真性红细胞增多症成年患者疾病管理的观察性研究：来自大型美国索赔数据库的结果。

IF 2.1 4区医学 Q2 HEMATOLOGY

Expert Review of Hematology Pub Date : 2025-07-27 DOI:10.1080/17474086.2025.2538542

Andrew Kuykendall, Lucy Bellamy, Lopa Desai, Omari Morrison, Larisa Gofman, Arturo Molina

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引用次数: 0

摘要

背景：真性红细胞增多症（PV）的特点是红细胞增多和血栓形成事件（TEs）的风险增加。目前，PV的标准治疗方法存在局限性，因此有必要了解PV的实际治疗模式和治疗负担。研究设计和方法：本回顾性观察性研究使用美国Komodo Health索赔数据库（2016-2022）分析了成年PV患者的真实索赔数据。繁重的治疗被归类为6个月内接受≥3次放血（phl）和/或每天高剂量羟基脲(HU)≥1,000 mg的患者。结果：44,766例治疗患者(平均年龄：65岁；（64%男性），55%接受了繁重的治疗，其中包括频繁PHL（33%）、高剂量HU（17%）或两者联合（频繁PHL +高剂量HU， 5%）。PHL和HU是最常见的一线治疗(PHL, 71%；HU, 27%)， 87%的患者在开始接受PHL单药治疗时从未进展到另一种治疗方案。16%的患者发生TEs。结论：这些数据表明，相当大比例的PV患者接受繁重的治疗，55%的治疗患者接受频繁的PHL和/或大剂量HU，突出了治疗优化的必要性。但是，应当考虑到使用索赔数据的固有局限性。

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An observational study of disease management in adult patients with polycythemia vera: results from a large U.S. claims database.

Background: Polycythemia vera (PV) is characterized by erythrocytosis and an increased risk of thrombotic events (TEs). Currently, standard-of-care therapies for PV have limitations, which indicate the need to understand real-world treatment patterns and treatment burden in PV.

Research design and methods: This retrospective observational study analyzed real-world claims data in adult patients with PV using the Komodo Health claims database (2016-2022) in the United States. Burdensome treatment was classified as patients receiving ≥ 3 phlebotomies (PHLs) within a 6-month period and/or high-dose hydroxyurea (HU) ≥ 1,000 mg per day.

Results: Of 44,766 treated patients (mean age: 65 years; 64% male), 55% received burdensome treatment, which included frequent PHL (33%), high-dose HU (17%), or a combination of both (frequent PHL + high-dose HU, 5%). PHL and HU were the most common first-line treatments (PHL, 71%; HU, 27%), and 87% of patients initiating treatment with PHL monotherapy never advanced to another therapy regimen. TEs occurred in 16% of the treated patients.

Conclusions: These data suggest a substantial proportion of patients with PV receive burdensome treatments, with 55% of treated patients receiving frequent PHL and/or high-dose HU, highlighting need for therapy optimization. However, inherent limitations of using claims data should be taken into consideration.

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来源期刊

Expert Review of Hematology HEMATOLOGY-

CiteScore

4.70

自引率

3.60%

发文量

审稿时长

6-12 weeks

期刊介绍： Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.