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Deferasirox: appraisal of safety and efficacy in long-term therapy

Authors Chaudhary P, Pullarkat V

Received 10 June 2013

Accepted for publication 17 July 2013

Published 5 August 2013 Volume 2013:4 Pages 101—110

DOI https://doi.org/10.2147/JBM.S35478

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 4


Preeti Chaudhary, Vinod Pullarkat

Jane Ann Nohl Division of Hematology, University of Southern California Keck School of Medicine, Los Angeles, CA, USA

Abstract: Deferasirox is a once-daily, oral iron chelator that is widely used in the management of patients with transfusional hemosiderosis. Several Phase II trials along with their respective extension studies as well as a Phase III trial have established the efficacy and safety of this novel agent in transfusion-dependent patients with β-thalassemia, sickle-cell disease and bone marrow-failure syndromes, including myelodysplastic syndrome and aplastic anemia. Data from various clinical trials show that a deferasirox dose of 20 mg/kg/day stabilizes serum ferritin levels and liver iron concentration, while a dose of 30–40 mg/kg/day reduces these parameters and achieves negative iron balance in red cell transfusion-dependent patients with iron overload. Across various pivotal clinical trials, deferasirox was well tolerated, with the most common adverse events being gastrointestinal disturbances, skin rash, nonprogressive increases in serum creatinine, and elevations in liver enzyme levels. Longer-term extension studies have also confirmed the efficacy and safety of deferasirox. However, it is essential that patients on deferasirox therapy are monitored regularly to ensure timely management for any adverse events that may occur with long-term therapy.

Keywords: deferasirox, iron overload, thalassemia, sickle-cell disease, myelodysplastic syndrome

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