Hematopoietic Stem Cell Transplantation in Thalassemia

Luisa Strocchio, Franco Locatelli

Research output: Contribution to journalReview articlepeer-review


Although recent advances in gene therapy are expected to increase the chance of disease cure in thalassemia major, at present hematopoietic stem cell transplantation (HSCT) remains the only consolidated curative approach for this disorder. The widest experience has been obtained in the HLA-matched family donor (MFD) setting, with probabilities of overall and thalassemia-free survival exceeding 90% and 85%, respectively. As for most patients a suitable MFD is not available, alternative donors (HLA-matched unrelated donor, unrelated cord blood, HLA-haploidentical relative) have been increasingly explored, translating into the expansion of the number of patients treatable with HSCT.

Original languageEnglish
Pages (from-to)317-328
Number of pages12
JournalHematology/Oncology Clinics of North America
Issue number2
Publication statusPublished - Apr 2018


  • Cord Blood Stem Cell Transplantation
  • Disease Management
  • Graft vs Host Disease/etiology
  • Hematopoietic Stem Cell Transplantation/adverse effects
  • Histocompatibility Testing
  • Humans
  • Prognosis
  • Risk Assessment
  • Thalassemia/diagnosis
  • Tissue Donors
  • Transplantation Chimera
  • Transplantation Conditioning
  • Transplantation, Homologous
  • Treatment Outcome


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