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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.

DOI:10.1002/hem3.70165
PMID:40657307
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12255901/
Abstract
摘要

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Luspatercept versus mitapivat for non-transfusion-dependent β-thalassemia: Dare to compare?卢司帕西普与米塔匹瓦特治疗非输血依赖型β地中海贫血:敢比较吗?
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2
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。
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3
Long-term safety and erythroid response with luspatercept treatment in patients with β-thalassemia.卢司帕安治疗β地中海贫血患者的长期安全性和红系反应
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本文引用的文献

1
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.
4
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.
5
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.
6
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.
7
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。
Haematologica. 2023 Sep 1;108(9):2535-2541. doi: 10.3324/haematol.2022.282614.
8
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Hemasphere. 2022 Nov 15;6(12):e806. doi: 10.1097/HS9.0000000000000806. eCollection 2022 Dec.
9
Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial.芦可替尼治疗非输血依赖型β-地中海贫血(BEYOND)的贫血:一项 2 期、随机、双盲、多中心、安慰剂对照试验。
Lancet Haematol. 2022 Oct;9(10):e733-e744. doi: 10.1016/S2352-3026(22)00208-3. Epub 2022 Aug 22.
10
Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study.口服丙酮酸激酶激活剂米他匹法特在非输血依赖型α地中海贫血或β地中海贫血成人患者中的安全性和有效性:一项开放标签、多中心、2期研究。
Lancet. 2022 Aug 13;400(10351):493-501. doi: 10.1016/S0140-6736(22)01337-X.