@article {117521, title = {Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major.}, journal = {Haematologica}, volume = {97}, year = {2012}, month = {2012 Jun}, pages = {842-8}, abstract = {

BACKGROUND: 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 {\textpm} 9.8 mg/kg per day. Myocardial T2*, assessed by cardiovascular magnetic resonance, significantly increased from 12.0 ms {\textpm} 39.1\% at baseline to 17.1 ms {\textpm} 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 {\textpm} 1.2 mg Fe/g dry weight (dw) at baseline to 1.80 {\textpm} 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 drug-related 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.

}, keywords = {Adolescent, Adult, Benzoates, beta-Thalassemia, Blood Transfusion, Cardiomyopathies, Chelation Therapy, Child, Drug Administration Schedule, Heart, Humans, Iron, Iron Chelating Agents, Iron Overload, Longitudinal Studies, Magnetic Resonance Angiography, Triazoles}, issn = {1592-8721}, doi = {10.3324/haematol.2011.049957}, 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 S. Elalfy and Antonis Kattamis and Gillian Smith and Dany Habr and Gabor Domokos and Bernard Roubert and Ali Taher} }