Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients

Abstract

Objective: Minimal data are available on the combined two oral iron chelators in β-thalassemia major (β-TM). Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and deferasirox (DFX) were studied.

Methods: A prospective randomized trial (NCT01511848) was conducted on 96 young β-TM patients with severe iron overload. Patients were randomized to receive either DFP with DFO (arm 1) or DFP and DFX (arm 2). Efficacy endpoints were the difference between two groups in the change of serum ferritin (SF), liver iron concentration (LIC), cardiac MRI, and quality of life (QoL).

Results: In both arms, SF and LIC at 12 months were significantly lower, and geometric mean cardiac T2* was higher compared to baseline. On regression analysis of change in each studied variable against time, significant difference between slopes of the two groups regarding cardiac T2* (P = 0.001 with more improvement in DFP/DFX patients) was found with no significant difference in the slopes of SF and LIC (P = 0.218 and 0.340).

Conclusion: Both iron chelation combination regimens were equally effective in reducing iron overload and improving QoL.DFP/DFX combination proved superior in improving cardiac T2*, treatment compliance, and patients satisfaction with no greater adverse events.

Keywords: Cardiac MRI; combined oral iron chelators; liver iron; quality of life; thalassemia major.