title: Cord blood transplants for SCID: better B-cell engraftment?
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.
authors.affiliation: Department of Pediatrics, Division of Immunology, Allergy, and Rheumatology, University of California, Los Angeles, CA 90095, USA. wchan@mednet.ucla.edu
authors.forename: Wan-Yin
authors.initials: WY
authors.lastname: Chan
authors.forename: Robert Lloyd
authors.initials: RL
authors.lastname: Roberts
authors.forename: Theodore B
authors.initials: TB
authors.lastname: Moore
authors.forename: E Richard
authors.initials: ER
authors.lastname: Stiehm
mesh_terms: D001402:B-Lymphocytes / Q000276:immunology*; D016026:Bone Marrow Transplantation; D005260:Female; D005312:Fetal Blood / Q000637:transplantation*; D006086:Graft vs Host Disease; D006650:Histocompatibility Testing; D006801:Humans; D007231:Infant, Newborn; D008212:Lymphocyte Depletion; D008297:Male; D011379:Prognosis; D016511:Severe Combined Immunodeficiency / Q000628:therapy*