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

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Abstract
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 × 106/kg purified CD34+ and a mean number of 15.5 ± 20.4 × 103/kg CD3+ T lymphocytes. The first seven patients received short term (+ cells exceeded 20 × 106/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. Bone Marrow Transplantation (2001) 27, 777–783.