Voncento预防与按需治疗血管性血友病在英国的成本效益

IF 7.4 1区 医学 Q1 HEMATOLOGY
Michele Wilson, Giancarlo Castaman, Will Thomas, Carolyn Millar, Ginés Escolar, Wolfgang Miesbach, Cheryl McDade, Radovan Tomic, Songkai Yan
{"title":"Voncento预防与按需治疗血管性血友病在英国的成本效益","authors":"Michele Wilson, Giancarlo Castaman, Will Thomas, Carolyn Millar, Ginés Escolar, Wolfgang Miesbach, Cheryl McDade, Radovan Tomic, Songkai Yan","doi":"10.1182/bloodadvances.2024014376","DOIUrl":null,"url":null,"abstract":"<p><strong>Abstract: </strong>von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in patients with VWD treated with Voncento in the United Kingdom. A Markov structure was developed to estimate the quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon. Treatment options included ODT or LTP. For both options, we assumed plasma-derived VWF/factor VIII 2.4:1 (Voncento) as the VWF product used. Clinical parameters were obtained from published literature and Voncento's summary characteristics. Utility weights were obtained from published literature. Costs (in 2021 GBP [£]) and outcomes were discounted annually by 3.5%. Sensitivity analyses were conducted. Three baseline annual bleed rate (ABR) scenarios (11, 26.5, and 39.6) were considered. In the base-case analyses, Voncento LTP resulted in lower costs (-£831 206) and greater QALYs (6.14) vs ODT. Savings were primarily due to reductions in product use required (-£529 571) and bleed-related other medical costs (-£301 352). Compared with ODT, LTP also resulted in 322.52 fewer major bleeds and 515.68 fewer minor bleeds over a lifetime horizon. Probabilistic sensitivity analyses showed dominance in 96.12% of simulations and cost-effectiveness in 97.68% of simulations. For the 39.6 ABR scenario also, LTP was dominant compared with ODT. Results suggest that Voncento LTP is more effective and cost saving than ODT in the United Kingdom for patients with VWD with higher ABR. Prophylaxis for patients with frequent bleeds is likely to be a cost-saving and effective strategy.</p>","PeriodicalId":9228,"journal":{"name":"Blood advances","volume":" ","pages":"1312-1319"},"PeriodicalIF":7.4000,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Cost-effectiveness of Voncento prophylaxis vs on-demand treatment in von Willebrand disease in the United Kingdom.\",\"authors\":\"Michele Wilson, Giancarlo Castaman, Will Thomas, Carolyn Millar, Ginés Escolar, Wolfgang Miesbach, Cheryl McDade, Radovan Tomic, Songkai Yan\",\"doi\":\"10.1182/bloodadvances.2024014376\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Abstract: </strong>von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in patients with VWD treated with Voncento in the United Kingdom. A Markov structure was developed to estimate the quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon. Treatment options included ODT or LTP. For both options, we assumed plasma-derived VWF/factor VIII 2.4:1 (Voncento) as the VWF product used. Clinical parameters were obtained from published literature and Voncento's summary characteristics. Utility weights were obtained from published literature. Costs (in 2021 GBP [£]) and outcomes were discounted annually by 3.5%. Sensitivity analyses were conducted. Three baseline annual bleed rate (ABR) scenarios (11, 26.5, and 39.6) were considered. In the base-case analyses, Voncento LTP resulted in lower costs (-£831 206) and greater QALYs (6.14) vs ODT. Savings were primarily due to reductions in product use required (-£529 571) and bleed-related other medical costs (-£301 352). Compared with ODT, LTP also resulted in 322.52 fewer major bleeds and 515.68 fewer minor bleeds over a lifetime horizon. Probabilistic sensitivity analyses showed dominance in 96.12% of simulations and cost-effectiveness in 97.68% of simulations. For the 39.6 ABR scenario also, LTP was dominant compared with ODT. Results suggest that Voncento LTP is more effective and cost saving than ODT in the United Kingdom for patients with VWD with higher ABR. Prophylaxis for patients with frequent bleeds is likely to be a cost-saving and effective strategy.</p>\",\"PeriodicalId\":9228,\"journal\":{\"name\":\"Blood advances\",\"volume\":\" \",\"pages\":\"1312-1319\"},\"PeriodicalIF\":7.4000,\"publicationDate\":\"2025-03-25\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Blood advances\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1182/bloodadvances.2024014376\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood advances","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1182/bloodadvances.2024014376","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0

摘要

血管性血友病(VWD)的按需治疗(ODT)或长期预防(LTP)可能需要血管性血友病因子(VWF)浓缩物。本研究评估了英国Voncento治疗VWD患者LTP与ODT的成本-效果。开发了一个马尔可夫结构来估计质量调整生命年(QALYs)和VWD治疗在生命周期内的成本。治疗方案包括ODT或LTP。对于这两种选择,我们假设使用血浆源性VWF/因子VIII (pdVWF/FVIII) 2.4:1 (Voncento)作为VWF产品。临床参数从已发表的文献和Voncento的总结特征中获得。效用权重从已发表的文献中获得。成本(2021年英镑)和结果每年折现3.5%。进行敏感性分析。考虑了三种基线年出血率(ABR)情景(11,26.5和39.6)。在基本案例分析中,与ODT相比,Voncento LTP的成本更低(- 831,206英镑),质量质量(6.14)更高。节余主要是由于所需产品使用量减少(- 529 571英镑)和与出血有关的其他医疗费用减少(- 301 352英镑)。与ODT相比,LTP在一生中也减少了322.52例大出血和515.68例小出血。概率敏感性分析显示,96.12%的模拟占主导地位,97.68%的模拟具有成本效益。在ABR为39.6的情况下,LTP也比ODT占主导地位。结果表明,在英国,对于ABR较高的VWD患者,Voncento LTP比ODT更有效,成本更低。对频繁出血的患者进行预防可能是一种节省成本和有效的策略。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Cost-effectiveness of Voncento prophylaxis vs on-demand treatment in von Willebrand disease in the United Kingdom.

Abstract: von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in patients with VWD treated with Voncento in the United Kingdom. A Markov structure was developed to estimate the quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon. Treatment options included ODT or LTP. For both options, we assumed plasma-derived VWF/factor VIII 2.4:1 (Voncento) as the VWF product used. Clinical parameters were obtained from published literature and Voncento's summary characteristics. Utility weights were obtained from published literature. Costs (in 2021 GBP [£]) and outcomes were discounted annually by 3.5%. Sensitivity analyses were conducted. Three baseline annual bleed rate (ABR) scenarios (11, 26.5, and 39.6) were considered. In the base-case analyses, Voncento LTP resulted in lower costs (-£831 206) and greater QALYs (6.14) vs ODT. Savings were primarily due to reductions in product use required (-£529 571) and bleed-related other medical costs (-£301 352). Compared with ODT, LTP also resulted in 322.52 fewer major bleeds and 515.68 fewer minor bleeds over a lifetime horizon. Probabilistic sensitivity analyses showed dominance in 96.12% of simulations and cost-effectiveness in 97.68% of simulations. For the 39.6 ABR scenario also, LTP was dominant compared with ODT. Results suggest that Voncento LTP is more effective and cost saving than ODT in the United Kingdom for patients with VWD with higher ABR. Prophylaxis for patients with frequent bleeds is likely to be a cost-saving and effective strategy.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
Blood advances
Blood advances Medicine-Hematology
CiteScore
12.70
自引率
2.70%
发文量
840
期刊介绍: Blood Advances, a semimonthly medical journal published by the American Society of Hematology, marks the first addition to the Blood family in 70 years. This peer-reviewed, online-only, open-access journal was launched under the leadership of founding editor-in-chief Robert Negrin, MD, from Stanford University Medical Center in Stanford, CA, with its inaugural issue released on November 29, 2016. Blood Advances serves as an international platform for original articles detailing basic laboratory, translational, and clinical investigations in hematology. The journal comprehensively covers all aspects of hematology, including disorders of leukocytes (both benign and malignant), erythrocytes, platelets, hemostatic mechanisms, vascular biology, immunology, and hematologic oncology. Each article undergoes a rigorous peer-review process, with selection based on the originality of the findings, the high quality of the work presented, and the clarity of the presentation.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信