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

N Engl J Med. 1977 Dec 15;297(24):1311-8. doi: 10.1056/NEJM197712152972403.

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 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.

Publication types

  • Case Reports
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Bone Marrow Transplantation*
  • Cyclophosphamide / pharmacology
  • Graft vs Host Reaction
  • HLA Antigens*
  • Hematopoiesis / drug effects
  • Humans
  • Immunity, Cellular / drug effects
  • Immunologic Deficiency Syndromes / therapy*
  • Infant
  • Lymphocytes / immunology
  • Male
  • Tissue Donors
  • Transplantation, Homologous

Substances

  • HLA Antigens
  • Cyclophosphamide