TY - JOUR
T1 - Reconstitution in Severe Combined Immunodeficiency by Transplantation of Marrow from an Unrelated Donor
AU - O'reilly, Richard J.
AU - Dupont, bo
AU - Pahwa, Savita
AU - Grimes, Elena
AU - Smithwick, Elizabeth M.
AU - Pahwa, Rajendra
AU - Schwartz, Stanley
AU - Hansen, John A.
AU - Siegal, Frederick P.
AU - Sorell, Michael
AU - Svejgaard, Arne
AU - Jersild, Casper
AU - Thomsen, Mogens
AU - Platz, Per
AU - L'esperance, Pierre
AU - Good, Robert A.
N1 - Copyright:
Copyright 2017 Elsevier B.V., All rights reserved.
PY - 1977/12/15
Y1 - 1977/12/15
N2 - A patient with severe combined immunodeficiency received seven transplants of bone marrow from an HLA-B-compatible and HLA-D-compatible unrelated donor in an attempt to provide immunologic reconstitution. The first 4 transplants achieved restricted engraftment with evidence of rudimentary immunologic function. A fifth transplant, given after low-dose cyclophosphamide, produced reconstitution of cell-mediated immunity. Marrow aplasia developed after recontamination with a non-pathogenic microflora. Transplantation of marrow previously stored in liquid nitrogen was ineffective. A subsequent transplant, administered after high-dose cyclophosphamide, achieved durable engraftment, with complete hematopoietic and immunologic reconstitution. Seventeen months after transplantation, full functional engraftment persists. Graft-versus-host disease has been chronic and moderately severe, but limited to the skin and oral mucosa. Transplantation of marrow from unrelated histocompatible donors may provide a useful treatment for patients with severe combined immunodeficiency or aplastic anemia who lack a mtched sibling or related donor.
AB - A patient with severe combined immunodeficiency received seven transplants of bone marrow from an HLA-B-compatible and HLA-D-compatible unrelated donor in an attempt to provide immunologic reconstitution. The first 4 transplants achieved restricted engraftment with evidence of rudimentary immunologic function. A fifth transplant, given after low-dose cyclophosphamide, produced reconstitution of cell-mediated immunity. Marrow aplasia developed after recontamination with a non-pathogenic microflora. Transplantation of marrow previously stored in liquid nitrogen was ineffective. A subsequent transplant, administered after high-dose cyclophosphamide, achieved durable engraftment, with complete hematopoietic and immunologic reconstitution. Seventeen months after transplantation, full functional engraftment persists. Graft-versus-host disease has been chronic and moderately severe, but limited to the skin and oral mucosa. Transplantation of marrow from unrelated histocompatible donors may provide a useful treatment for patients with severe combined immunodeficiency or aplastic anemia who lack a mtched sibling or related donor.
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U2 - 10.1056/NEJM197712152972403
DO - 10.1056/NEJM197712152972403
M3 - Article
C2 - 21351
AN - SCOPUS:0017697285
VL - 297
SP - 1311
EP - 1318
JO - New England Journal of Medicine
JF - New England Journal of Medicine
SN - 0028-4793
IS - 24
ER -