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ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease.美国血液学会(ASH)、国际血栓与止血学会(ISTH)、美国国家血友病基金会(NHF)、世界血友病联盟(WFH)2021年血管性血友病管理指南
Blood Adv. 2021 Jan 12;5(1):301-325. doi: 10.1182/bloodadvances.2020003264.
2
Major differences in clinical presentation, diagnosis and management of men and women with autosomal inherited bleeding disorders.常染色体遗传性出血性疾病男性和女性患者在临床表现、诊断及治疗方面的主要差异。
EClinicalMedicine. 2021 Jan 29;32:100726. doi: 10.1016/j.eclinm.2021.100726. eCollection 2021 Feb.
3
Management of von Willebrand disease with a factor VIII-poor von Willebrand factor concentrate: Results from a prospective observational post-marketing study.使用低VIII因子血管性血友病因子浓缩物治疗血管性血友病:一项上市后前瞻性观察研究的结果
J Thromb Haemost. 2020 Aug;18(8):1922-1933. doi: 10.1111/jth.14928. Epub 2020 Jun 25.
4
Replacement Therapy in Patients with Von Willebrand Disease-Indications and Monitoring.《血管性血友病患者的替代治疗:适应证和监测》
Hamostaseologie. 2019 Nov;39(4):326-338. doi: 10.1055/s-0039-1692688. Epub 2019 Jul 30.
5
The prevalence and burden of hand and wrist bleeds in von Willebrand disease.血管性血友病因子病中手部和腕部出血的患病率及负担
Haemophilia. 2019 Jan;25(1):e35-e38. doi: 10.1111/hae.13632. Epub 2018 Nov 5.
6
Advances in the diagnosis and treatment of Von Willebrand disease.血管性血友病诊断与治疗的进展
Blood. 2017 Nov 30;130(22):2386-2391. doi: 10.1182/blood-2017-05-782029.
7
The cost of severe haemophilia in Europe: the CHESS study.欧洲重度血友病的成本:CHESS研究
Orphanet J Rare Dis. 2017 May 31;12(1):106. doi: 10.1186/s13023-017-0660-y.
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Front Pharmacol. 2016 Dec 23;7:507. doi: 10.3389/fphar.2016.00507. eCollection 2016.
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Heavy menstrual bleeding and health-associated quality of life in women with von Willebrand's disease.血管性血友病女性的月经过多与健康相关生活质量
Exp Ther Med. 2016 May;11(5):1923-1929. doi: 10.3892/etm.2016.3144. Epub 2016 Mar 10.
10
Diagnostic approach to von Willebrand disease.血管性血友病的诊断方法。
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欧洲血管性血友病的成本:CVESS 研究。

The Cost of Von Willebrand Disease in Europe: The CVESS Study.

机构信息

597121HCD Economics, Daresbury, UK.

Centre for Haematology, Department of Immunology and Inflammation, 216773Imperial College London, London, UK.

出版信息

Clin Appl Thromb Hemost. 2022 Jan-Dec;28:10760296221120583. doi: 10.1177/10760296221120583.

DOI:10.1177/10760296221120583
PMID:35979588
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9393670/
Abstract

BACKGROUND

Von Willebrand disease (VWD) is one of the most common inherited bleeding disorders, imposing a substantial health impact and financial burden. The ost of on Willebrand disease in urope: A ocioeconomic tudy (CVESS) characterises the socio-economic cost of VWD across Germany, Spain, Italy, France, and the UK.

METHODS

A retrospective, cross-sectional design captured 12 months of patient disease management, collected from August-December 2018, for 974 patients. This enabled estimation of direct medical, direct non-medical and indirect costs, utilising prevalence estimates to extrapolate to population level.

RESULTS

Total annual direct medical cost (including/excluding von Willebrand factor [VWF]) across all countries was the highest cost (€2 845 510 345/€444 446 023), followed by indirect costs (€367 330 271) and direct non-medical costs (€60 223 234). Differences were seen between countries: the UK had the highest direct medical costs excluding VWF (€159 791 064), Italy the highest direct-non medical (€26 564 496), and Germany the highest indirect cost burden (€197 036 052). Total direct medical costs per adult patient increased across VWD types with Type 1 having the lowest cost (€23 287) and Type 3 having the highest cost (€133 518).

CONCLUSION

A substantial financial burden arises from the prevalence of VWD for the European healthcare systems considered.

摘要

背景

血管性血友病(VWD)是最常见的遗传性出血性疾病之一,对健康和经济造成重大影响。欧洲血管性血友病的成本:一项社会经济学研究(CVESS)描述了德国、西班牙、意大利、法国和英国的 VWD 社会经济成本。

方法

回顾性、横断面设计,对 974 例患者的 12 个月疾病管理数据进行了收集,时间为 2018 年 8 月至 12 月。这使我们能够利用患病率估计值估算直接医疗、直接非医疗和间接成本。

结果

所有国家的年直接医疗总费用(包括/不包括血管性血友病因子[VWF])均为最高成本(€2845510345/€444446023),其次是间接成本(€367330271)和直接非医疗成本(€60223234)。各国之间存在差异:英国不包括 VWF 的直接医疗费用最高(€159791064),意大利的直接非医疗费用最高(€26564496),德国的间接成本负担最高(€197036052)。每种 VWD 类型的成年患者的直接医疗总费用均随类型增加而增加,其中 1 型的成本最低(€23287),3 型的成本最高(€133518)。

结论

考虑到所考虑的欧洲医疗保健系统中 VWD 的流行程度,存在着巨大的经济负担。