Efficacy of deferasirox in reducing and preventing cardiac iron overload in β-thalassemia
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- Dudley J. Pennell
- Royal Brompton Hospital, London, United Kingdom;
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- John B. Porter
- University College London, London, United Kingdom;
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- Maria Domenica Cappellini
- Universitá di Milano, Policlinico Foundation, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Milan, Italy;
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- Amal El-Beshlawy
- Cairo University, Cairo, Egypt;
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- Lee Lee Chan
- University Malaya Medical Centre, Lembah Pantai, Kuala Lumpur, Malaysia;
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- Yesim Aydinok
- Ege University Medical Faculty, Izmir, Turkey;
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- Mohsen Saleh Elalfy
- Ain Shams University, Cairo, Egypt;
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- Pranee Sutcharitchan
- Chulalongkorn University and King Chulalongkorn Memorial Hospital, Bangkok, Thailand;
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- Chi-Kong Li
- Prince of Wales Hospital, Chinese University of Hong Kong, Hong Kong;
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- Hishamshah Ibrahim
- Hospital Kuala Lumpur, Kuala Lumpur, Malaysia;
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- Vip Viprakasit
- Faculty of Medicine Siriraj Hospital, Department of Pediatrics, Division of Haematology-Oncology, Bangkoknoi, Bangkok, Thailand;
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- Antonis Kattamis
- First Department of Pediatrics, University of Athens, Athens, Greece;
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- Gillian Smith
- Royal Brompton Hospital, London, United Kingdom;
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- Dany Habr
- Novartis Pharmaceuticals, East Hanover, NJ;
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- Gabor Domokos
- Novartis Pharma, Basel, Switzerland; and
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- Bernard Roubert
- Novartis Pharma, Basel, Switzerland; and
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- Ali Taher
- American University of Beirut, Beirut, Lebanon
書誌事項
- 公開日
- 2010-03-25
- DOI
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- 10.1182/blood-2009-04-217455
- 公開者
- American Society of Hematology
この論文をさがす
説明
<jats:p>Cardiac iron overload causes most deaths in β-thalassemia major. The efficacy of deferasirox in reducing or preventing cardiac iron overload was assessed in 192 patients with β-thalassemia in a 1-year prospective, multicenter study. The cardiac iron reduction arm (n = 114) included patients with magnetic resonance myocardial T2* from 5 to 20 ms (indicating cardiac siderosis), left ventricular ejection fraction (LVEF) of 56% or more, serum ferritin more than 2500 ng/mL, liver iron concentration more than 10 mg Fe/g dry weight, and more than 50 transfused blood units. The prevention arm (n = 78) included otherwise eligible patients whose myocardial T2* was 20 ms or more. The primary end point was the change in myocardial T2* at 1 year. In the cardiac iron reduction arm, the mean deferasirox dose was 32.6 mg/kg per day. Myocardial T2* (geometric mean ± coefficient of variation) improved from a baseline of 11.2 ms (± 40.5%) to 12.9 ms (± 49.5%) (+16%; P < .001). LVEF (mean ± SD) was unchanged: 67.4 (± 5.7%) to 67.0 (± 6.0%) (−0.3%; P = .53). In the prevention arm, baseline myocardial T2* was unchanged from baseline of 32.0 ms (± 25.6%) to 32.5 ms (± 25.1%) (+2%; P = .57) and LVEF increased from baseline 67.7 (± 4.7%) to 69.6 (± 4.5%) (+1.8%; P < .001). This prospective study shows that deferasirox is effective in removing and preventing myocardial iron accumulation. This study is registered at http://clinicaltrials.gov as NCT00171821.</jats:p>
収録刊行物
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- Blood
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Blood 115 (12), 2364-2371, 2010-03-25
American Society of Hematology
