Evaluation of Efficacy, Safety, and Satisfaction Taking Deferasirox Twice Daily Versus Once Daily in Patients With Transfusion-Dependent Thalassemia. Issue 1 (January 2020)

• Record Type: Journal Article
• Authors: Karimi, Mehran; Haghpanah, Sezaneh; Bahoush, Gholamreza; Ansari, Shahla; Azarkeivan, Azita; Shahsavani, Amin; Bazrafshan, Asghar; Jangjou, Ali
• Is Part Of: Journal of pediatric hematology/oncology. Volume 42:Issue 1(2020)
• Publication Date: 2020-01
• Subjects: deferasirox -- thalassemia -- safety -- efficacy; Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; 618.9215
• DOI: 10.1097/MPH.0000000000001596
• Languages: English
• ISSNs: 1077-4114

Abstract

Abstract : Objective: Deferasirox is a once-daily oral iron-chelation agent approved by the US Food and Drug Administration in November 2005. The authors aimed to evaluate efficacy, safety, and satisfaction of patients regarding twice-daily dose of deferasirox in patients with thalassemia who are resistant to once-daily regimen. Methods: In this historical cohort multicenter study, 34 patients with beta-thalassemia major resistant or intolerant to once-daily dose of deferasirox (35 mg/kg/d) were investigated in 2016. Patients were registered at 3 thalassemia referral centers in Shiraz, southern Iran and Tehran, the capital of Iran. All patients were followed for 1 year and monitored by regular physical examination, laboratory data, serum ferritin levels, and heart and liver T2 magnetic resonance imaging. Results: Mean age of thalassemia patients was 25.6±8.1 (8 to 40) years, including 22 female individuals and 12 male individuals. Serum ferritin levels significantly decreased during the study period (2021±955 at baseline vs. 1228±894 at the end of the study, P <0.001). Liver T2 magnetic resonance imaging of the patients demonstrated a significant improvement during the study. 73.3% of patients showed normal values at the end of study compared with 28.1% at the baseline ( P <0.001). Drug side effects were reported only in 2 patients (5.8%) including 1 patient with abdominal pain and 1 with leukopenia and thrombocytopenia. Conclusions: It seems that deferasirox can be used with increased dose and twice daily with acceptable efficacy in unresponsive or intolerant thalassemia patients to once-daily dose. Close monitoring of the patients is necessary to detect and manage any possible adverse events.