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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)

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

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 four transplants achieved restricted engraftment with evidence of rudimentary immunologic function. A fifth transplant, given after low-dose cyclophosphamide, produced reconstituion of cell-mediated immunity. Marrow aplasia developed after recontamination with a nonpathogenic 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 matched sibling or related donor.[1]


  1. Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor. 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. N. Engl. J. Med. (1977) [Pubmed]
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