Pazopanib in treatment of Hereditary Hemorrhagic Telangiectasia-related epistaxis and gastrointestinal bleeding.

IF 5.5 2区 医学 Q1 HEMATOLOGY Journal of Thrombosis and Haemostasis Pub Date : 2024-10-23 DOI:10.1016/j.jtha.2024.10.014
Magdalena D Lewandowska, Shelby Gordon, Anthony Betbadal, Amy D Shapiro
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Abstract

Background: Hereditary hemorrhagic telangiectasia (HHT) is a bleeding disorder characterized by arteriovenous malformations (AVMs), commonly presenting with epistaxis and gastrointestinal bleeding. Bleeding symptoms may be difficult to manage and may become life-threatening, with many patients developing dependence on parenteral iron and/or blood transfusion. There is a growing body of evidence that antiangiogenic therapies may be effective in management of bleeding symptoms, presumably targeting pathogenic HHT pathways such as vascular endothelial growth factor (VEGF) receptor.

Objectives: To report single-center, retrospective real-world use of pazopanib, an orally administered tyrosine kinase inhibitor that blocks VEGF receptors, in six patients with HHT-associated epistaxis and/or gastrointestinal (GI) bleeding.

Patient/methods: A retrospective observational analysis was performed to assess the safety/efficacy of pazopanib use in patients with confirmed HHT-associated epistaxis and/or GI bleeding between 01 January 2019 and 14 June 2023 The Indiana Hemophilia and Thrombosis (IHTC) institutional EMR was queried for HHT patients who were treated with pazopanib for >3 months. Patient data were obtained from patient documentation, physician/nursing notes, and on-call documentation Institutional IRB approval was obtained for data pull as an exempt study RESULTS AND CONCLUSIONS: Our observations on the real-world use of pazopanib in six HHT patients with moderate to severe bleeding showed improvement in hemoglobin levels, with reduction in iron infusions and red blood cell transfusion requirement. Pazopanib may be a reasonable option for patients with HHT with epistaxis or gastrointestinal bleeding that are refractory to standard treatment.

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帕唑帕尼(Pazopanib)用于治疗遗传性出血性远端血管扩张症相关的鼻衄和消化道出血。
背景:遗传性出血性毛细血管扩张症(HHT)是一种以动静脉畸形(AVM)为特征的出血性疾病,通常表现为鼻衄和消化道出血。出血症状可能难以控制,并可能危及生命,许多患者开始依赖肠外铁剂和/或输血。越来越多的证据表明,抗血管生成疗法可有效控制出血症状,可能是针对血管内皮生长因子(VEGF)受体等 HHT 致病途径:报告帕唑帕尼(一种阻断血管内皮生长因子受体的口服酪氨酸激酶抑制剂)在6例HHT相关性鼻衄和/或胃肠道(GI)出血患者中的单中心、回顾性实际应用情况:对2019年1月1日至2023年6月14日期间确诊的HHT相关性鼻衄和/或消化道出血患者使用帕唑帕尼的安全性/有效性进行了回顾性观察分析,并查询了印第安纳州血友病和血栓症(IHTC)机构的EMR,以了解接受帕唑帕尼治疗超过3个月的HHT患者的情况。患者数据来自患者文档、医生/护理记录和值班文档。作为一项豁免研究,数据拉取已获得机构 IRB 批准。 结果与结论:我们观察到帕唑帕尼在现实世界中的使用情况:我们对 6 名中重度出血的 HHT 患者实际使用帕唑帕尼的观察结果显示,血红蛋白水平有所改善,铁剂输注和红细胞输注需求减少。对于标准治疗难治的鼻衄或消化道出血 HHT 患者,帕唑帕尼可能是一个合理的选择。
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来源期刊
Journal of Thrombosis and Haemostasis
Journal of Thrombosis and Haemostasis 医学-外周血管病
CiteScore
24.30
自引率
3.80%
发文量
321
审稿时长
1 months
期刊介绍: The Journal of Thrombosis and Haemostasis (JTH) serves as the official journal of the International Society on Thrombosis and Haemostasis. It is dedicated to advancing science related to thrombosis, bleeding disorders, and vascular biology through the dissemination and exchange of information and ideas within the global research community. Types of Publications: The journal publishes a variety of content, including: Original research reports State-of-the-art reviews Brief reports Case reports Invited commentaries on publications in the Journal Forum articles Correspondence Announcements Scope of Contributions: Editors invite contributions from both fundamental and clinical domains. These include: Basic manuscripts on blood coagulation and fibrinolysis Studies on proteins and reactions related to thrombosis and haemostasis Research on blood platelets and their interactions with other biological systems, such as the vessel wall, blood cells, and invading organisms Clinical manuscripts covering various topics including venous thrombosis, arterial disease, hemophilia, bleeding disorders, and platelet diseases Clinical manuscripts may encompass etiology, diagnostics, prognosis, prevention, and treatment strategies.
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