Deferasirox—An Oral Agent for Chronic Iron Overload

Abstract

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. Data Sources: 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. Study Selection and Data Extraction: 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 Other information was obtained from the manufacturer, including unpublished studies in abstract form as well as available data on deferasirox. Data Synthesis: 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 β-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 β-thalassemia. Further studies are needed to confirm its efficacy in other chronic transfusion-requiring diseases.