title: Efficacy of deferasirox in children with β-thalassemia: single-center 3 year experience.
abstract: 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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Pediatric Hematology/Oncology Clinic, Eskisehir State Hospital, Eskisehir, Turkey.
authors.forename: Ali
authors.initials: A
authors.lastname: Aycicek
authors.forename: Ahmet
authors.initials: A
authors.lastname: Koc
authors.forename: Mahmut
authors.initials: M
authors.lastname: Abuhandan
mesh_terms: D000293:Adolescent; D001565:Benzoates / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D005260:Female; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008297:Male; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000188:drug therapy*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox
keywords: deferasirox; iron overload; iron-chelating agents; treatment outcome; β-thalassemia