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Cardiac iron load and function in transfused patients treated with deferasirox (the MILE study)
Author(s) -
Ho P. Joy,
Tay Lay,
Teo Juliana,
Marlton Paula,
Grigg Andrew,
St Pierre Tim,
Brown Greg,
Badcock CaroAnne,
Traficante Robert,
Gervasio Othon L.,
Bowden Donald K.
Publication year - 2017
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1111/ejh.12793
Subject(s) - deferasirox , medicine , mile , cardiac function curve , heart failure , thalassemia , physics , astronomy
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: NCT 00673608).