The Current Status of β-Thalassemia Major in Mainland China

Abstract

β-Thalassemia (β-thal) is caused by a decrease in the production of β-globin chains that is a genetic disorder worldwide. The diagnosis of β-thal major (β-TM) depends on clinical manifestations, laboratory investigations, family history and genetic analysis. Patients with β-TM require long-term blood transfusion and chelation therapy. Hematopoietic stem cell transplantation is the only strategy for curing this disease. Many patients with β-TM cannot get regular diagnosis and treatment in developing countries. With economic improvement and medical technology development, a great progress has been made in Mainland China. In this study, we describe the current status of diagnosis and treatment of β-thal in Mainland China.

Modified conditioning regimen improves outcomes of unrelated donor peripheral blood stem cell transplantation for β‐thalassaemia major patients

“…Currently, it is particularly difficult to identify compatible donors in China due to the nation's one‐child policy. Moreover, many patients with TM cannot financially afford long‐term supportive care with normative blood transfusions and iron chelation . Thus, although thalassaemia‐free survival (TFS) rates have increased to 52–82% following HSCT, an alternative source of donors is urgently needed.…”