Research advances in transplantation for thalassemia major / 中国当代儿科杂志

ABSTRACT

Thalassemia is an inherited blood disorder caused by disordered globin chain synthesis due to mutations in the regulatory genes for hemoglobin. At present, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is recognized as the only curative method for treatment. Through the revolution of pretransplantation regimens and selection of donor and source of stem cells, patients' survival has been greatly improved. This article reviews the development of transplantation for thalassemia and related research advances, in order to provide suitable treatment options for clinical application.