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Treatment for beta-thalassemia with hematopoietic stem cell transplantation.

??Abstract??Allogeneic hematopoietic stem cell transplantation ??HSCT?? is the only curative therapy for severe beta-thalassemia major. Patients with an available human leukocyte antigen ??HLA?? identical sibling donor should be offered HSCT as soon as possible before development of iron overload and transfusion associated complications?? and the cure rate was up to 80%??90%. High-resolution HLA typing matched unrelated donor transplants also achieved good results. HSCT from HLA-mismatched relatives or HLA-phenotypically-identical donor is an option to be performed in expert centers. Optimization of conditioning regimen and techniques to control transplant-related complications will further improve outcomes.