- Dudley J. Pennell1,*,
- John B. Porter2,
- Maria Domenica Cappellini3,
- Lee Lee Chan4,
- Amal El-Beshlawy5,
- Yesim Aydinok6,
- Hishamshah Ibrahim7,
- Chi-Kong Li8,
- Vip Viprakasit9,
- Mohsen Saleh Elalfy10,
- Antonis Kattamis11,
- Gillian Smith12,
- Dany Habr13,
- Gabor Domokos14,
- Bernard Roubert14 and
- Ali Taher15
- 1 Royal Brompton hospital, UK;
- 2 University College London, UK;
- 3 Universita' di Milano, Policlinico Foundation IRCCS;
- 4 University Malaya Medical Centre;
- 5 Cairo University, Egypt;
- 6 Ege University Medical Faculty, Turkey;
- 7 Hospital Kuala Lumpur;
- 8 Prince of Wales Hospital, Hong Kong;
- 9 Department of Pediatrics, Siriraj Hospital;
- 10 Ain Shams University;
- 11 First Dept of Pediatrics, University of Athens;
- 12 Royal Brompton Hospital;
- 13 Novartis Pharmaceuticals;
- 14 Novartis Pharma AG;
- 15 American University of Beirut
- ↵* Corresponding author; email:
Background. The efficacy of cardiac iron chelation in transfusion-dependent patients has been demonstrated in 1-year prospective trials. Since normalization of cardiac T2* takes several years, the efficacy and safety of deferasirox was assessed for 2 years in patients with β thalassemia major in the cardiac substudy of EPIC trial.
Design and Methods: Eligible patients with myocardial T2* >5 to <20 ms received deferasirox, with the primary endpoint being the change in T2* from baseline to 2 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/day in the 1-year core study increasing to 36.1+/- 7.7 mg/kg/day during the second-year of treatment. Geometric mean myocardial T2* increased from a baseline of 11.2 to 14.8 ms at 2 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 includeded (≥5%), increased serum creatinine, rash and increased alanine aminotransferase.
Conclusions: Continuous treatment with deferasirox for 2 years with a target dose of 40 mg/kg/day 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
- Received August 2, 2010.
- Accepted November 9, 2010.
- Copyright © 2010, Ferrata Storti Foundation