Treatment with Azacitidine of Patients with End-Stage β-Thalassemia

Abstract

Regular erythrocyte transfusions form the cornerstone of treatment for patients with severe β-thalassemia. Despite the use of iron-chelation therapy, fatal iron overload develops in many patients, even when they are under supervision and taking part in studies designed to test the effectiveness of intense chelation^1,2. Another potential consequence of long-term transfusions is the development of antierythrocyte antibodies, making effective transfusion difficult or, in rare cases, impossible^3-6.