Purified T-depleted, CD34+ Peripheral Blood and Bone Marrow Cell Transplantation From Haploidentical Mother to Child With Thalassemia

Blood. 2010 Feb 11;115(6):1296-302. doi: 10.1182/blood-2009-05-218982. Epub 2009 Nov 6.


Fetomaternal microchimerism suggests immunological tolerance between mother and fetus. Thus, we performed primary hematopoietic stem cell transplantation from a mismatched mother to thalassemic patient without an human leukocyte antigen-identical donor. Twenty-two patients with thalassemia major were conditioned with 60 mg/kg hydroxyurea and 3 mg/kg azathioprine from day -59 to -11; 30 mg/m(2) fludarabine from day -17 to -11; 14 mg/kg busulfan starting on day -10; and 200 mg/kg cyclophosphamide, 10 mg/kg thiotepa, and 12.5 mg/kg antithymocyte globulin daily from day -5 to -2. Fourteen patients received CD34(+)-mobilized peripheral blood and bone marrow progenitor cells; 8 patients received marrow graft-selected peripheral blood stem cells CD34(+) and bone marrow CD3/CD19-depleted cells. T-cell dose was adjusted to 2 x 10(5)/kg by fresh marrow cell addback at the time of transplantation. Both groups received cyclosporine for graft-versus-host disease prophylaxis for 2 months after transplantation. Two patients died (cerebral Epstein-Barr virus lymphoma or cytomegalovirus pneumonia), 6 patients reject their grafts, and 14 showed full chimerism with functioning grafts at a median follow-up of 40 months. None of the 14 patients who showed full chimerism developed acute or chronic graft-versus-host disease. These results suggest that maternal haploidentical hematopoietic stem cell transplantation is feasible in patients with thalassemia who lack a matched related donor.

MeSH terms

  • Adolescent
  • Adult
  • Antigens, CD34 / metabolism*
  • Bone Marrow Transplantation*
  • Child
  • Child, Preschool
  • Feasibility Studies
  • Flow Cytometry
  • Graft Survival / immunology
  • Graft vs Host Disease / prevention & control*
  • HLA Antigens / metabolism
  • Humans
  • In Situ Hybridization, Fluorescence
  • Lymphocyte Depletion*
  • Middle Aged
  • Mothers
  • Peripheral Blood Stem Cell Transplantation*
  • Pilot Projects
  • Polymerase Chain Reaction
  • Prospective Studies
  • T-Lymphocytes*
  • Thalassemia / therapy*
  • Transplantation Conditioning
  • Transplantation, Homologous
  • Young Adult


  • Antigens, CD34
  • HLA Antigens