Dudley J. PennellJohn B. PorterMaria Domenica CappelliniLee Lee ChanAmal El-BeshlawyYesim AydinokHishamshah IbrahimChi Kong LiVip ViprakasitMohsen S. ElalfyAntonis KattamisGillian SmithDany HabrGabor DomokosBernard RoubertAli TaherRoyal Brompton HospitalUCLUniversita degli Studi di MilanoUniversity of Malaya Medical CentreCairo UniversityEge UniversitesiKuala Lumpur HospitalPrince of Wales Hospital Hong KongMahidol UniversityAin Shams UniversityUniversity of AthensNovartis PharmaceuticalsNovartis International AGAmerican University of Beirut2018-06-112018-06-112012-06-01Haematologica. Vol.97, No.6 (2012), 842-84815928721039060782-s2.0-84862141974https://repository.li.mahidol.ac.th/handle/123456789/14800Background Prospective data on cardiac iron removal are limited beyond one year and longer-term studies are, therefore, important. Design and Methods Seventy-one patients in the EPIC cardiac substudy elected to continue into the 3 rd year, allowing cardiac iron removal to be analyzed over three years. Results Mean deferasirox dose during year 3 was 33.6±9.8 mg/kg per day. Myocardial T2*, assessed by cardiovascular magnetic resonance, significantly increased from 12.0 ms ±39.1% at baseline to 17.1 ms ±62.0% at end of study (P < 0.001), corresponding to a decrease in cardiac iron concentration (based on ad hoc analysis of T2*) from 2.43±1.2 mg Fe/g dry weight (dw) at baseline to 1.80 ±1.4 mg Fe/g dw at end of study (P < 0.001). After three years, 68.1% of patients with baseline T2* 10 to < 20 ms normalized (≥20 ms) and 50.0% of patients with baseline T2* > 5 to < 10 ms improved to 10 to < 20 ms. There was no significant variation in left ventricular ejection fraction over the three years. No deaths occurred and the most common investigator-assessed drugrelated adverse event in year 3 was increased serum creatinine (n=9, 12.7%). Conclusions Three years of deferasirox treatment along with a clinically manageable safety profile significantly reduced cardiac iron overload versus baseline and normalized T2* in 68.1% (32 of 47) of patients with T2* 10 to < 20 ms. © 2012 Ferrata Storti Foundation.Mahidol UniversityMedicineDeferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia majorArticleSCOPUS10.3324/haematol.2011.049957