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Profile of deferasirox for the treatment of patients with non-transfusion-dependent thalassemia syndromes

Authors Ricchi P, Marsella M

Received 26 August 2015

Accepted for publication 25 November 2015

Published 16 December 2015 Volume 2015:9 Pages 6475—6482

DOI https://doi.org/10.2147/DDDT.S40694

Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Prof. Dr. Wei Duan


Paolo Ricchi,1 Maria Marsella1,2

1UOSD Malattie Rare del Globulo Rosso, Azienda Ospedaliera di Rilievo Nazionale “Antonio Cardarelli”, Naples, 2UOC Pediatria, Azienda Ospedaliera di Rilievo Nazionale G. Rummo, Benevento, Italy

Abstract: It has been clearly shown that iron overload adds progressively significant morbidity and mortality in patients with non-transfusion-dependent thalassemia (NTDT). The lack of physiological mechanisms to eliminate the excess of iron requires effective iron chelation therapy. The reduced compliance to deferoxamine and the risk of severe hematological adverse events during deferiprone treatment have limited the use of both these drugs to correct iron imbalance in NTDT. According to the principles of evidence-based medicine, following the demonstration of the effectiveness and the safety of deferasirox (Exjade®) in a prospective, randomized, controlled trial, deferasirox was approved by the US Food and Drug Administration in May 2013 for the treatment of iron overload associated with NTDT. This review, assessing the available scientific literature, will focus on the profile of DFX in the treatment of non-transfusional hemosiderosis in patients with NTDT.

Keywords:
non-transfusion-dependent thalassemia, deferasirox, profile, iron overload

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