Transplantation of megadoses of purified haploidentical stem cells

Ann N Y Acad Sci. 1999 Apr 30:872:351-61; discussion 361-2. doi: 10.1111/j.1749-6632.1999.tb08479.x.

Abstract

Peripheral mobilized parental CD34+ progenitors were isolated and used for the hematopoietic reconstitution after a myeloablative therapy in 23 pediatric patients with various diseases. Fourteen donors were human leukocyte antigen (HLA) three-loci mismatches, 6 donors were two-loci and 3 donors were one-locus mismatches. For depletion of T-lymphocytes, a positive selection of the mobilized peripheral CD34+ progenitors using the method of magnetic-activated cell sorting (MACS) was used. The purity of the CD34+ cells after MACS-sorting was 98-99%, the average number of transplanted CD34+ cells was 14.2 x 10(6)/kg (range 5.4-3.9 x 10(6)/kg) and the average number of infused T-lymphocytes was 1.4 x 10(4)/kg. Due to this low T cell number, only a short-term or no prophylaxis of graft-versus-host disease (GVHD) was necessary and no GVHD was seen. A significant GVHD was only seen in patients after add-back of donor T-lymphocytes, which was performed in some patients for prevention of relapse or in patients who showed a transient mixed chimerism. Since the B lymphocyte contamination of the isolated CD34+ cells was low in the range of 0.2%, no Epstein-Barr virus (EBV)-associated lymphoproliferative syndrome was observed. A primary engraftment was seen in 18 patients. Nonengraftment and rejection occurred in three and two patients, respectively. In four of these 5 patients, a second transplant using purified CD34+ cells from the same donor after an immunological reconditioning regimen resulted in a complete and sustained hematopoietic reconstitution. The speed of the immunological recovery was dependent on the number of transplanted CD34+ cells and was more rapid if this number was > 20 x 10(6)/kg. Eleven of the 23 patients are alive and disease free with a median follow-up of 12 months (range 2-30). The main cause of death was relapse (7 patients), and only one fatal infection was seen. Our data suggest that the transplantation of megadoses of haploidentical CD34+ cells is a realistic therapeutic option for patients who otherwise have no suitable donor, and an alternative to the use of unrelated cord blood.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / therapy
  • Antigens, CD34 / analysis
  • Biomarkers / analysis
  • Child
  • Child, Preschool
  • Graft vs Host Disease / etiology
  • Graft vs Host Disease / immunology
  • Haplotypes
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cells / immunology
  • Histocompatibility Testing
  • Humans
  • Immunologic Deficiency Syndromes / therapy
  • Infant
  • Leukemia / therapy
  • Lymphocyte Subsets / immunology*
  • Myelodysplastic Syndromes / therapy
  • Osteopetrosis / therapy

Substances

  • Antigens, CD34
  • Biomarkers