A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in β-thalassemia major (CORDELIA)
Dudley J Pennell, John B Porter, Antonio Piga, Yongrong Lai, Amal El-Beshlawy, Khawla M Belhoul, Mohsen Elalfy, Akif Yesilipek, Yurdanur Kilinç, Tomasz Lawniczek, Dany Habr, Marianne Weisskopf, Yiyun Zhang, Yesim Aydinok, CORDELIA study investigators, Yongrong Lai, Michalis Hatjigavriel, Isaac Odame, Mohsen Elalfy, Hadi Goubran, Amal El-Beshlawy, Gianluca Forni, Ali Taher, Khawla Belhoul, John Porter, Sally Kinsey, Farrukh Shah, Quentin Hill, Kai-Hsin Lin, Pranee Sutcharitchan, Vip Viprakasit, Noppadol Siritanaratkul, Yesim Aydinok, Leyla Agaoglu, Yurdanur Kilinc, Akif Yesilipek, Dudley J Pennell, John B Porter, Antonio Piga, Yongrong Lai, Amal El-Beshlawy, Khawla M Belhoul, Mohsen Elalfy, Akif Yesilipek, Yurdanur Kilinç, Tomasz Lawniczek, Dany Habr, Marianne Weisskopf, Yiyun Zhang, Yesim Aydinok, CORDELIA study investigators, Yongrong Lai, Michalis Hatjigavriel, Isaac Odame, Mohsen Elalfy, Hadi Goubran, Amal El-Beshlawy, Gianluca Forni, Ali Taher, Khawla Belhoul, John Porter, Sally Kinsey, Farrukh Shah, Quentin Hill, Kai-Hsin Lin, Pranee Sutcharitchan, Vip Viprakasit, Noppadol Siritanaratkul, Yesim Aydinok, Leyla Agaoglu, Yurdanur Kilinc, Akif Yesilipek
Abstract
Randomized comparison data on the efficacy and safety of deferasirox for myocardial iron removal in transfusion dependent patients are lacking. CORDELIA was a prospective, randomized comparison of deferasirox (target dose 40 mg/kg per day) vs subcutaneous deferoxamine (50-60 mg/kg per day for 5-7 days/week) for myocardial iron removal in 197 β-thalassemia major patients with myocardial siderosis (T2* 6-20 milliseconds) and no signs of cardiac dysfunction (mean age, 19.8 years). Primary objective was to demonstrate noninferiority of deferasirox for myocardial iron removal, assessed by changes in myocardial T2* after 1 year using a per-protocol analysis. Geometric mean (Gmean) myocardial T2* improved with deferasirox from 11.2 milliseconds at baseline to 12.6 milliseconds at 1 year (Gmeans ratio, 1.12) and with deferoxamine (11.6 milliseconds to 12.3 milliseconds; Gmeans ratio, 1.07). The between-arm Gmeans ratio was 1.056 (95% confidence interval [CI], 0.998, 1.133). The lower 95% CI boundary was greater than the prespecified margin of 0.9, establishing noninferiority of deferasirox vs deferoxamine (P = .057 for superiority of deferasirox). Left ventricular ejection fraction remained stable in both arms. Frequency of drug-related adverse events was comparable between deferasirox (35.4%) and deferoxamine (30.8%). CORDELIA demonstrated the noninferiority of deferasirox compared with deferoxamine for myocardial iron removal. This trial is registered at www.clinicaltrials.gov as #NCT00600938.
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Source: PubMed