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Blood ; 97(3): 809-11, 2001 Feb 01.
Article in English | MEDLINE | ID: mdl-11157502

ABSTRACT

Bone marrow transplantation (BMT) for severe combined immunodeficiency (SCID) with human leukocyte antigen (HLA)-identical sibling donors but no pretransplantation cytoreduction results in T-lymphocyte engraftment and correction of immune dysfunction but not in full hematopoietic engraftment. A case of a 17-month-old girl with adenosine deaminase (ADA) deficiency SCID in whom full hematopoietic engraftment developed after BMT from her HLA-identical sister is reported. No myeloablative or immunosuppressive therapy or graft-versus-host disease (GVHD) prophylaxis was given. Mild acute and chronic GVHD developed, her B- and T-cell functions became reconstituted, and she is well almost 11 years after BMT. After BMT, repeated studies demonstrated: (1) Loss of a recipient-specific chromosomal marker in peripheral blood leukocytes (PBLs) and bone marrow, (2) conversion of recipient red blood cell antigens to donor type, (3) conversion of recipient T-cell, B-cell, and granulocyte lineages to donor origin by DNA analysis, and (4) increased ADA activity and metabolic correction in red blood cells and PBLs.


Subject(s)
Adenosine Deaminase/deficiency , Bone Marrow Transplantation , Hematopoietic Stem Cell Transplantation , Severe Combined Immunodeficiency/surgery , Adenosine Deaminase/metabolism , Blood Cell Count , Erythrocytes/enzymology , Female , Humans , Infant , Leukocytes, Mononuclear/enzymology , Severe Combined Immunodeficiency/blood , Severe Combined Immunodeficiency/enzymology , Transplantation, Homologous
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