Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study.
作者信息
Taher Ali T, Karakas Zeynep, Cassinerio Elena, Siritanaratkul Noppadol, Kattamis Antonis, Maggio Aurelio, Rivella Stefano, Hollaender Norbert, Mahuzier Bruyère, Gadbaw Brian, Aydinok Yesim
机构信息
Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon.
Department of Pediatric Hematology and Oncology, Istanbul University, Istanbul Medical Faculty, Istanbul, Turkey.
出版信息
Blood. 2018 Jan 11;131(2):263-265. doi: 10.1182/blood-2017-06-790121. Epub 2017 Nov 2.
Abstract
摘要
相似文献
1
Efficacy and safety of ruxolitinib in regularly transfused patients with thalassemia: results from a phase 2a study.芦可替尼在定期输血的地中海贫血患者中的疗效和安全性：一项2a期研究的结果。
Blood. 2018 Jan 11;131(2):263-265. doi: 10.1182/blood-2017-06-790121. Epub 2017 Nov 2.
2
Efficacy and safety of ruxolitinib in ineffective erythropoiesis suppression as a pretransplantation treatment for pediatric patients with beta-thalassemia major.罗昔替尼在β-地中海贫血患儿移植前治疗中作为无效红细胞生成抑制的疗效和安全性。
Pediatr Blood Cancer. 2021 Nov;68(11):e29338. doi: 10.1002/pbc.29338. Epub 2021 Sep 14.
3
Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia.红系细胞分化减少会加剧β地中海贫血中无效的红细胞生成。
Blood. 2008 Aug 1;112(3):875-85. doi: 10.1182/blood-2007-12-126938. Epub 2008 May 14.
4
Ineffective erythropoiesis in beta-thalassemia major is due to apoptosis at the polychromatophilic normoblast stage.重型β地中海贫血中无效红细胞生成是由于多染性正成红细胞阶段的细胞凋亡所致。
Exp Hematol. 2000 Dec;28(12):1343-53. doi: 10.1016/s0301-472x(00)00555-5.
5
Effect of Tumor Necrosis Factor-Alpha on Erythropoietin and Erythropoietin Receptor-Induced Erythroid Progenitor Cell Proliferation in β-Thalassemia/Hemoglobin E Patients.肿瘤坏死因子-α对β地中海贫血/血红蛋白E患者中促红细胞生成素及促红细胞生成素受体诱导的红系祖细胞增殖的影响
Turk J Haematol. 2015 Dec;32(4):304-10. doi: 10.4274/tjh.2014.0079. Epub 2015 Aug 6.
6
Successful correction of the human Cooley's anemia beta-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator.使用侧翼带有鸡高敏位点4染色质绝缘子的慢病毒载体成功矫正人类重型库利贫血（β-地中海贫血）主要表型。
Ann N Y Acad Sci. 2005;1054:238-49. doi: 10.1196/annals.1345.030.
7
8
Enhanced activation of autophagy in β-thalassemia/Hb E erythroblasts during erythropoiesis.β-地中海贫血/血红蛋白 E 红细胞在红细胞生成过程中自噬的增强激活。
Ann Hematol. 2011 Jul;90(7):747-58. doi: 10.1007/s00277-010-1152-5. Epub 2011 Jan 8.
9
Serum transferrin receptor as a marker of erythropoiesis suppression in patients on chronic transfusion.血清转铁蛋白受体作为慢性输血患者红细胞生成抑制的标志物。
Am J Hematol. 1999 Feb;60(2):121-5. doi: 10.1002/(sici)1096-8652(199902)60:2<121::aid-ajh6>3.0.co;2-2.
10
Ineffective erythropoiesis in β -thalassemia.β地中海贫血中的无效红细胞生成
ScientificWorldJournal. 2013 Mar 28;2013:394295. doi: 10.1155/2013/394295. Print 2013.
引用本文的文献
1
Skin complications during iron chelation therapy for beta-thalassemia: overview and treatment approach.β-地中海贫血症铁螯合疗法期间的皮肤并发症：概述与治疗方法。
Int J Hematol. 2024 Sep;120(3):271-277. doi: 10.1007/s12185-024-03825-w. Epub 2024 Aug 1.
2
Extramedullary haematopoiesis in patients with thalassemia: a cross-sectional description of its prevalence, clinical features and survival.地中海贫血患者的髓外造血：关于其患病率、临床特征及生存率的横断面描述
Hematol Transfus Cell Ther. 2024 Nov;46 Suppl 5(Suppl 5):S143-S151. doi: 10.1016/j.htct.2023.07.005. Epub 2023 Aug 26.
3
Normal and dysregulated crosstalk between iron metabolism and erythropoiesis.正常和失调的铁代谢与红细胞生成之间的相互作用。
Elife. 2023 Aug 14;12:e90189. doi: 10.7554/eLife.90189.
4
Novel potential therapeutics to modify iron metabolism and red cell synthesis in diseases associated with defective erythropoiesis.新型潜在治疗药物可调节与红细胞生成缺陷相关疾病的铁代谢和红细胞生成。
Haematologica. 2023 Oct 1;108(10):2582-2593. doi: 10.3324/haematol.2023.283057.
5
Non-Transfusion-Dependent Thalassemia: A Panoramic Review.非输血依赖型地中海贫血症：全景综述。
Medicina (Kaunas). 2022 Oct 21;58(10):1496. doi: 10.3390/medicina58101496.
6
The Roles of Mitophagy and Autophagy in Ineffective Erythropoiesis in β-Thalassemia.自噬和线粒体自噬在β-地中海贫血无效造血中的作用。
Int J Mol Sci. 2022 Sep 16;23(18):10811. doi: 10.3390/ijms231810811.
7
The unexpected impact of cabozantinib on red blood cells consumption in patients with transfusion-dependent thalassemia.卡博替尼对依赖输血的地中海贫血患者红细胞消耗的意外影响。
Ann Hematol. 2022 Jul;101(7):1621-1623. doi: 10.1007/s00277-022-04810-1. Epub 2022 Mar 5.
8
β-Thalassemia: evolving treatment options beyond transfusion and iron chelation.β-地中海贫血：超越输血和铁螯合的治疗选择。
Hematology Am Soc Hematol Educ Program. 2021 Dec 10;2021(1):600-606. doi: 10.1182/hematology.2021000313.
9
Improving Ineffective Erythropoiesis in Thalassemia: A Hope on the Horizon.改善地中海贫血中无效红细胞生成：曙光在前。
Cureus. 2021 Oct 5;13(10):e18502. doi: 10.7759/cureus.18502. eCollection 2021 Oct.
10
Advancing the care of β-thalassaemia patients with novel therapies.推进新型疗法治疗β-地中海贫血患者。
Blood Transfus. 2022 Jan;20(1):78-88. doi: 10.2450/2021.0265-21. Epub 2021 Oct 21.
本文引用的文献
1
2017 Clinical trials update in new treatments of β-thalassemia.2017 年β-地中海贫血新治疗方法的临床试验更新。
Am J Hematol. 2016 Nov;91(11):1135-1145. doi: 10.1002/ajh.24530.
2
Long-term findings from COMFORT-II, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis.COMFORT-II的长期研究结果，这是一项关于芦可替尼与最佳可用疗法治疗骨髓纤维化的3期研究。
Leukemia. 2016 Aug;30(8):1701-7. doi: 10.1038/leu.2016.148. Epub 2016 May 23.
3
Ruxolitinib versus standard therapy for the treatment of polycythemia vera.芦可替尼与标准疗法治疗真性红细胞增多症的比较。
N Engl J Med. 2015 Apr 23;372(17):1670-1. doi: 10.1056/NEJMc1502524.
4
Emerging EPO and EPO receptor regulators and signal transducers.新型促红细胞生成素及其受体调节剂和信号转导分子
Blood. 2015 Jun 4;125(23):3536-41. doi: 10.1182/blood-2014-11-575357. Epub 2015 Apr 17.
5
The role of ineffective erythropoiesis in non-transfusion-dependent thalassemia.无效红细胞生成在非输血依赖型地中海贫血中的作用。
Blood Rev. 2012 Apr;26 Suppl 1(0 1):S12-5. doi: 10.1016/S0268-960X(12)70005-X.
6
beta-Thalassemia: HiJAKing Ineffective Erythropoiesis and Iron Overload.β地中海贫血：抑制无效红细胞生成与铁过载
Adv Hematol. 2010;2010:938640. doi: 10.1155/2010/938640. Epub 2010 May 19.
7
Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia.红系细胞分化减少会加剧β地中海贫血中无效的红细胞生成。
Blood. 2008 Aug 1;112(3):875-85. doi: 10.1182/blood-2007-12-126938. Epub 2008 May 14.
Zotero 插件