Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors

Bone Marrow Transplant. 2016 Jun;51(6):813-8. doi: 10.1038/bmt.2016.7. Epub 2016 Feb 15.

Abstract

Thalassemia-free survival after allogeneic stem cell transplantation (SCT) is about 80-90% with either matched-related or -unrelated donors. We explored the use of a mismatched-related ('haplo- ') donor. All patients received two courses of pretransplant immunosuppressive therapy (PTIS) with fludarabine (Flu) and dexamethasone (Dxm). After two courses of PTIS, a conditioning regimen of rabbit antithymocyte globulin, Flu and IV busulfan (Bu) was given followed by T-cell-replete peripheral blood progenitor cells. GvHD prophylaxis consisted of cyclophosphamide (Cy) on days SCT +3 and +4 (post-Cy), and on day SCT +5 tacrolimus or sirolimus was started together with a short course of mycophenolate mofetil. Thirty-one patients underwent haplo-SCT. Their median age was 10 years (range, 2-20 years). Twenty-nine patients engrafted with 100% donor chimerism. Two patients suffered primary graft failure. Median time to neutrophil engraftment was 14 days (range, 11-18 days). Five patients developed mild to moderate, reversible veno-occlusive disease, while nine patients developed acute GvHD grade II. Only five patients developed limited-chronic GvHD. Projected overall and event-free survival rates at 2 years are 95% and 94%, respectively. The median follow up time is 12 months (range, 7-33 months).

MeSH terms

  • Adolescent
  • Blood Component Removal
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Graft Survival
  • Hematopoietic Stem Cell Transplantation / methods*
  • Hematopoietic Stem Cell Transplantation / mortality
  • Hemoglobin E
  • Homozygote
  • Humans
  • Immunosuppressive Agents / therapeutic use
  • Infant
  • Peripheral Blood Stem Cell Transplantation / methods*
  • Peripheral Blood Stem Cell Transplantation / mortality
  • Survival Rate
  • Transplantation Conditioning / methods
  • Transplantation, Haploidentical / methods*
  • Transplantation, Haploidentical / mortality
  • Young Adult
  • beta-Thalassemia / therapy*

Substances

  • Immunosuppressive Agents
  • Hemoglobin E