Effect of deferasirox on iron overload in patients with transfusion-dependent haemoglobinopathies

Background: Patients with haematopoietic disorders requiring long-term blood transfusions are at risk of iron overload. This study aimed to investigate the efficacy and safety of long-term deferasirox monotherapy in patients with transfusion-dependent anaemia in the routine clinical practice setting. Methods: This was a retrospective analysis of patients who commenced deferasirox therapy at the Hospital Bianchi Melacrino Morelli in Reggio Calabria, Italy. Data collected included cardiac and hepatic iron load (assessed by magnetic resonance imaging); left ventricular ejection fraction (LVEF). Patients were divided into two groups for analysis: group A (baseline information collected prior to deferasirox initiation) and group B (baseline information collected after deferasirox initiation). Results: Forty-six patients were included (group A: n = 25; group B: n = 21). The overall population was 63% male, with a mean age of 33 years. The majority of patients (65%) had thalassaemia major. In the overall population, cardiac iron levels between the baseline and first follow-up visits improved in both groups A and B (292 vs. 32.5 ms; p = 0.04 and 28.4 vs. 31.4 ms; p = 0.038). Liver iron levels improved significantly from baseline to visit 1 in group A (7.2 vs. 12.1 ms; p<0.004) and from baseline to visit 3 (6.9 vs. 10.7; p = 0.049) in group B. Generally, there was no correlation between cardiac and liver iron levels. LVEF remained stable throughout the study period. Deferasirox was well tolerated and was not associated with significant adverse events. Conclusion: Long-term treatment with deferasirox is effective and safe in patients with transfusion-dependent haemoglobinopathies monitored in the clinical practice setting. (C) 2015 Published by Elsevier Inc.