Full hematopoietic engraftment after allogeneic bone marrow transplantation without cytoreduction in a child with severe combined immunodeficiency

Full hematopoietic engraftment after allogeneic bone marrow transplantation without cytoreduction in a child with severe combined immunodeficiency

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...

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Bibliographic Details
Published in:Blood Vol. 97; no. 3; pp. 809 - 811
Main Authors: Rubocki, Ronald J., Parsa, Jennifer R., Hershfield, Michael S., Sanger, Warren G., Pirruccello, Samuel J., Santisteban, Ines, Gordon, Bruce G., Strandjord, Sarah E., Warkentin, Phyllis I., Coccia, Peter F.
Format: Journal Article
Language:English
Published: Washington, DC Elsevier Inc 01-02-2001
The Americain Society of Hematology
Subjects:
Adenosine Deaminase - deficiency
Adenosine Deaminase - metabolism
Biological and medical sciences
Blood Cell Count
Bone Marrow Transplantation
Erythrocytes - enzymology
Female
Hematopoietic Stem Cell Transplantation
Humans
Immunodeficiencies
Immunodeficiencies. Immunoglobulinopathies
Immunopathology
Infant
Leukocytes, Mononuclear - enzymology
Medical sciences
Severe Combined Immunodeficiency - blood
Severe Combined Immunodeficiency - enzymology
Severe Combined Immunodeficiency - surgery
Transplantation, Homologous
Human
Immunopathology
Severe combined immunodeficiency
Enzyme
Stem cell
Deficiency
Related donor
Hematopoietic cell
Homograft
Infant
Adenosine deaminase
Genetic disease
Case study
Treatment
EC 3.5.4.4
Bone marrow
Graft
Hydrolases
Female
Combined immune deficiency
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Summary: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.
Bibliography:ObjectType-Case Study-3
ObjectType-Article-1
SourceType-Scholarly Journals-1
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ISSN:0006-4971
1528-0020
DOI:10.1182/blood.V97.3.809