Feasibility of an outpatient HLA haploidentical stem cell transplantation program in children using a reduced-intensity conditioning regimen and CD3-CD19 depletion

Hematology. 2014 Jan;19(1):10-7. doi: 10.1179/1607845413Y.0000000088. Epub 2013 Nov 25.

Abstract

Haploidentical hematopoietic stem cell transplantation (haplo-HSCT) has been developed as an alternative transplant strategy for children with hematological disorders who do not have an HLA-matched donor. We report the analysis of the outcome for 18 consecutive pediatric patients with various hematological diseases, who underwent haplo-HSCT using a reduced-intensity conditioning regimen and CD3/CD19 depletion in an outpatient setting. Twelve of the 18 patients (66.6%) engrafted either transiently or definitively (9 patients engrafted with full donor chimerism and 3 with mixed chimerism). Six patients with acute lymphoblastic leukemia were disease-free between 2 and 35 months (median 25 months) post-HSCT. The overall survival was 33.3% with a median of 25 months (range 2-35). Our results suggest that haplo-HSCT can be a feasible therapeutic alternative for children who do not have a suitable family donor or available cord blood units. These results also demonstrate that it is possible to perform this regimen on an outpatient basis.

Keywords: Children; Haploidentical; Outpatient; Transplantation.

MeSH terms

  • Adolescent
  • Adult
  • Antigens, CD19 / analysis
  • Antigens, CD19 / immunology*
  • CD3 Complex / analysis
  • CD3 Complex / immunology*
  • Child
  • Female
  • HLA Antigens / immunology*
  • Haploidy
  • Hematopoietic Stem Cell Transplantation / methods*
  • Humans
  • Lymphocyte Depletion / methods*
  • Male
  • Middle Aged
  • Survival Analysis
  • Transplantation Chimera
  • Transplantation Conditioning / methods*
  • Transplantation, Autologous
  • Young Adult

Substances

  • Antigens, CD19
  • CD3 Complex
  • HLA Antigens