title: Deferasirox: pharmacokinetics and clinical experience.
abstract: BACKGROUND
  Iron overload is an inevitable consequence of transfusion therapy for a variety of underlying anemias. Iron overload, without effective chelation, will lead to significant morbidity and mortality. Deferasirox (Exjade®) is an oral tridentate iron chelator used for reducing iron overload.
  
  
  METHODS
  In addition to the pharmacokinetic and pharmacodynamic profile of deferasirox, this review examines the efficacy and safety data from pivotal studies with deferasirox in iron-overloaded patients with various anemias, including thalassemia, sickle cell disease and myelodysplastic syndromes. A qualitative literature search for deferasirox was performed using PubMed and recent key congress publications (EHA and ASH); key search terms were deferasirox, pharmacokinetic, pharmacodynamic, efficacy and safety.
  
  
  CONCLUSIONS
  Based on the current available data, deferasirox treatment is effective with a clinically manageable safety profile although appropriate dosing according to the severity of iron burden and iron intake, together with the careful monitoring of laboratory parameters and adverse events, is recommended. However, despite advances made in the treatment of iron overload, some patients still do not respond adequately to iron chelators, and increased awareness, understanding and further research are still needed.
authors.affiliation: 2a Clinica Pediatrica, Ospedale Regionale Microcitemie, Via Jenner s/n 09121 Cagliari, Italy. renzo.galanello@mcweb.unica.it
authors.forename: Renzo
authors.initials: R
authors.lastname: Galanello
authors.forename: Simona
authors.initials: S
authors.lastname: Campus
authors.forename: Raffaella
authors.initials: R
authors.lastname: Origa
mesh_terms: D000818:Animals; D001565:Benzoates / Q000493:pharmacokinetics* / Q000627:therapeutic use*; D002986:Clinical Trials as Topic / Q000379:methods; D000077588:Deferasirox; D006801:Humans; D007502:Iron Chelating Agents / Q000493:pharmacokinetics / Q000627:therapeutic use; D019190:Iron Overload / Q000188:drug therapy* / Q000378:metabolism*; D019266:Iron, Dietary / Q000009:adverse effects / Q000378:metabolism; D065227:Transfusion Reaction; D014230:Triazoles / Q000493:pharmacokinetics* / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D019266:Iron, Dietary; D014230:Triazoles; D000077588:Deferasirox