Megadose transplantation of purified peripheral blood CD34(+) progenitor cells from HLA-mismatched parental donors in children

Bone Marrow Transplant. 2001 Apr;27(8):777-83. doi: 10.1038/sj.bmt.1702996.


We performed HLA-mismatched stem cell transplantation with megadoses of purified positively selected mobilized peripheral blood CD34(+) progenitor cells (PBPC) from related adult donors in 39 children lacking an otherwise suitable donor. The patients received a mean number of 20.7 +/- 9.8 x 10(6)/kg purified CD34(+) and a mean number of 15.5 +/- 20.4 x 10(3)/kg CD3(+) T lymphocytes. The first seven patients received short term (<4 weeks) GVHD prophylaxis with cyclosporin A, whereas in all the following 32 patients no GVHD prophylaxis was used. In 38 evaluable patients, five patients experienced primary acute GVHD grade I and one patient grade II. In 32 patients, no signs of primary GVHD were seen and GVHD only occurred after T cell add backs. T cell reconstitution was more rapid if the number of transplanted CD34(+) cells exceeded 20 x 10(6)/kg. Of the 39 patients, 15 are alive and well, 13 died due to relapse and 10 transplant-related deaths occurred. We conclude that the HLA barrier can be overcome by transplantation of megadoses of highly purified mismatched CD34(+) stem cells. GVHD can be prevented without pharmacological immunosuppression by the efficient T cell depletion associated with the CD34(+) positive selection procedure. This approach offers a promising therapeutic option for every child without an otherwise suitable donor.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antigens, CD34 / blood*
  • Blood Donors
  • Child
  • Child, Preschool
  • Female
  • Graft vs Host Disease / prevention & control
  • Hematopoiesis
  • Hematopoietic Stem Cell Transplantation / methods*
  • Histocompatibility Testing
  • Histocompatibility*
  • Humans
  • Infant
  • Lymphocyte Depletion
  • Male
  • Parents
  • Survival Analysis
  • T-Lymphocytes / immunology
  • Transplantation, Homologous / immunology
  • Transplantation, Homologous / methods
  • Treatment Outcome


  • Antigens, CD34