Efficacy and Tolerability of Twice-Daily Dosing Schedule of Deferasirox in Transfusion-Dependent Paediatric Beta-Thalassaemia Patients: A Randomized Controlled Study

• Published in: Journal of pharmacy practice 2023-08, Vol.36 (4), p.749-755
• Main Authors: Panachiyil, George Mathew, Babu, Tirin, Sebastian, Juny, Ravi, Mandyam Dhati
• Format: Article
• Language: English
• Subjects: Benzoates - adverse effects; beta-Thalassemia - drug therapy; Child; Deferasirox; Ferritins; Humans; Iron Chelating Agents - adverse effects; Iron Overload; Prospective Studies; Thalassemia; Triazoles - adverse effects
• ISSN: 0897-1900; 1531-1937
• DOI: 10.1177/08971900211038301

Background: Deferasirox has proved good efficacy and acceptable safety for the management of thalassaemia patients. However, some patients are unresponsive or intolerant to once-daily administration of deferasirox even at a high dose. The current study evaluated the effectiveness and tolerability of twice-daily dosing of deferasirox among transfusion-dependent paediatric beta-thalassaemia patients. Methods: This prospective randomized single-blinded parallel study included all transfusion-dependent paediatric beta-thalassaemia patients prescribed with deferasirox, who visit the study site for their regular blood transfusions and follow-up. The enrolled patients were randomized into intervention and control groups by using a simple block randomization method. In the intervention group, the once-daily dosing of deferasirox was changed to twice-daily dosing with the same total daily dose. Whereas, in the control group, the patients continued with the once-daily deferasirox dosing. The serum ferritin levels of both groups were determined on the enrolment day and after 6 months of follow-up. Results: Forty-one patients were included for analysis. A statistically significant mean decrease in serum ferritin levels was detected in the intervention group, while the serum ferritin levels of the control group significantly increased from baseline. The twice-daily dosing of deferasirox was better tolerated by the thalassaemia patients when compared to once-daily dosing. Conclusion: This study concludes that twice-daily dosing of deferasirox with the same total daily dose significantly enhances the iron chelation efficacy and tolerability among transfusion-dependent paediatric beta-thalassaemia patients when compared to once-daily regimen.