Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Haemate P) in von Willebrand Disease: A Systematic Review and Pharmacovigilance Update

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2025-02-09 DOI:10.1111/hae.15138

Carmen Escuriola Ettingshausen, Riitta Lassila, Gines Escolar, Christoph Male, Kathrin Schirner, Lisa Heyder, Erik Berntorp

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Abstract

Background

Von Willebrand disease (VWD) is an inherited bleeding disorder caused by deficient or dysfunctional von Willebrand factor (VWF). VWF replacement therapy is indicated in VWD management.

Methods

This systematic review was conducted to evaluate all available evidence of the efficacy, safety, dosing and consumption of pasteurized plasma-derived human coagulation FVIII/human VWF (pdVWF/FVIII; Haemate P/Humate-P) concentrate for on-demand (OD) treatment, surgical prophylaxis and long-term prophylaxis of patients with VWD. A systematic search was performed in MEDLINE and Cochrane Library databases to identify studies (7 June 1982–31 May 2023) reporting the use of pdVWF/FVIII in VWD according to predefined selection criteria. Pharmacovigilance data were also retrieved for the same period.

Results

Fifteen studies were identified, 12 being observational and three interventional. Efficacy and safety assessments and treatment protocols varied across the studies which hindered direct comparisons. Haemostatic efficacy of pdVWF/FVIII was rated excellent/good for OD treatment in 95%–98% of bleeds and in 94%–100% of surgeries. In two separate studies, prophylactic efficacy was rated excellent/good in 100% of treatment cycles. Where reported, median annualized bleeding rates decreased from 3–24 prior prophylaxis to 0.5–6 during prophylaxis. Analysis of pharmacovigilance safety reports showed that pdVWF/FVIII was associated with a low rate of adverse events.

Conclusions

This systematic literature review and analysis of pharmacovigilance data summarize evidence of over 40 years of clinical use of pdVWF/FVIII, supporting its safety and efficacy in VWD.

Abstract Image

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血管性血友病血浆源性血管性血友病因子/因子VIII浓缩物（Haemate P）：系统综述和药物警戒更新

背景：血管性血友病（VWD）是一种由血管性血友病因子（VWF）缺陷或功能障碍引起的遗传性出血性疾病。VWF替代疗法适用于VWD的治疗。方法：本系统综述旨在评估巴氏消毒血浆源性人凝血FVIII/人VWF (pdVWF/FVIII；Haemate P/Humate-P)浓缩物用于VWD患者的按需（OD）治疗、手术预防和长期预防。在MEDLINE和Cochrane图书馆数据库中进行系统检索，根据预先确定的选择标准确定报告在VWD中使用pdVWF/FVIII的研究（1982年6月7日至2023年5月31日）。还检索了同一时期的药物警戒数据。结果：共纳入15项研究，其中12项为观察性研究，3项为干预性研究。不同研究的疗效和安全性评估以及治疗方案各不相同，妨碍了直接比较。pdVWF/FVIII在OD治疗中95%-98%的出血和94%-100%的手术中止血效果为优/良。在两项独立的研究中，预防效果在100%的治疗周期中被评为优秀/良好。在报告中，年平均出血率从预防前的3-24下降到预防期间的0.5-6。药物警戒安全性报告分析显示，pdVWF/FVIII与不良事件发生率低相关。结论：本系统的文献综述和药物警戒数据分析总结了40多年来pdVWF/FVIII临床使用的证据，支持其治疗VWD的安全性和有效性。

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来源期刊

Haemophilia 医学-血液学

CiteScore

6.50

自引率

28.20%

发文量

226

审稿时长

3-6 weeks

期刊介绍： Haemophilia is an international journal dedicated to the exchange of information regarding the comprehensive care of haemophilia. The Journal contains review articles, original scientific papers and case reports related to haemophilia care, with frequent supplements. Subjects covered include: clotting factor deficiencies, both inherited and acquired: haemophilia A, B, von Willebrand''s disease, deficiencies of factor V, VII, X and XI replacement therapy for clotting factor deficiencies component therapy in the developing world transfusion transmitted disease haemophilia care and paediatrics, orthopaedics, gynaecology and obstetrics nursing laboratory diagnosis carrier detection psycho-social concerns economic issues audit inherited platelet disorders.