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早期使用卡帕西珠单抗和奥滨尤妥珠单抗可在不进行治疗性血浆置换的情况下成功治疗复发性血栓性血小板减少性紫癜:一例报告

Early caplacizumab and obinutuzumab enable successful treatment of relapsing thrombotic thrombocytopenic purpura without therapeutic plasma exchange: a case report.

作者信息

Schimpf Judith, Haller Patrizia, Zitt Emanuel

机构信息

Department of Internal Medicine 3 (Nephrology, Dialysis and Hypertension), Landeskrankenhaus (LKH) Feldkirch, Feldkirch, Austria.

Vorarlberg Institute for Vascular Investigation and Treatment (VIVIT), Feldkirch, Austria.

出版信息

Front Immunol. 2025 Apr 28;16:1588471. doi: 10.3389/fimmu.2025.1588471. eCollection 2025.

DOI:10.3389/fimmu.2025.1588471
PMID:40356910
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12066623/
Abstract

Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening disorder due to a severe acquired or inherited ADAMTS13 deficiency. So far, therapeutic algorithms almost universally include the prompt initiation of therapeutic plasma exchange (TPE). We firstly report a 55-year-old female with a history of relapsing TTP who was managed exclusively with caplacizumab, steroids and the second generation fully humanized anti-CD20 monoclonal antibody obinutuzumab during a relapse without the need of TPE throughout the whole disease course. This case illustrates the safety and effectiveness of a TPE-free TTP management using prompt initiation of caplacizumab and obinutuzumab.

摘要

血栓性血小板减少性紫癜(TTP)是一种罕见且危及生命的疾病,由严重的获得性或遗传性ADAMTS13缺乏引起。到目前为止,治疗方案几乎普遍包括立即开始治疗性血浆置换(TPE)。我们首次报告了一名55岁复发性TTP女性患者,在复发期间仅使用卡泊单抗、类固醇和第二代全人源抗CD20单克隆抗体奥妥珠单抗进行治疗,整个病程无需TPE。该病例说明了立即使用卡泊单抗和奥妥珠单抗进行无TPE的TTP管理的安全性和有效性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/37af/12066623/2770dcf182ca/fimmu-16-1588471-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/37af/12066623/2770dcf182ca/fimmu-16-1588471-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/37af/12066623/2770dcf182ca/fimmu-16-1588471-g001.jpg

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本文引用的文献

1
Efficacy and Safety of Obinutuzumab in Immune-Mediated Thrombotic Thrombocytopenic Purpura.奥妥珠单抗治疗免疫性血栓性血小板减少性紫癜的疗效与安全性
Am J Hematol. 2025 Feb;100(2):350-353. doi: 10.1002/ajh.27550. Epub 2024 Dec 12.
2
Management of immune thrombotic thrombocytopenic purpura without therapeutic plasma exchange.免疫性血栓性血小板减少性紫癜的管理,不包括治疗性血浆置换。
Blood. 2024 Oct 3;144(14):1486-1495. doi: 10.1182/blood.2023023780.
3
European Renal Best Practice endorsement of guidelines for diagnosis and therapy of thrombotic thrombocytopaenic purpura published by the International Society on Thrombosis and Haemostasis.
欧洲肾脏最佳实践组织认可国际血栓与止血学会发布的血栓性血小板减少性紫癜诊断和治疗指南。
Nephrol Dial Transplant. 2022 Jun 23;37(7):1229-1234. doi: 10.1093/ndt/gfac034.
4
Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura.卡普拉珠单抗治疗获得性血栓性血小板减少性紫癜。
N Engl J Med. 2019 Jan 24;380(4):335-346. doi: 10.1056/NEJMoa1806311. Epub 2019 Jan 9.
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Obinutuzumab: what is there to learn from clinical trials?奥滨尤妥珠单抗:临床试验中有哪些值得学习的?
Blood. 2017 Aug 3;130(5):581-589. doi: 10.1182/blood-2017-03-771832. Epub 2017 Jun 5.
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Thrombotic thrombocytopenic purpura.血栓性血小板减少性紫癜。
Blood. 2017 May 25;129(21):2836-2846. doi: 10.1182/blood-2016-10-709857. Epub 2017 Apr 17.
7
Preclinical activity of the type II CD20 antibody GA101 (obinutuzumab) compared with rituximab and ofatumumab in vitro and in xenograft models.GA101(奥滨尤妥珠单抗,一种 II 型 CD20 抗体)与利妥昔单抗和奥法木单抗的体外及异种移植模型中的临床前活性比较。
Mol Cancer Ther. 2013 Oct;12(10):2031-42. doi: 10.1158/1535-7163.MCT-12-1182. Epub 2013 Jul 19.
8
Increasing the efficacy of CD20 antibody therapy through the engineering of a new type II anti-CD20 antibody with enhanced direct and immune effector cell-mediated B-cell cytotoxicity.通过工程改造新型 II 型抗 CD20 抗体,增强直接和免疫效应细胞介导的 B 细胞细胞毒性,提高 CD20 抗体治疗的疗效。
Blood. 2010 Jun 3;115(22):4393-402. doi: 10.1182/blood-2009-06-225979. Epub 2010 Mar 1.
9
ADAMTS-13 assays in thrombotic thrombocytopenic purpura.ADAMTS-13 检测在血栓性血小板减少性紫癜中的应用。
J Thromb Haemost. 2010 Apr;8(4):631-40. doi: 10.1111/j.1538-7836.2010.03761.x. Epub 2010 Jan 21.