title: Profile of deferasirox for the treatment of patients with non-transfusion-dependent thalassemia syndromes.
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.
authors.affiliation: UOSD Malattie Rare del Globulo Rosso, Azienda Ospedaliera di Rilievo Nazionale "Antonio Cardarelli", Naples, Italy.
authors.forename: Paolo
authors.initials: P
authors.lastname: Ricchi
authors.affiliation: UOSD Malattie Rare del Globulo Rosso, Azienda Ospedaliera di Rilievo Nazionale "Antonio Cardarelli", Naples, Italy ; UOC Pediatria, Azienda Ospedaliera di Rilievo Nazionale G. Rummo, Benevento, Italy.
authors.forename: Maria
authors.initials: M
authors.lastname: Marsella
mesh_terms: D001565:Benzoates / Q000009:adverse effects / Q000627:therapeutic use*; D015415:Biomarkers / Q000097:blood; D000077588:Deferasirox; D006801:Humans; D007501:Iron / Q000097:blood*; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology; D013577:Syndrome; D013789:Thalassemia / Q000150:complications* / Q000175:diagnosis; D016896:Treatment Outcome; D014230:Triazoles / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D015415:Biomarkers; D007502:Iron Chelating Agents; D014230:Triazoles; D007501:Iron; D000077588:Deferasirox
keywords: deferasirox; iron overload; non-transfusion-dependent thalassemia; profile