Hematopoietic stem cell transplantation in patients with transfusion-dependent β-thalassemia. Review article

Thalassemia is the most common form of hereditary anemia from the hemoglobinopathy group. The genetic disorder underlying thalassemia leads to impaired erythrocyte maturation, hemolysis, and the development of ineffective erythropoiesis with erythroid gland hyperplasia in the bone marrow and extramedullary. Regular blood transfusions and chelator therapy are standard therapy for patients with b-thalassemia. This method increases life expectancy, but does not improve its quality and does not cure the disease. Currently, allogeneic hematopoietic stem cell transplantation remains the only radical treatment for thalassemia. The paper discusses the historical aspects of the development of allogeneic hematopoietic stem cell transplantation in the context of transfusion-dependent form of b-thalassemia treatment.