Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor

R. J. O'Reilly, B. Dupont, Savita G Pahwa, E. Grimes, E. M. Smithwick, Rajendra Pahwa, S. Schwartz, J. A. Hansen, F. P. Siegal, M. Sorell, A. Svejgaard, C. Jersild, M. Thomsen, P. Platz, P. L'Esperance, R. A. Good

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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
Pages (from-to)1311-1318
Number of pages8
JournalNew England Journal of Medicine
Volume297
Issue number24
StatePublished - Dec 1 1977
Externally publishedYes

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ASJC Scopus subject areas

  • Medicine(all)

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O'Reilly, R. J., Dupont, B., Pahwa, S. G., 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.