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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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
Issue number24
StatePublished - Dec 1 1977
Externally publishedYes


ASJC Scopus subject areas

  • Medicine(all)

Cite this

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.