Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor.
A patient with severe combined immunodeficiency received seven transplants of bone marrow from an HLA-B-compatible and HLA-D-compatible unrelated donor in an attempt to provide immunologic reconstitution. The first four transplants achieved restricted engraftment with evidence of rudimentary immunologic function. A fifth transplant, given after low-dose cyclophosphamide, produced reconstituion of cell-mediated immunity. Marrow aplasia developed after recontamination with a nonpathogenic microflora. Transplantation of marrow previously stored in liquid nitrogen was ineffective. A subsequent transplant, administered after high-dose cyclophosphamide, achieved durable engraftment, with complete hematopoietic and immunologic reconstitution. Seventeen months after transplantation, full functional engraftment persists. Graft-versus-host disease has been chronic and moderately severe, but limited to the skin and oral mucosa. Transplantation of marrow from unrelated histocompatible donors may provide a useful treatment for patients with severe combined immunodeficiency or aplastic anemia who lack a matched sibling or related donor.
D016026:Bone Marrow Transplantation; D003520:Cyclophosphamide / Q000494:pharmacology; D006087:Graft vs Host Reaction; D006680:HLA Antigens; D006410:Hematopoiesis / Q000187:drug effects; D006801:Humans; D007111:Immunity, Cellular / Q000187:drug effects; D007153:Immunologic Deficiency Syndromes / Q000628:therapy*; D007223:Infant; D008214:Lymphocytes / Q000276:immunology; D008297:Male; D014019:Tissue Donors; D014184:Transplantation, Homologous
D006680:HLA Antigens; D003520:Cyclophosphamide