants. Cord blood is an alternative source of stem cells for transplantation in children with SCID and has a higher likelihood of B-cell reconstitution.

Publication types

  • Case Reports
  • Comparative Study

MeSH terms

  • B-Lymphocytes / immunology*
  • Bone Marrow Transplantation*
  • Female
  • Fetal Blood / transplantation*
  • Graft vs Host Disease*
  • Histocompatibility Testing
  • Humans
  • Infant, Newborn
  • Lymphocyte Depletion
  • Male
  • Prognosis
  • Severe Combined Immunodeficiency / therapy*