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High-titre inhibitors in previously untreated patients with severe haemophilia A receiving recombinant or plasma-derived factor VIII: a budget-impact analysis.在接受重组或血浆源性凝血因子VIII治疗的既往未治疗的重度甲型血友病患者中高滴度抑制剂：预算影响分析
Blood Transfus. 2018 Feb;16(2):215-220. doi: 10.2450/2017.0352-16. Epub 2017 May 15.
2
The economics of inpatient on-demand treatment for haemophilia with high-responding inhibitors: a US retrospective data analysis.高反应性抑制剂血友病按需住院治疗的经济学：美国回顾性数据分析。
Haemophilia. 2012 Mar;18(2):284-90. doi: 10.1111/j.1365-2516.2011.02623.x. Epub 2011 Aug 4.
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Hemostatic treatment using factor VIII concentrates for neutralizing high-responding inhibitors prior to CVAD insertion for immune-tolerance induction therapy.在进行免疫耐受诱导治疗前，使用因子 VIII 浓缩物进行止血治疗以中和高反应性抑制剂，以进行 CVAD 插入。
Clin Appl Thromb Hemost. 2012 Jan-Feb;18(1):66-71. doi: 10.1177/1076029611412364. Epub 2011 Jul 6.
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Successful immune tolerance induction with factor VIII/von Willebrand factor concentrate in an elderly patient with severe haemophilia A and a high responder inhibitor.在一名患有严重甲型血友病且抑制剂高反应性的老年患者中，使用凝血因子VIII/血管性血友病因子浓缩物成功诱导免疫耐受。
Blood Transfus. 2010 Jan;8(1):66-8. doi: 10.2450/2009.0078-09.
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High titre inhibitor to factor VIII in a haemophilia carrier.一名血友病携带者体内存在高滴度的凝血因子VIII抑制物。
Haemophilia. 2014 May;20(3):e237-40. doi: 10.1111/hae.12417.
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Inhibitors in Severe Hemophilia A: 25-Year Experience in Slovakia.重度A型血友病的抑制剂：斯洛伐克的25年经验
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Long-term treatment course of a patient with mild haemophilia A who developed a high titre factor VIII inhibitor.一名轻度甲型血友病患者出现高滴度VIII因子抑制物的长期治疗过程。
Haemophilia. 2014 Nov;20(6):e402-4. doi: 10.1111/hae.12491.
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Cost analysis of plasma-derived factor VIII/von Willebrand factor versus recombinant factor VIII for treatment of previously untreated patients with severe hemophilia A in the United States.美国血浆源性凝血因子VIII/血管性血友病因子与重组凝血因子VIII治疗既往未治疗的重度甲型血友病患者的成本分析
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Factor VIII products in haemophilia A: one size fits all?甲型血友病中的凝血因子VIII产品：一刀切可行吗？
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Reduced early prophylaxis of children with haemophilia in a developing country, Turkey.发展中国家土耳其血友病患儿早期预防措施减少
Haemophilia. 2011 Sep;17(5):e840-1. doi: 10.1111/j.1365-2516.2011.02581.x. Epub 2011 Jun 20.

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1
"The times they are a-changin", ... or maybe not?“时代在变”，…… 或者也许并非如此？
Blood Transfus. 2019 Nov;17(6):401-402. doi: 10.2450/2019.0259-19. Epub 2019 Dec 11.
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Emicizumab for the treatment of haemophilia A: a narrative review.依库珠单抗治疗 A 型血友病：一篇叙述性综述。
Blood Transfus. 2019 May;17(3):223-228. doi: 10.2450/2019.0026-19.
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Factors affecting the quality, safety and marketing approval of clotting factor concentrates for haemophilia.影响血友病凝血因子浓缩物质量、安全性和上市批准的因素。
Blood Transfus. 2018 Nov;16(6):525-534. doi: 10.2450/2018.0150-18. Epub 2018 Sep 3.
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Further comments on "High-titre inhibitors in previously untreated patients with severe haemophilia A receiving recombinant or plasma-derived factor VIII: a budget-impact analysis".对“在接受重组或血浆源性凝血因子VIII治疗的既往未治疗的重度A型血友病患者中高滴度抑制剂：预算影响分析”的进一步评论
Blood Transfus. 2019 Jan;17(1):86. doi: 10.2450/2017.0253-17. Epub 2017 Dec 22.
5
Reply to "High-titre inhibitors in previously untreated patients with severe haemophilia A receiving recombinant or plasma-derived factor VIII: a budget-impact analysis" by Messori et al.对梅索里等人《接受重组或血浆源性凝血因子 VIII 治疗的既往未治疗的重度甲型血友病患者中的高滴度抑制剂：预算影响分析》一文的回复
Blood Transfus. 2019 Jan;17(1):85. doi: 10.2450/2017.0160-17. Epub 2017 Oct 3.

本文引用的文献

1
Economic analysis of not running tenders for recombinant Factor VIII procurement: a simplified analysis to estimate an otherwise unknown pharmacoeconomic index.重组凝血因子VIII采购不进行招标的经济分析：一种用于估算未知药物经济学指标的简化分析方法。
Eur J Hosp Pharm. 2016 Jul;23(4):219-223. doi: 10.1136/ejhpharm-2015-000728. Epub 2015 Dec 23.
2
A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A.A 随机对照试验VIII 因子和中和抗体在血友病 A.
N Engl J Med. 2016 May 26;374(21):2054-64. doi: 10.1056/NEJMoa1516437.
3
Risk of inhibitors in previously untreated patients with hemophilia: a meta-analysis of literature studies.既往未经治疗的血友病患者中出现抑制物的风险：文献研究的荟萃分析
Blood. 2015 Jun 11;125(24):3819-21. doi: 10.1182/blood-2015-01-621383.
4
Using value-of-information methods when the disease is rare and the treatment is expensive--the example of hemophilia A.在疾病罕见且治疗昂贵的情况下使用信息价值方法——以甲型血友病为例。
J Gen Intern Med. 2014 Aug;29 Suppl 3(Suppl 3):S767-73. doi: 10.1007/s11606-014-2880-3.
5
Purchasing factor concentrates in the 21st century through competitive tendering.21 世纪通过竞争性招标集中采购要素。
Haemophilia. 2013 Sep;19(5):660-7. doi: 10.1111/hae.12169. Epub 2013 May 28.
6
Consolidated Health Economic Evaluation Reporting Standards (CHEERS)--explanation and elaboration: a report of the ISPOR Health Economic Evaluation Publication Guidelines Good Reporting Practices Task Force.健康经济评估报告标准（CHEERS）——解释与说明：国际卫生经济学会健康经济评估报告指南良好报告实践工作组报告。
Value Health. 2013 Mar-Apr;16(2):231-50. doi: 10.1016/j.jval.2013.02.002.
7
How we choose factor VIII to treat hemophilia.我们如何选择因子 VIII 治疗血友病。
Blood. 2012 May 3;119(18):4108-14. doi: 10.1182/blood-2012-01-394411. Epub 2012 Mar 12.
8
Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group.血友病合并抑制物患者的护理成本与生活质量：COCIS研究组
Blood. 2003 Oct 1;102(7):2358-63. doi: 10.1182/blood-2003-03-0941. Epub 2003 Jun 19.
9
Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper.
Blood. 2000 Sep 1;96(5):1698-702.

High-titre inhibitors in previously untreated patients with severe haemophilia A receiving recombinant or plasma-derived factor VIII: a budget-impact analysis.

作者信息

Messori Andrea, Peyvandi Flora, Trippoli Sabrina, Palla Roberta, Rosendaal Frits R, Mannucci Pier Mannuccio

机构信息

HTA Unit, ESTAR, Regional Health Service, Florence, Italy.

"A. Bianchi Bonomi" Haemophilia and Thrombosis Centre, "Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico", Milan, Italy.

出版信息

Blood Transfus. 2018 Feb;16(2):215-220. doi: 10.2450/2017.0352-16. Epub 2017 May 15.

DOI:10.2450/2017.0352-16
PMID:28686156
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5839620/
Abstract
摘要