Hematopoietic stem cell transplantation for severe combined immune deficiency or what the children have taught ss Journal Article


Authors: Rappeport, J. M.; O'Reilly, R. J.; Kapoor, N.; Parkman, R.
Article Title: Hematopoietic stem cell transplantation for severe combined immune deficiency or what the children have taught ss
Abstract: It is now more than 40 years since the first successful allogeneic hematopoietic stem cell transplantation (HSCT) for a child with severe combined immunodeficiency (SCID). In the succeeding years, HSCT for SCID patients have represented only a small portion of the total number of allogeneic HSCT performed. Nevertheless, the clinical and biologic importance of the patients transplanted for SCID has continued. SCID patients were the first to be successfully transplanted with nonsibling related bone marrow, unrelated bone marrow, T-cell depleted HSCT, and genetically corrected (gene transfer) autologous HSC. Many of the biologic insights now widely applied to allogeneic HSCT were first identified in the transplantation of SCID patients. This article reviews the clinical and biologic lessons that have been learned from HSCT for SCID patients, and how the information has impacted the general field of allogeneic HSCT. © 2010 Elsevier Inc. All rights reserved.
Keywords: child; treatment outcome; child, preschool; leukemia; transplantation, homologous; clinical trial; neutropenia; review; nonhuman; t-lymphocytes; bone marrow cells; thrombocytopenia; cyclophosphamide; hematopoietic stem cell transplantation; gene transfer; chimera; immunological tolerance; tissue donors; infant; graft versus host reaction; hematopoietic stem cells; allogeneic hematopoietic stem cell transplantation; sibling; t cell depletion; immunosuppressive treatment; bone marrow transplantation; donor selection; graft vs host disease; transplantation, autologous; severe combined immunodeficiency; hla antigen; lymphocyte depletion; immunosuppressive agent; lymphocyte function; thymocyte antibody; gene transfer techniques; genetic correlation; in utero; severe combined immune deficiency; unrelated; in utero stem cell transplantation; hemoglobinopathy; blood donor; sibling relation
Journal Title: Immunology and Allergy Clinics of North America
Volume: 30
Issue: 1
ISSN: 0889-8561
Publisher: Elsevier Inc.  
Date Published: 2010-02-01
Start Page: 17
End Page: 30
Language: English
DOI: 10.1016/j.iac.2009.10.002
PUBMED: 20113884
PROVIDER: scopus
DOI/URL:
Notes: --- - "Cited By (since 1996): 1" - "Export Date: 20 April 2011" - "CODEN: INCAE" - "Source: Scopus"
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  1. Richard O'Reilly
    748 O'Reilly