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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.

DOI:10.1182/bloodadvances.2016002832
PMID:29296765
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5728542/
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

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Blood. 2016 Feb 25;127(8):989-96. doi: 10.1182/blood-2015-08-663435. Epub 2015 Nov 24.
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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。
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Biol Blood Marrow Transplant. 2014 Apr;20(4):518-25. doi: 10.1016/j.bbmt.2013.12.565. Epub 2013 Dec 25.
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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.
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N Engl J Med. 2013 Jun 6;368(23):2169-81. doi: 10.1056/NEJMoa1208981.
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The binding of factor H to a complex of physiological polyanions and C3b on cells is impaired in atypical hemolytic uremic syndrome.在非典型溶血性尿毒症综合征中，补体因子H与细胞上的生理性多阴离子和C3b复合物的结合受损。
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