Advances in the Allogeneic Transplantation for Thalassemia

Blood Rev. 2008 Mar;22(2):53-63. doi: 10.1016/j.blre.2007.10.001. Epub 2007 Nov 26.

Abstract

Hematopoietic stem cell transplantation (HSCT) remains the only curative option for patients with thalassemia. Current results of transplantation in patients aged less than 17 years from matched related donors offer 80% to 87% probability of cure according to risk classes. Adult thalassemics treated with myeloablative conditioning continue to have inferior results because of their advanced stage of disease. With the introduction of high-resolution tissue typing techniques transplant centres worldwide are able to offer allogeneic HSCT to a much larger cohort of patients who could not benefit from transplantation because of lack of matched family donor. Although limited number of patients treated, results of transplant from unrelated matched donors are comparable to those obtained using sibling donors. Graft failure or rejection remains a significant cause of transplant failure in patients with thalassemia making difficult to perform reduced intensity conditioning regimens. Mixed chimerism is a common phenomenon after transplantation and is a risk factor for rejection. Ex-thalassemics still carry the clinical complications acquired during years of transfusion and chelation therapy. Longer follow-up and management of these complications in ex-thalassemics are essential.

Publication types

  • Review

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Hematopoietic Stem Cell Transplantation / methods*
  • Histocompatibility Testing
  • Humans
  • Infant
  • Thalassemia / therapy*
  • Transplantation Chimera
  • Transplantation, Homologous / methods