Deferasirox for the treatment of iron overload in non-transfusion-dependent thalassemia

Expert Review of Hematology
Ali TaherMaria Domenica Cappellini


Non-transfusion-dependent thalassemia (NTDT) defines a group of patients who do not require regular transfusions for survival, but are at significant risk of iron accumulation from underlying disease-related mechanisms distinct from transfusional iron overload. Management of iron overload in NTDT has received little attention compared with that of β-thalassemia major, despite evidence of significant iron-induced complications with advancing age. The efficacy and safety of the iron chelator deferasirox in NTDT has been evaluated in two pilot studies and the first prospective, randomized, placebo-controlled study (THALASSA) of any chelator in NTDT. Treatment with deferasirox for up to 2 years yielded a sustained reduction in iron burden, with a clinically manageable safety profile. Following these trial data, deferasirox is the first iron chelator approved for use in NTDT patients, and with NTDT guidelines now available, physicians are better equipped to achieve effective monitoring and management of iron burden in NTDT.


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Related Concepts

Blood Transfusion
Iron Chelating Agents
Iron Overload
Blood Transfusion
Chelating Agents
Clinical Trials

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