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.米塔匹瓦特用于非输血依赖型α地中海贫血或β地中海贫血成人患者（ENERGIZE）：一项3期国际随机双盲安慰剂对照试验。
Lancet. 2025 Jul 5;406(10498):33-42. doi: 10.1016/S0140-6736(25)00635-X. Epub 2025 Jun 19.
2
Mitapivat metabolically reprograms human β-thalassemic erythroblasts, increasing their responsiveness to oxidation.米塔匹瓦特可对人类β地中海贫血成红细胞进行代谢重编程，增强其对氧化的反应能力。
Blood Adv. 2025 Jun 10;9(11):2818-2830. doi: 10.1182/bloodadvances.2024013591.
3
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.确定 NTDT-PRO 评分有意义改善的阈值，以支持非输血依赖型β地中海贫血患者临床研究中关于治疗获益的结论：来自 2 期、双盲、安慰剂对照、随机试验的汇总数据的分析。
BMJ Open. 2024 Nov 14;14(11):e085234. doi: 10.1136/bmjopen-2024-085234.
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Genetic iron overload aggravates, and pharmacological iron restriction improves, MDS pathophysiology in a preclinical study.在一项临床前研究中，遗传性铁过载会加重骨髓增生异常综合征（MDS）的病理生理学，而药物性铁限制则可改善该病理生理学。
Blood. 2025 Jan 9;145(2):155-169. doi: 10.1182/blood.2024026135.
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Novel therapeutic approaches in thalassemias, sickle cell disease, and other red cell disorders.新型治疗方法在地中海贫血症、镰状细胞病和其他红细胞疾病中的应用。
Blood. 2024 Aug 22;144(8):853-866. doi: 10.1182/blood.2023022193.
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Combination of a TGF-β ligand trap (RAP-GRL) and TMPRSS6-ASO is superior for correcting β-thalassemia.TGF-β 配体陷阱（RAP-GRL）与 TMPRSS6-ASO 的联合应用优于 β-地中海贫血的矫正。
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.米他匹伐，一种丙酮酸激酶激活剂，可减轻β地中海贫血小鼠的输血负担并减少铁过载。
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