Continued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overload

Dudley J. PennellJohn B. PorterMaria Domenica CappelliniLee Lee ChanAmal El-BeshlawyYesim AydinokHishamshah IbrahimChi Kong LiVip ViprakasitMohsen Saleh ElalfyAntonis KattamisGillian SmithDany HabrGabor DomokosBernard RoubertAli TaherRoyal Brompton HospitalUCLUniversita degli Studi di MilanoUniversity of Malaya Medical CentreCairo UniversityEge University Medical SchoolKuala Lumpur HospitalPrince of Wales Hospital Hong KongMahidol UniversityAin Shams UniversityUniversity of AthensNovartis PharmaceuticalsNovartis International AGAmerican University of Beirut2018-05-032018-05-032011-01-01Haematologica. Vol.96, No.1 (2011), 48-5415928721039060782-s2.0-78650996705https://repository.li.mahidol.ac.th/handle/20.500.14594/12780Background 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 β-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 β-thalassemia major and mild, moderate and severe cardiac siderosis. © 2011 Ferrata Storti Foundation.Mahidol UniversityMedicineContinued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overloadArticleSCOPUS10.3324/haematol.2010.031468