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1
Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy.镰状细胞病中丙酮酸激酶活性和稳定性降低：mitapivat治疗的新靶点。
Blood. 2021 May 27;137(21):2997-3001. doi: 10.1182/blood.2020008635.
2
Mitapivat versus Placebo for Pyruvate Kinase Deficiency.米替培南侧重于安慰剂用于治疗丙酮酸激酶缺乏症。
N Engl J Med. 2022 Apr 14;386(15):1432-1442. doi: 10.1056/NEJMoa2116634.
3
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.米他培坦治疗丙酮酸激酶缺乏症的安全性和疗效。
N Engl J Med. 2019 Sep 5;381(10):933-944. doi: 10.1056/NEJMoa1902678.
4
Mitapivat for sickle cell disease and thalassemia.米替膦酸治疗镰状细胞病和地中海贫血。
Drugs Today (Barc). 2023 Mar;59(3):125-134. doi: 10.1358/dot.2023.59.3.3521880.
5
Hereditary spherocytosis is associated with decreased pyruvate kinase activity due to impaired structural integrity of the red blood cell membrane.遗传性球形红细胞增多症与丙酮酸激酶活性降低有关，这是由于红细胞膜的结构完整性受损所致。
Br J Haematol. 2019 Nov;187(3):386-395. doi: 10.1111/bjh.16084. Epub 2019 Jul 5.
6
Functional and multi-omics signatures of mitapivat efficacy upon activation of pyruvate kinase in red blood cells from patients with sickle cell disease.米他匹韦激活镰状细胞病患者红细胞中丙酮酸激酶后的功能及多组学特征与疗效
Haematologica. 2024 Aug 1;109(8):2639-2652. doi: 10.3324/haematol.2023.284831.
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Mitapivat increases ATP and decreases oxidative stress and erythrocyte mitochondria retention in a SCD mouse model.米他培南增加 ATP 并减少 SCD 小鼠模型中的氧化应激和红细胞线粒体滞留。
Blood Cells Mol Dis. 2022 Jul;95:102660. doi: 10.1016/j.bcmd.2022.102660. Epub 2022 Mar 12.
8
The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model.丙酮酸激酶激活剂米替膦酸酯可减少β-地中海贫血小鼠模型的溶血并改善贫血。
J Clin Invest. 2021 May 17;131(10). doi: 10.1172/JCI144206.
9
Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in sickle cell disease: A phase 2, open-label study.口服丙酮酸激酶激活剂米塔匹瓦特治疗镰状细胞病的安全性和有效性：一项2期开放标签研究。
Am J Hematol. 2022 Jul;97(7):E226-E229. doi: 10.1002/ajh.26554. Epub 2022 Apr 16.
10
A phase 1 dose escalation study of the pyruvate kinase activator mitapivat (AG-348) in sickle cell disease.一项关于丙酮酸激酶激活剂米替泊肟（AG-348）在镰状细胞病中的 1 期剂量递增研究。
Blood. 2022 Nov 10;140(19):2053-2062. doi: 10.1182/blood.2022015403.

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1
Use of the microfluidic impedance red cell assay in sickle cell disease.微流控阻抗红细胞检测法在镰状细胞病中的应用。
Blood Adv. 2025 Aug 12;9(15):4004-4015. doi: 10.1182/bloodadvances.2024015590.
2
Pyruvate Kinase Function Correlates With Red Blood Cell Properties and Clinical Manifestations in Sickle Cell Disease.丙酮酸激酶功能与镰状细胞病中的红细胞特性及临床表现相关。
Am J Hematol. 2025 May;100(5):785-796. doi: 10.1002/ajh.27644. Epub 2025 Feb 21.
3
Metabolic blood profile and response to treatment with the pyruvate kinase activator mitapivat in patients with sickle cell disease.镰状细胞病患者的代谢血液指标及对丙酮酸激酶激活剂米他匹伐治疗的反应
Hemasphere. 2024 Jun 25;8(6):e109. doi: 10.1002/hem3.109. eCollection 2024 Jun.
4
Cardiac diastolic maladaptation is associated with the severity of exercise intolerance in sickle cell anemia patients.心脏舒张适应不良与镰状细胞贫血患者运动不耐受的严重程度相关。
Sci Rep. 2024 May 15;14(1):11095. doi: 10.1038/s41598-024-61689-w.
5
Recent developments in the use of pyruvate kinase activators as a new approach for treating sickle cell disease.丙酮酸激酶激活剂在治疗镰状细胞病中的新应用研究进展。
Blood. 2024 Mar 7;143(10):866-871. doi: 10.1182/blood.2023021167.
6
Pyruvate kinase activators: targeting red cell metabolism in sickle cell disease.丙酮酸激酶激活剂：靶向镰状细胞病的红细胞代谢。
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):107-113. doi: 10.1182/hematology.2023000467.
7
Oxygen gradient ektacytometry-derived biomarkers are associated with acute complications in sickle cell disease.氧梯度 ektacytometry 衍生的生物标志物与镰状细胞病的急性并发症相关。
Blood Adv. 2024 Jan 23;8(2):276-286. doi: 10.1182/bloodadvances.2023011013.
8
One-year safety and efficacy of mitapivat in sickle cell disease: follow-up results of a phase 2, open-label study.一项开放标签、2 期研究的随访结果：米替泊肟治疗镰状细胞病的 1 年安全性和疗效。
Blood Adv. 2023 Dec 26;7(24):7539-7550. doi: 10.1182/bloodadvances.2023011477.
9
Hope on the Horizon: New and Future Therapies for Sickle Cell Disease.地平线上的希望：镰状细胞病的新型及未来疗法
J Clin Med. 2023 Sep 1;12(17):5692. doi: 10.3390/jcm12175692.
10
Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease.治疗地中海贫血症和镰状细胞病患儿及青少年的新视角。
Eur J Pediatr. 2023 Jun;182(6):2509-2519. doi: 10.1007/s00431-023-04900-w. Epub 2023 Mar 31.

本文引用的文献

1
Oxygen gradient ektacytometry-derived biomarkers are associated with vaso-occlusive crises and correlate with treatment response in sickle cell disease.氧梯度流式细胞术衍生的生物标志物与血管闭塞性危象相关，并与镰状细胞病的治疗反应相关。
Am J Hematol. 2021 Jan;96(1):E29-E32. doi: 10.1002/ajh.26031. Epub 2020 Nov 11.
2
Recent Advances in the Treatment of Sickle Cell Disease.镰状细胞病治疗的最新进展
Front Physiol. 2020 May 20;11:435. doi: 10.3389/fphys.2020.00435. eCollection 2020.
3
The pyruvate kinase (PK) to hexokinase enzyme activity ratio and erythrocyte PK protein level in the diagnosis and phenotype of PK deficiency.丙酮酸激酶（PK）与己糖激酶酶活性比值和红细胞 PK 蛋白水平在 PK 缺乏症的诊断和表型中的应用。
Br J Haematol. 2021 Mar;192(6):1092-1096. doi: 10.1111/bjh.16724. Epub 2020 May 28.
4
Emerging therapies in sickle cell disease.镰状细胞病的新兴疗法。
Br J Haematol. 2020 Jul;190(2):149-172. doi: 10.1111/bjh.16504. Epub 2020 Mar 6.
5
AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes.AG-348（Mitapivat）是一种红血球丙酮酸激酶的别构激活剂，能够在广泛的 PKLR 基因型范围内提高酶活性、蛋白稳定性和 ATP 水平。
Haematologica. 2021 Jan 1;106(1):238-249. doi: 10.3324/haematol.2019.238865.
6
Methodological aspects of the oxygenscan in sickle cell disease: A need for standardization.镰状细胞病氧扫描的方法学方面：标准化的必要性。
Am J Hematol. 2020 Jan;95(1):E5-E8. doi: 10.1002/ajh.25655. Epub 2019 Oct 26.
7
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency.米他培坦治疗丙酮酸激酶缺乏症的安全性和疗效。
N Engl J Med. 2019 Sep 5;381(10):933-944. doi: 10.1056/NEJMoa1902678.
8
Rapid and reproducible characterization of sickling during automated deoxygenation in sickle cell disease patients.自动化去氧条件下镰状细胞病患者镰变的快速可重现性特征。
Am J Hematol. 2019 May;94(5):575-584. doi: 10.1002/ajh.25443. Epub 2019 Mar 8.
9
Addressing the diagnostic gaps in pyruvate kinase deficiency: Consensus recommendations on the diagnosis of pyruvate kinase deficiency.解决丙酮酸激酶缺乏症的诊断差距：丙酮酸激酶缺乏症诊断的共识建议。
Am J Hematol. 2019 Jan;94(1):149-161. doi: 10.1002/ajh.25325. Epub 2018 Nov 28.
10
Phase 1 Single- and Multiple-Ascending-Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers.AG-348，一种新型别构激活剂的安全、药代动力学和药效学的 1 期单剂量和多剂量递增随机研究，在健康志愿者中。
Clin Pharmacol Drug Dev. 2019 Feb;8(2):246-259. doi: 10.1002/cpdd.604. Epub 2018 Aug 9.

Decreased activity and stability of pyruvate kinase in sickle cell disease: a novel target for mitapivat therapy.

作者信息

Rab Minke A E, Bos Jennifer, van Oirschot Brigitte A, van Straaten Stephanie, Kosinski Penelope A, Chubukov Victor, Kim Hyeryun, Mangus Heidi, Schutgens Roger E G, Pasterkamp Gerard, Dang Lenny, Kung Charles, van Beers Eduard J, van Wijk Richard

机构信息

Central Diagnostic Laboratory-Research and.

Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.

出版信息

Blood. 2021 May 27;137(21):2997-3001. doi: 10.1182/blood.2020008635.

DOI:10.1182/blood.2020008635
PMID:33690814
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8160499/
Abstract
摘要