Pennell, Dudley J.Porter, John B.Cappellini, Maria DomenicaChan, Lee LeeEl-Beshlawy, AmalAydinok, YesimIbrahim, HishamshahLi, Chi-KongViprakasit, VipElalfy, Mohsen SalehKattamis, AntonisSmith, GillianHabr, DanyDomokos, GaborRoubert, BernardTaher, Ali2019-10-272019-10-2720110390-60781592-8721https://doi.org/10.3324/haematol.2010.031468https://hdl.handle.net/11454/45058Background The efficacy of cardiac iron chelation in transfusion-dependent patients has been demonstrated in one-year prospective trials. Since normalization of cardiac T2* takes several years, the efficacy and safety of deferasirox was assessed for two years in patients with beta-thalassemia major in the cardiac sub-study of the EPIC trial. Design and Methods Eligible patients with myocardial T2* greater than 5 to less than 20 ms received deferasirox, with the primary endpoint being the change in T2* from baseline to two years. Results Baseline myocardial T2* was severe (>5 to <10 ms) in 39 patients, and moderate-to-mild (10 to <20 ms) in 62 patients. Mean deferasirox dose was 33.1+/-3.7 mg/kg/d in the one-year core study increasing to 36.1+/-7.7 mg/kg/d during the second year of treatment. Geometric mean myocardial T2* increased from a baseline of 11.2 to 14.8 ms at two years (P<0.001). In patients with moderate-to-mild baseline T2*, an increase was seen from 14.7 to 20.1 ms, with normalization (20 ms) in 56.7% of patients. In those with severe cardiac iron overload at baseline, 42.9% improved to the moderate-to-mild group. The incidence of drug-related adverse events did not increase during the extension relative to the core study and included (>= 5%) increased serum creatinine, rash and increased alanine aminotransferase. Conclusions Continuous treatment with deferasirox for two years with a target dose of 40 mg/kg/d continued to remove iron from the heart in patients with beta-thalassemia major and mild, moderate and severe cardiac siderosis. (Clinicaltrials.gov identifier: NCT 00171821)en10.3324/haematol.2010.031468info:eu-repo/semantics/openAccessbeta-thalassemia majorcardiac iron overloaddeferasiroxiron chelationmyocardial T2*Article9614854WOS:00028690240001021071497Q1