Antibody-treated bone marrow transplantation for patients with severe combined immune deficiency.
Histocompatible bone marrow transplantation is the treatment of choice for patients with severe combined immunodeficiency (SCID). Patients without histocompatible donors have been treated with a variety of nontransplant therapies; however, normal immunocompetence has not been achieved. Bone marrow transplantation with untreated histoincompatible bone marrow has resulted in fatal graft-versus-host disease and/or no lymphoid engraftment. Based upon murine experiments, the use of monoclonal antibody- and complement-treated parental bone marrow has been used to successfully transplant SCID patients. Monoclonal antibodies to a variety of T-lymphocyte differentiation antigens (OKT-3, T-12, CT-2, and Leu-1) have been used. The antibody-treated transplants differ in several important respects from histocompatible transplants for SCID. Many recipients for treated marrow grafts require immunosuppression. In certain cases, hematopoietic stem cell ablation with total body irradiation (TBI) or busulfan has been required to achieve stable lymphoid engraftment although the biological basis for the requirement for hematopoietic stem cell elimination is unclear. The use of antibody-treated parental bone marrow is the most effective form of therapy for patients with SCID who do not have a histocompatible donor.
D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D002648:Child; D003165:Complement System Proteins; D003602:Cytotoxicity, Immunologic; D006648:Histocompatibility; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000276:immunology / Q000628:therapy*; D008213:Lymphocyte Activation; D010835:Phytohemagglutinins / Q000494:pharmacology; D012276:Ricin / Q000627:therapeutic use; D013601:T-Lymphocytes / Q000276:immunology
D000911:Antibodies, Monoclonal; D010835:Phytohemagglutinins; D003165:Complement System Proteins; D012276:Ricin