Blood, 1 February 2001, Vol. 97, No. 3, pp. 809-811
BRIEF REPORT
Full hematopoietic engraftment after allogeneic bone marrow
transplantation without cytoreduction in a child with severe combined
immunodeficiency
Ronald J. Rubocki,
Jennifer
R. Parsa,
Michael S. Hershfield,
Warren G. Sanger,
Samuel J. Pirruccello,
Ines Santisteban,
Bruce G. Gordon,
Sarah E. Strandjord,
Phyllis I. Warkentin, and
Peter F. Coccia
From the Departments of Pediatric-Hematology/Oncology
and Pathology and Microbiology, University of Nebraska Medical Center,
Omaha, NE; and the Department of Rheumatology and Immunology, Duke
University Medical Center, Durham, NC.
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.
