title: Haploidentical bone marrow stem cell transplantation in human severe combined immunodeficiency.
abstract: From May 1992 to March 1993, 50 infants with severe combined immunodeficiency (SCID) were given bone marrow transplants at Duke University Medical Center. None received chemotherapy for conditioning or for graft-versus-host disease (GVHD) prophylaxis. Forty-one received haploidentical parental marrow depleted of T cells by soybean lectin and sheep red blood cell resetting, and nine received HLA-identical marrow. Forty (80%) survived from 1 week to almost 11 years posttransplantation, including nine of nine (100%) HLA-identical marrow recipients and 31 of 41 haploidentical recipients. T-cell function was present within 2 weeks after transplantation of unfractionated HLA-identical marrow, but not until 3 to 4 months after T-cell-depleted haploidentical marrow stem cells. All 37 patients who are more than 4 months posttransplantation have good T-cell function, and all but one have 100% donor T cells. B-cell function developed slowly or not at all in some recipients of haploidentical marrow. Fourteen (four HLA-identical and 10 haploidentical recipients) have some donor B cells; 19 patients are receiving intravenous immune globulin (IVIG) therapy.
authors.affiliation: Department of Pediatrics, Duke University Medical Center, Durham, NC 27710.
authors.forename: R H
authors.initials: RH
authors.lastname: Buckley
authors.forename: S E
authors.initials: SE
authors.lastname: Schiff
authors.forename: R I
authors.initials: RI
authors.lastname: Schiff
authors.forename: J L
authors.initials: JL
authors.lastname: Roberts
authors.forename: M L
authors.initials: ML
authors.lastname: Markert
authors.forename: W
authors.initials: W
authors.lastname: Peters
authors.forename: L W
authors.initials: LW
authors.lastname: Williams
authors.forename: F E
authors.initials: FE
authors.lastname: Ward
mesh_terms: D001402:B-Lymphocytes / Q000276:immunology; D016026:Bone Marrow Transplantation; D002423:Cause of Death; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000628:therapy*; D013577:Syndrome; D013601:T-Lymphocytes / Q000276:immunology