Reconstitution in Severe Combined Immunodeficiency by Transplantation of Marrow from an Unrelated Donor

Richard J. O'reilly, bo Dupont, Savita Pahwa, Elena Grimes, Elizabeth M. Smithwick, Rajendra Pahwa, Stanley Schwartz, John A. Hansen, Frederick P. Siegal, Michael Sorell, Arne Svejgaard, Casper Jersild, Mogens Thomsen, Per Platz, Pierre L'esperance, Robert A. Good

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128 Scopus citations

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1311-1318
Number of pages8
JournalNew England Journal of Medicine
Volume297
Issue number24
DOIs
StatePublished - Dec 15 1977

ASJC Scopus subject areas

  • Medicine(all)

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    O'reilly, R. J., Dupont, B., Pahwa, S., Grimes, E., Smithwick, E. M., Pahwa, R., Schwartz, S., Hansen, J. A., Siegal, F. P., Sorell, M., Svejgaard, A., Jersild, C., Thomsen, M., Platz, P., L'esperance, P., & Good, R. A. (1977). Reconstitution in Severe Combined Immunodeficiency by Transplantation of Marrow from an Unrelated Donor. New England Journal of Medicine, 297(24), 1311-1318. https://doi.org/10.1056/NEJM197712152972403