Thalassemia incorporates a broad clinical spectrum characterized by decreased or absent production of normal
hemoglobin leading to decreased red blood cell survival and ineffective erythropoiesis. Chronic
iron overload remains an inevitable complication resulting from regular
blood transfusions (transfusion-dependent) and/or increased
iron absorption (mainly non-transfusion-dependent
thalassemia), requiring adequate treatment to prevent the significant associated morbidity and mortality.
Iron chelation therapy has become a cornerstone in the management of
thalassemia patients, leading to improvements in their outcome and quality of life.
Deferasirox (DFX), an oral
iron chelating agent, is approved for use in transfusion dependent and non-transfusion-dependent
thalassemia and has shown excellent efficacy in this setting. We herein present an updated review of the role of
deferasirox in
thalassemia, exploring over a decade of experience, which has documented its effectiveness and convenience; in addition to its manageable safety profile.