@article {117527, title = {Continued improvement in myocardial T2* over two years of deferasirox therapy in β-thalassemia major patients with cardiac iron overload.}, journal = {Haematologica}, volume = {96}, year = {2011}, month = {2011 Jan}, pages = {48-54}, abstract = {

BACKGROUND: 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 {\textpm} 3.7 mg/kg/d in the one-year core study increasing to 36.1 {\textpm} 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. (Clinicaltrials.gov identifier: NCT 00171821).

}, keywords = {Adolescent, Adult, Benzoates, beta-Thalassemia, Child, Female, Ferritins, Humans, Iron, Iron Chelating Agents, Iron Overload, Magnetic Resonance Imaging, Male, Middle Aged, Myocardium, Prognosis, Prospective Studies, Triazoles, Ventricular Function, Left, Young Adult}, issn = {1592-8721}, doi = {10.3324/haematol.2010.031468}, author = {Dudley J. Pennell and John B. Porter and Maria Domenica Cappellini and Lee Lee Chan and Amal El-Beshlawy and Yesim Aydinok and Hishamshah Ibrahim and Chi-Kong Li and Vip Viprakasit and Mohsen Saleh Elalfy and Antonis Kattamis and Gillian Smith and Dany Habr and Gabor Domokos and Bernard Roubert and Ali Taher} }