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骨髓增殖性肿瘤中的血小板增多症和出血:探索临床多样性及获得性血管性血友病综合征的风险——来自英国一个中心的见解

Thrombocytosis and bleeding in myeloproliferative neoplasms: exploring clinical diversity and risk of acquired von Willebrand syndrome-insights from a UK center.

作者信息

Simini Giulia, Innes Andrew, Vinayagam Saravanan, Mobayen Golzar, Karawitage Nilanthi, Claudiani Simone, Odho Zain, Laffan Mike, Arachchillage Deepa J

机构信息

Department of Haematology, Imperial College Healthcare National Health Service Trust, London, UK.

Department of Haematology, Cancer Institute, University College London, London, UK.

出版信息

Res Pract Thromb Haemost. 2025 Jun 24;9(5):102954. doi: 10.1016/j.rpth.2025.102954. eCollection 2025 Jul.

DOI:10.1016/j.rpth.2025.102954
PMID:40709222
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12284493/
Abstract

BACKGROUND

Myeloproliferative neoplasms (MPNs) represent a group of blood disorders characterized by myeloid cell proliferation and an associated increased risk of thrombosis and bleeding. Platelet count may have a direct link to these complications.

OBJECTIVES

To share our MPN clinic's experience with hemostatic testing and bleeding outcomes in patients with platelets ≥ 800 × 10/L.

METHODS

This was a single-center retrospective study from May 2022 to September 2024. Clinical characteristics, treatments, and bleeding events of patients with MPN or chronic myeloid leukemia were recorded. Laboratory assessments included full blood count, renal function, coagulation profiles, platelet function test, and von Willebrand factor (VWF) assays.

RESULTS

A total of 39 patients were included, majority of whom received aspirin for thrombosis prevention (76%). The study found that bleeding complications occurred in 33% of patients, with mucocutaneous bleeding being the most common. There was a trend toward bleeding in patients on aspirin ( = .07). However, platelet count alone did not predict bleeding risk. While some patients showed abnormal VWF function, low VWF levels were not consistently associated with increased bleeding. Interestingly, we found moderate negative correlation between baseline VWF ristocetin/antigen and activated partial thromboplastin time ( = .02; = -.37) and prothrombin time ( = .009, = -.45), suggesting other potential coagulation imbalances associated with bleeding diathesis in MPNs. Post cytoreduction, there was a significant increase in mean VWF ristocetin/antigen ratio ( = .0009).

CONCLUSION

The study illustrates the limitations of relying solely on platelet counts to estimate bleeding risk in MPN patients. Assessment of VWF activity and careful selection of antithrombotic therapy were highlighted as important considerations.

摘要

背景

骨髓增殖性肿瘤(MPN)是一组以髓系细胞增殖以及血栓形成和出血风险增加为特征的血液疾病。血小板计数可能与这些并发症直接相关。

目的

分享我们MPN诊所对血小板计数≥800×10⁹/L患者进行止血检测和出血结局的经验。

方法

这是一项2022年5月至2024年9月的单中心回顾性研究。记录了MPN或慢性髓系白血病患者的临床特征、治疗方法和出血事件。实验室评估包括全血细胞计数、肾功能、凝血指标、血小板功能测试和血管性血友病因子(VWF)检测。

结果

共纳入39例患者,其中大多数(76%)接受阿司匹林预防血栓形成。研究发现33%的患者发生出血并发症,最常见的是皮肤黏膜出血。服用阿司匹林的患者有出血趋势(P = 0.07)。然而,仅血小板计数并不能预测出血风险。虽然一些患者显示VWF功能异常,但低VWF水平与出血增加并无一致关联。有趣的是,我们发现基线VWF瑞斯托霉素辅因子/抗原与活化部分凝血活酶时间(P = 0.02;r = -0.37)和凝血酶原时间(P = 0.009,r = -0.45)之间存在中度负相关,提示MPN中与出血倾向相关的其他潜在凝血失衡。细胞减灭术后,平均VWF瑞斯托霉素辅因子/抗原比值显著增加(P = 0.0009)。

结论

该研究说明了仅依靠血小板计数来评估MPN患者出血风险的局限性。强调评估VWF活性和谨慎选择抗血栓治疗是重要的考虑因素。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8ad7/12284493/f346c2e6f07d/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8ad7/12284493/5feaae827896/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8ad7/12284493/f346c2e6f07d/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8ad7/12284493/5feaae827896/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8ad7/12284493/f346c2e6f07d/gr2.jpg

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本文引用的文献

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Investigation of von Willebrand factor multimer abnormalities before and after aortic valve replacement using the Hydragel-5 assay.应用 Hydragel-5 assay 研究主动脉瓣置换术前及术后血管性血友病因子多聚体异常
Thromb Res. 2024 Sep;241:109094. doi: 10.1016/j.thromres.2024.109094. Epub 2024 Jul 6.
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Discrepant low von Willebrand factor activity results on the ACL TOP analyzer are frequent in unselected patients with myeloproliferative neoplasms and show no correlation with high-molecular-weight multimer loss or bleeding phenotype.在未经选择的骨髓增殖性肿瘤患者中,ACL TOP 分析仪上差异明显的低血管性血友病因子活性结果很常见,但与高分子量多聚体缺失或出血表型无关。
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Von Willebrand factor testing ratios in the diagnosis and subtyping of von Willebrand disease.在血管性血友病的诊断和分型中,血管性血友病因子检测比值的应用。
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Increased cleavage of von Willebrand factor by ADAMTS13 may contribute strongly to acquired von Willebrand syndrome development in patients with essential thrombocythemia.ADAMTS13 对血管性血友病因子的裂解增加可能是导致原发性血小板增多症患者获得性血管性血友病综合征发展的重要原因。
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Management Issues and Controversies in Low-Risk Patients with Essential Thrombocythemia and Polycythemia Vera.低危原发性血小板增多症和真性红细胞增多症患者的管理问题和争议。
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Acquired von Willebrand syndrome in myeloproliferative neoplasms with extreme thrombocytosis.伴有极度血小板增多的骨髓增殖性肿瘤中的获得性血管性血友病综合征
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