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.