Iranian experience of deferasirox (Exjade®) in transfusion-dependent patients with iron overload: what is the most effective dose based on serum ferritin levels?

Background: The aim of this study was to evaluate the efficacy and safety of deferasirox in patients from Iran. Methods: This was a retrospective, observational study in regularly transfused, iron-overloaded patients who received deferasirox 20-38 mg/kg/day for up to 12 months. Changes in serum ferritin were assessed as follows: from baseline to 3 months with deferasirox doses of 20-24 mg/kg/day; from 3 to 6 months with doses of 25-29 mg/kg/day; and from 6 to 12 months with doses of 30-38 mg/kg/day. The safety of deferasirox was evaluated monthly. Patients' satisfaction with treatment was assessed after 9 months. Results: One hundred and nineteen patients were included. Overall mean serum ferritin levels were significantly decreased from baseline after 12 months of deferasirox therapy (2510 +/- 1210 to 1665 +/- 1240 ng/ml; P < 0.002). A significant decrease was observed once doses were increased to >= 30 mg/kg/day (P = 0.0003). Most adverse events were mild and observed at the dose of 20-24 mg/kg. Only one patient discontinued treatment. Around 90% of patients were satisfied with therapy. Conclusion: This is the first study evaluating deferasirox in heavily iron-overloaded patients from Iran and confirms that deferasirox is effective and well tolerated; however, dose increases to >= 30 mg/kg/day should be considered if efficacy is insufficient.