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 chelation1,2. Another potential consequence of long-term transfusions is the development of antierythrocyte antibodies, making effective transfusion difficult or, in rare cases, impossible3–6. A potential alternative to transfusion is the use of the nucleoside analogue azacitidine to stimulate transcription of the fetal globin genes by pharmacologic means and thus lead to more effective . . .