使用补体抑制剂考维辛治疗造血干细胞移植相关血栓性微血管病。
Use of the complement inhibitor Coversin to treat HSCT-associated TMA.
作者信息
Goodship Timothy H J, Pinto Fernando, Weston-Davies Wynn H, Silva Juliana, Nishimura Jun-Ichi, Nunn Miles A, Mackie Ian, Machin Samuel J, Palm Liina, Pryce Jeremy W, Chiesa Robert, Amrolia Persis, Veys Paul
机构信息
Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, United Kingdom.
Royal Hospital for Sick Children, Glasgow, United Kingdom.
出版信息
Blood Adv. 2017 Jul 3;1(16):1254-1258. doi: 10.1182/bloodadvances.2016002832. eCollection 2017 Jul 11.
Abstract
Finding an inherited complement abnormality in HSCT-associated TMA provides a rationale for the use of a complement inhibitor.Alternative complement inhibitors such as Coversin should be considered in patients who are resistant to eculizumab.
摘要
在异基因造血干细胞移植相关血栓性微血管病中发现遗传性补体异常为使用补体抑制剂提供了理论依据。对于对依库珠单抗耐药的患者，应考虑使用如考维西单抗等替代性补体抑制剂。
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本文引用的文献
1
The genetic fingerprint of susceptibility for transplant-associated thrombotic microangiopathy.移植相关血栓性微血管病易感性的基因指纹图谱。
Blood. 2016 Feb 25;127(8):989-96. doi: 10.1182/blood-2015-08-663435. Epub 2015 Nov 24.
2
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Biol Blood Marrow Transplant. 2016 Feb;22(2):307-315. doi: 10.1016/j.bbmt.2015.10.002. Epub 2015 Oct 9.
3
Diagnostic and risk criteria for HSCT-associated thrombotic microangiopathy: a study in children and young adults.异基因造血干细胞移植相关血栓性微血管病的诊断和风险标准：一项针对儿童和青年的研究。
Blood. 2014 Jul 24;124(4):645-53. doi: 10.1182/blood-2014-03-564997. Epub 2014 May 29.
4
Genetic variants in C5 and poor response to eculizumab.C5 中的遗传变异与依库珠单抗反应不佳有关。
N Engl J Med. 2014 Feb 13;370(7):632-9. doi: 10.1056/NEJMoa1311084.
5
Eculizumab therapy in children with severe hematopoietic stem cell transplantation-associated thrombotic microangiopathy.依库珠单抗治疗儿童严重造血干细胞移植相关性血栓性微血管病。
Biol Blood Marrow Transplant. 2014 Apr;20(4):518-25. doi: 10.1016/j.bbmt.2013.12.565. Epub 2013 Dec 25.
6
Abnormalities in the alternative pathway of complement in children with hematopoietic stem cell transplant-associated thrombotic microangiopathy.造血干细胞移植相关性血栓性微血管病患儿补体旁路途径的异常。
Blood. 2013 Sep 19;122(12):2003-7. doi: 10.1182/blood-2013-05-501445. Epub 2013 Jun 27.
7
Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome.依库珠单抗治疗非典型溶血尿毒综合征。
N Engl J Med. 2013 Jun 6;368(23):2169-81. doi: 10.1056/NEJMoa1208981.
8
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9
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10
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J Immunol. 2009 Jun 1;182(11):7009-18. doi: 10.4049/jimmunol.0804031.
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