Deferasirox--an oral agent for chronic iron overload.
OBJECTIVE
To review the available literature on the pharmacology, pharmacokinetics, efficacy, toxicology, adverse effects, drug interactions, and dosage guidelines for deferasirox, an oral iron chelator, in Phase III trials.


METHODS
Reviewers searched the following databases for English-language studies: MEDLINE (1966-April 2006), International Pharmaceutical Abstracts (1970-April 2006), and the Cochrane Library Database. Key search terms included iron chelation, chelation, iron overload, deferasirox, and ICL670.


METHODS
Data on efficacy, toxicology, adverse effects, and pharmacokinetics for deferasirox were obtained from randomized, open-label, blinded clinical trials. Other information was obtained from the manufacturer, including unpublished studies in abstract form as well as available data on deferasirox.


RESULTS
Deferasirox is an orally active iron chelator. In clinical trials, deferasirox demonstrated efficacy at dosages of 20 and 30 mg/kg/day in treating iron overload in patients with beta-thalassemia. Deferasirox has been studied in patients older than 2 years and appears to be safe, with the most common adverse effects reported being mild, transient nausea, gastrointestinal disturbances, and rash. There were no reports of serious adverse effects in trials to date.


CONCLUSIONS
Deferasirox represents a new approach to the management of chronic iron overload in patients with chronic anemias who require blood transfusions. The available literature suggests that deferasirox is safe and as effective as the current standard of therapy at dosages of 20-30 mg/kg/day for beta-thalassemia. Further studies are needed to confirm its efficacy in other chronic transfusion-requiring diseases.
Vanorden
Heidi E
HE
Center for Cancer and Blood Disorders, Children's Medical Center of Dallas, TX, USA.
Hagemann
Tracy M
TM
D001565:Benzoates / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use* / Q000633:toxicity; D002986:Clinical Trials as Topic; D000077588:Deferasirox; D004347:Drug Interactions; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use* / Q000633:toxicity; D019190:Iron Overload / Q000188:drug therapy*; D014230:Triazoles / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use* / Q000633:toxicity
D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox