Twice-daily deferasirox effective, well-tolerated in paediatric thalassaemia patients

Treatment with deferasirox twice daily, compared with a once-daily regimen, results in significant improvements in the iron chelation efficacy and tolerability among transfusion-dependent paediatric beta-thalassaemia patients, a study has shown.

Forty-one patients participated in this prospective randomized single-blinded parallel study. Those assigned to the intervention group demonstrated a statistically significant mean reduction in serum ferritin levels. While those in the control group had a significant increase in their serum ferritin levels.

Notably, twice-daily dosing of deferasirox was well tolerated by thalassaemia patients relative to a once-daily dosing.

This study included all transfusion-dependent paediatric beta-thalassaemia patients prescribed with deferasirox, who visited the study site for their regular blood transfusions and follow-up. Using a simple block randomization method, the investigators randomly assigned the enrolled patients into the intervention or control group.

Patients in the intervention group switched from once-daily dosing of deferasirox to twice-daily dosing with the same total daily dose. On the other hand, those assigned to the control group continued with the once-daily deferasirox dosing.

The investigators then determined the serum ferritin levels of both groups on the enrolment day and after 6 months of follow-up.

“Deferasirox has proven good efficacy and acceptable safety for the management of thalassaemia patients,” the investigators said. “However, some patients are unresponsive or intolerant to once-daily administration of deferasirox even at a high dose.”

Results of this study provide evidence of the efficacy and tolerability of twice-daily dosing of deferasirox among transfusion-dependent paediatric beta-thalassaemia patients.