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Efficacy of deferasirox in children with β‐thalassemia: Single‐center 3 year experience
Author(s) -
Aycicek Ali,
Koc Ahmet,
Abuhandan Mahmut
Publication year - 2014
Publication title -
pediatrics international
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.49
H-Index - 63
eISSN - 1442-200X
pISSN - 1328-8067
DOI - 10.1111/ped.12323
Subject(s) - medicine , deferasirox , nausea , adverse effect , gastroenterology , creatinine , thalassemia , abdominal pain , vomiting , single center , surgery
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.