Efficacy of a 1:1 ratio VWF/FVIII concentrate in patients with von Willebrand disease

IF 3 2区医学 Q2 HEMATOLOGY

Haemophilia Pub Date : 2024-07-15 DOI:10.1111/hae.15079

Geoffrey Z. L. Kuppens, Kathelijn Fischer, Karin P. M. van Galen, Eduard J. van Beers, Paul R. Van der Valk, Idske C. L. Kremer Hovinga, Lize F. D. van Vulpen, Roger E. G. Schutgens

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Abstract

Introduction

Patients with von Willebrand disease (VWD) require administration of von Willebrand factor (VWF) concentrates peri-operatively. Concerns about FVIII accumulation after repetitive injections of a 1:1 ratio VWF/FVIII clotting factor concentrate (CFC) led this study to explore the recovery and FVIII accumulation over time.

Methods

This monocentre study examined patients with VWD receiving perioperative 1:1 ratio CFC infusions. CFC dosing was based on body weight and endogenous VWF/FVIII activity. FVIII and VWF activity was monitored at T0 (baseline), T1 (15 min postinfusion), and trough levels at T2-T6 (24-120 h).

Results

We included 125 patients, undergoing 125 procedures (63 major surgeries, 62 minor), with a median of two CFC infusions (IQR 1–3). With a mean administered dose of 35.7 IU/kg CFC, recovery rates of FVIII and VWF were 2.6 IU/dL per IU/kg and 2.4 IU/dL per IU/kg, respectively. Mean FVIII levels at T0 were 62 (SD 51.9), T1: 164 (SD 80.4), T2: 155 (SD 62.8), T3: 162 (SD 59.8), T4: 124 (SD 78.4), and T5: 120 (SD 65.3) IU/dL. Mean VWF activity levels at T0 were 29 (SD 25.0), T1: 133 (SD 43.7), T2: 92 (SD 37.2), and T3: 86 (SD 37.5) IU/dL. Subgroup analysis in 47 patients with more than three infusions, showed no accumulation of mean FVIII levels.

Conclusion

This perioperative study demonstrated excellent FVIII and VWF recovery of a 1:1 ratio VWF product in patients with VWD. Stable FVIII and VWF activity levels were observed after repeated infusions, without accumulation. Most major surgeries required only three CFC infusions.

Abstract Image

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1:1比例的VWF/FVIII浓缩液对von Willebrand病患者的疗效。

简介：冯-威廉氏病（VWD）患者需要在围手术期注射冯-威廉因子（VWF）浓缩物。由于担心重复注射比例为 1:1 的 VWF/FVIII 凝血因子浓缩物（CFC）后会导致 FVIII 的蓄积，本研究对 FVIII 随时间推移的恢复和蓄积情况进行了探讨：这项单中心研究考察了接受围手术期 1:1 比例 CFC 输注的 VWD 患者。CFC 剂量根据体重和内源性 VWF/FVIII 活性而定。FVIII和VWF活性在T0（基线）、T1（输液后15分钟）和T2-T6（24-120小时）的谷值水平进行监测：我们纳入了 125 名患者，他们接受了 125 次手术（63 次大手术，62 次小手术），中位数为两次 CFC 输注（IQR 1-3）。CFC 的平均给药剂量为 35.7 IU/kg，FVIII 和 VWF 的恢复率分别为 2.6 IU/dL per IU/kg 和 2.4 IU/dL per IU/kg。T0 时的平均 FVIII 水平为 62（标清 51.9），T1：164（标清 80.4），T2：155（标清 62.8），T3：162（标清 59.8），T4：124（标清 78.4），T5：120（标清 65.3）IU/dL。T0 时的平均 VWF 活性水平为 29（标清 25.0），T1：133（标清 43.7），T2：92（标清 37.2），T3：86（标清 37.5）IU/dL。对输注三次以上的 47 名患者进行的亚组分析表明，平均 FVIII 水平没有累积：这项围手术期研究表明，1:1 比例的 VWF 产品对 VWD 患者的 FVIII 和 VWF 恢复效果极佳。反复输注后观察到稳定的 FVIII 和 VWF 活性水平，没有出现累积现象。大多数大手术只需输注三次 CFC。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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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.