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

BackgroundIron chelation therapy is an important component in the management of patients with -thalassemia. MethodsThe study included 87 children with transfusion-dependent -thalassemia aged 2-17 years (mean, 8.2 4.1 years), 49 (56%) of whom were male. The patients received deferasirox 9-40mg/kg per day as a single dose for 36months. They were clinically and laboratory monitored. ResultsThe 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 +/- 7.6mg/kg per day at 0, 12, 24 and 36months, respectively. Mean (median) serum ferritin level was found to increase progressively during the first 22months of treatment, from 3.161 +/- 1.683ng/mL (2.760ng/mL) to 3.679 +/- 1.997ng/mL (3.071ng/mL; P < 0.001) and then decreased gradually to 2.907 +/- 1.436ng/mL (2.670ng/mL; P = 0.023) at 36months. ConclusionDeferasirox is safe and well tolerated; doses 21-24mg/kg per day were not able to maintain stable iron balance, but 30mg/kg per day was able to reduce iron in regularly transfused pediatric patients.