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


METHODS
This phase IV, single-arm, open-label study over 53 wk evaluated the change in cardiac and liver iron load with deferasirox (up to 40 mg/kg/d), measured by magnetic resonance imaging (MRI).


RESULTS
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 8.2 mg Fe/g dry tissue (dw); P = 0.024], particularly in those with baseline LIC >7 mg Fe/g dw (19.9 to 15.6 mg Fe/g dw; P = 0.002). Furthermore, myocardial T2* significantly increased in patients with LIC <7 mg Fe/g dw, but not in those with a higher LIC. Safety was consistent with previous reports.


CONCLUSIONS
Once-daily deferasirox over 1 yr 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).
['Ho|P Joy|PJ|', 'Tay|Lay|L|', 'Teo|Juliana|J|', 'Marlton|Paula|P|', 'Grigg|Andrew|A|', 'St Pierre|Tim|T|http://orcid.org/0000-0003-4682-4972', 'Brown|Greg|G|', 'Badcock|Caro-Anne|CA|', 'Traficante|Robert|R|', 'Gervasio|Othon L|OL|', 'Bowden|Donald K|DK|']
D000293:Adolescent D000328:Adult D000368:Aged D001565:Benzoates D001803:Blood Transfusion D015913:Chelation Therapy D000077588:Deferasirox D005260:Female D005293:Ferritins D006334:Heart Function Tests D006453:Hemoglobinopathies D006801:Humans D007501:Iron D007502:Iron Chelating Agents D019190:Iron Overload D008279:Magnetic Resonance Imaging D008297:Male D008875:Middle Aged D009206:Myocardium D065227:Transfusion Reaction D016896:Treatment Outcome D014230:Triazoles D055815:Young Adult
cardiac iron deferasirox hepatic iron magnetic resonance imaging iron assessment transfusional siderosis