Efficacy of Oral Deferasirox by Twice-daily Dosing in Patients with Transfusion-dependent Beta Thalassemia

Background and Patients with Beta thalassemia major need consistent blood transfusion from early years of life. Deferasirox is used as an oral chelating agent (once daily) to excrete excess iron. This study aimed to compare the efficacy of deferasirox twice daily and the usual once daily dosing. This before after clinical trial was performed in 2013-2014 in patients who were at least 2 years of age and received only deferasirox as the chelating agent. All patients had received deferasirox for at least six month once daily. The last ferritin before entering the study and the mean deferasirox daily dose during the previous six months were considered as baseline ferritin and deferasirox dose, respectively. Laboratory tests were performed including CBC-diff, serum ferritin, Creatinine (Cr), Aspartate Aminotransferase (AST) and Alanine Aminotransferase (ALT) to evaluate the efficacy and safety of deferasirox. A total of 21 transfusion-dependent patients (mean age: 21±6 years old) were included of whom 67% were male. The mean ferritin level decreased significantly from 1814± 922 ng/ml to 1472± 907 ng/ml (P= 0.02). There were no any significant changes in AST, ALT and Cr levels compared to baseline values. Twice daily dosing of deferasirox was associated with more decrease in ferritin level compared to baseline single daily dose values without any hepatic or renal adverse effects.