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.
Hematopoietic Stem Cell Transplantation in Thalassemia / Strocchio, L.; Locatelli, F.. - In: HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA. - ISSN 0889-8588. - 32:2(2018), pp. 317-328. [10.1016/j.hoc.2017.11.011]
Hematopoietic Stem Cell Transplantation in Thalassemia
Locatelli F.
2018
Abstract
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
