Publication: Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major
Issued Date
2012-06-01
Resource Type
ISSN
15928721
03906078
03906078
Other identifier(s)
2-s2.0-84862141974
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Mahidol University
Rights Holder(s)
SCOPUS
Bibliographic Citation
Haematologica. Vol.97, No.6 (2012), 842-848
Suggested Citation
Dudley J. Pennell, John B. Porter, Maria Domenica Cappellini, Lee Lee Chan, Amal El-Beshlawy, Yesim Aydinok, Hishamshah Ibrahim, Chi Kong Li, Vip Viprakasit, Mohsen S. Elalfy, Antonis Kattamis, Gillian Smith, Dany Habr, Gabor Domokos, Bernard Roubert, Ali Taher Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major. Haematologica. Vol.97, No.6 (2012), 842-848. doi:10.3324/haematol.2011.049957 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/14800
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Title
Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major
Other Contributor(s)
Royal Brompton Hospital
UCL
Universita degli Studi di Milano
University of Malaya Medical Centre
Cairo University
Ege Universitesi
Kuala Lumpur Hospital
Prince of Wales Hospital Hong Kong
Mahidol University
Ain Shams University
University of Athens
Novartis Pharmaceuticals
Novartis International AG
American University of Beirut
UCL
Universita degli Studi di Milano
University of Malaya Medical Centre
Cairo University
Ege Universitesi
Kuala Lumpur Hospital
Prince of Wales Hospital Hong Kong
Mahidol University
Ain Shams University
University of Athens
Novartis Pharmaceuticals
Novartis International AG
American University of Beirut
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±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.