Luspatercept versus mitapivat for non-transfusion-dependent β-thalassemia: Dare to compare?
作者信息
Musallam Khaled M, Sheth Sujit, Cappellini Maria Domenica, Kuo Kevin H M, Kattamis Antonis, Aydinok Yesim, Viprakasit Vip, Taher Ali T
机构信息
Center for Research on Rare Blood Disorders (CR-RBD) and Thalassemia & Sickle Cell Center Burjeel Medical City Abu Dhabi United Arab Emirates.
Department of Public Health & Epidemiology Khalifa University Abu Dhabi United Arab Emirates.
出版信息
Hemasphere. 2025 Jul 13;9(7):e70165. doi: 10.1002/hem3.70165. eCollection 2025 Jul.
Abstract
摘要
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本文引用的文献
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Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial.
Lancet. 2025 Jul 5;406(10498):33-42. doi: 10.1016/S0140-6736(25)00635-X. Epub 2025 Jun 19.
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Mitapivat metabolically reprograms human β-thalassemic erythroblasts, increasing their responsiveness to oxidation.
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Identifying thresholds for meaningful improvements in NTDT-PRO scores to support conclusions about treatment benefit in clinical studies of patients with non-transfusion-dependent beta-thalassaemia: analysis of pooled data from a phase 2, double-blind, placebo-controlled, randomised trial.
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Novel therapeutic approaches in thalassemias, sickle cell disease, and other red cell disorders.
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Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia.
Am J Hematol. 2024 Jul;99(7):1300-1312. doi: 10.1002/ajh.27332. Epub 2024 Apr 25.
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Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice.
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Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial.
Lancet Haematol. 2022 Oct;9(10):e733-e744. doi: 10.1016/S2352-3026(22)00208-3. Epub 2022 Aug 22.
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