Efficacy of deferasirox in children with β‐thalassemia: Single‐center 3 year experience

Background Iron chelation therapy is an important component in the management of patients with β‐thalassemia. Methods The study included 87 children with transfusion‐dependent β‐thalassemia aged 2–17 years (mean, 8.2 ± 4.1 years), 49 (56%) of whom were male. The patients received deferasirox 9–40 mg/kg per day as a single dose for 36 months. They were clinically and laboratory monitored. Results The treatment was generally well tolerated. Drug‐related adverse events, including abdominal pain (14.9%) and nausea (5.8%), high alanine aminotransferase more than double the upper limit of normal (5.8%), and non‐progressive rise in serum creatinine (2.3%), were generally mild to moderate, transient, and reduced in frequency over time. Two patients discontinued treatment due to severe abdominal pain and nausea. Mean deferasirox dose was calculated as 21.2 ± 8.6, 23.7 ± 8.1, 30.7 ± 8.2 and 32.4 ± 7.6 mg/kg per day at 0, 12, 24 and 36 months, respectively. Mean (median) serum ferritin level was found to increase progressively during the first 22 months of treatment, from 3.161 ± 1.683 ng/mL (2.760 ng/mL) to 3.679 ± 1.997 ng/mL (3.071 ng/mL; P < 0.001) and then decreased gradually to 2.907 ± 1.436 ng/mL (2.670 ng/mL; P = 0.023) at 36 months. Conclusion Deferasirox is safe and well tolerated; doses 21–24 mg/kg per day were not able to maintain stable iron balance, but ≥30 mg/kg per day was able to reduce iron in regularly transfused pediatric patients.