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获得性因子X缺乏症中因子X浓缩物的治疗方案及剂量

Factor X Concentrate Treatment Schedule and Dosing in Acquired FX Deficiency.

作者信息

Ross Andrew, Shaw Rebecca J, Garth Louise, Farrelly Cathy

机构信息

Liverpool University Hospital NHS Foundation Trust, Liverpool L7 8XP, UK.

Department of Clinical Infection, Microbiology and Immunology, Institute of Infection, Veterinary and Ecological Sciences, Leahurst Campus, University of Liverpool, Liverpool CH64 7TEK, UK.

出版信息

Hematol Rep. 2025 Feb 21;17(2):10. doi: 10.3390/hematolrep17020010.

DOI:10.3390/hematolrep17020010
PMID:40126219
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11932198/
Abstract

BACKGROUND

Acquired factor X (FX) deficiency is a rare condition that can cause life threatening bleeding. Here we outline a successful management strategy for gastrointestinal bleeding (GI) using human FX concentrate.

CASE DESCRIPTION

A 61-year-old male presented with upper GI bleeding and a prolonged prothrombin time. Investigations demonstrated an acquired FX deficiency (determined to be secondary to AL amyloidosis).

RESULTS

Treatment with FX concentrate to maintain trough FX levels >20% resulted in successful cessation of bleeding symptoms, and levels >50% facilitated urgent invasive procedures.

CONCLUSIONS

This case report adds valuable insight into the management of this rare condition, and how best to utilize FX concentrates in acquired FX deficiency.

摘要

背景

获得性因子X(FX)缺乏症是一种罕见的疾病,可导致危及生命的出血。在此,我们概述了一种使用人FX浓缩物治疗胃肠道出血(GI)的成功管理策略。

病例描述

一名61岁男性出现上消化道出血,凝血酶原时间延长。检查显示为获得性FX缺乏症(确定为继发于AL淀粉样变性)。

结果

用FX浓缩物治疗以维持谷值FX水平>20%,成功停止了出血症状,而水平>50%则有助于进行紧急侵入性操作。

结论

本病例报告为这种罕见疾病的管理以及如何在获得性FX缺乏症中最佳利用FX浓缩物提供了宝贵的见解。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3ded/11932198/d9a0751e1ae7/hematolrep-17-00010-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3ded/11932198/e91f79200d9a/hematolrep-17-00010-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3ded/11932198/d9a0751e1ae7/hematolrep-17-00010-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3ded/11932198/e91f79200d9a/hematolrep-17-00010-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3ded/11932198/d9a0751e1ae7/hematolrep-17-00010-g002.jpg

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Factor X Concentrate Treatment Schedule and Dosing in Acquired FX Deficiency.获得性因子X缺乏症中因子X浓缩物的治疗方案及剂量
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本文引用的文献

1
Thromboembolism and bleeding in systemic amyloidosis: a review.系统性淀粉样变性中的血栓栓塞和出血:综述。
ESC Heart Fail. 2022 Feb;9(1):11-20. doi: 10.1002/ehf2.13701. Epub 2021 Nov 16.
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Diagnosis, therapeutic advances, and key recommendations for the management of factor X deficiency.因子 X 缺乏症的诊断、治疗进展及治疗管理的关键建议。
Blood Rev. 2021 Nov;50:100833. doi: 10.1016/j.blre.2021.100833. Epub 2021 Apr 27.
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Acquired Factor X Deficiency in Patients With Primary Light Chain Amyloidosis.原发性轻链淀粉样变性患者获得性因子X缺乏症
J Investig Med High Impact Case Rep. 2019 Jan-Dec;7:2324709619832332. doi: 10.1177/2324709619832332.
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Acquired factor X deficiency in light-chain (AL) amyloidosis is rare and associated with advanced disease.轻链(AL)淀粉样变性中获得性因子X缺乏症罕见,且与晚期疾病相关。
Hematol Oncol Stem Cell Ther. 2019 Mar;12(1):10-14. doi: 10.1016/j.hemonc.2018.05.002. Epub 2018 Jun 28.
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Complex formation with pentraxin-2 regulates factor X plasma levels and macrophage interactions.与 pentraxin-2 形成复合物可调节因子 X 的血浆水平和巨噬细胞相互作用。
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Guideline for the diagnosis and management of the rare coagulation disorders: a United Kingdom Haemophilia Centre Doctors' Organization guideline on behalf of the British Committee for Standards in Haematology.罕见凝血障碍的诊断与管理指南:英国血友病中心医生组织代表英国血液学标准委员会发布的指南
Br J Haematol. 2014 Nov;167(3):304-26. doi: 10.1111/bjh.13058. Epub 2014 Aug 6.
7
Utility of factor X concentrate for the treatment of acquired factor X deficiency in systemic light-chain amyloidosis.凝血因子X浓缩物在治疗系统性轻链淀粉样变性所致获得性凝血因子X缺乏症中的应用。
Blood. 2014 May 1;123(18):2899-900. doi: 10.1182/blood-2014-02-556514.
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Factor X deficiency.凝血因子X缺乏症
Semin Thromb Hemost. 2009 Jun;35(4):407-15. doi: 10.1055/s-0029-1225763. Epub 2009 Jul 13.
9
Poor recovery and short survival of infused factor X in a case of acquired factor X deficiency and amyloidosis.一例获得性因子X缺乏症合并淀粉样变性患者中,输注的因子X恢复不佳且存活时间短。
Thromb Haemost. 1999 Oct;82(4):1375-6.