Cardiac iron load and function in transfused patients treated with deferasirox (the MILE study)

P. Joy Ho, Lay Tay, Juliana Teo, Paula Marlton, Andrew Grigg, Tim St Pierre, Greg Brown, Caro‐Anne Badcock, Robert Traficante, Othon L. Gervasio, Donald K. Bowden

To assess the effect of iron chelation therapy with deferasirox on cardiac iron and function in patients with transfusion-dependent thalassemia major, sickle-cell disease (SCD) and myelodysplastic syndromes (MDS). This phase IV, single-arm, open-label study over 53 weeks evaluated change in cardiac and liver iron load with deferasirox (up to 40mg/kg/day), measured by magnetic resonance imaging (MRI). Cardiac iron load (myocardial T2*) significantly improved (P=0.002) overall (N=46; n=36 thalassemia major, n=4 SCD, n=6 MDS). Results were significant for patients with normal and moderate baseline cardiac iron (P=0.017 and P=0.015, respectively) but not in the five patients with severe cardiac iron load. Liver iron concentration (LIC) significantly decreased overall (mean LIC 10.4 to 8.2mg Fe/g dry tissue [dw]; P=0.024), particularly in those with baseline LIC >7mg Fe/g dw (19.9 to 15.6mg Fe/g dw; P=0.002). Furthermore, myocardial T2* significantly increased in patients with LIC <7mg Fe/g dw but not in those with a higher LIC. Safety was consistent with previous reports. Once-daily deferasirox over 1year significantly increased myocardial T2* and reduced LIC. This confirms that single-agent deferasirox is effective in the management of cardiac iron, especially for patients with myocardial T2* >10 ms. Clinicaltrials . gov identifier:NCT00673608. This article is protected by copyright. All rights reserved.