ABSTRACT
<p><b>OBJECTIVE</b>To evaluate the efficacy and safety of deferasirox in heavily iron-overloaded patients with beta-thalassemia major.</p><p><b>METHODS</b>A single arm, open-label clinical trial was conducted to evaluate the efficacy and safety of deferasirox in the treatment for 23 patients with beta-thalassemia major and heavily iron-overloaded in 3 years follow-up.</p><p><b>RESULTS</b>The 23 patients never received regular chelation before enrolling this trial [the mean baseline of serum ferritin was (5433.96 ± 2873.90) µg/L]. In this trial, a deferasirox dose of 20 mg×kg(-1)×d(-1) could stabilize serum ferritin levels, while of ≥ 30 mg×kg(-1)×d(-1) reduced the levels and achieved negative iron balance. There were no serious adverse events related to the drug. Most common adverse events were mild increases of liver enzyme and serum creatinine levels. Overall, 23 patients could tolerate the drug on schedule and all completed the trial.</p><p><b>CONCLUSION</b>As a new oral iron chelator, deferasirox has a significant efficacy for the treatment of iron overload. The effectiveness is dependent on the courses of treatment and the dose of deferasirox. The single-dose used is safe and tolerated, so deferasirox can remarkably improve life quality of patients.</p>