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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.

DOI:10.1182/blood-2017-06-790121
PMID:29097381
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5757682/
Abstract
摘要

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本文引用的文献

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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.