Haemophilia：1：1 比例的 VWF/FVIII 复合物对血管性血友病患者的疗效

2024-08-15 将军的九分裤 MedSci原创发表于上海

该围手术期研究证明VWF浓缩液的比例为1:1在VWD患者中具有良好的FVIII和VWF恢复效果

血管性血友病(VWD)患者因血管性血友病因子(VWF)缺陷和/或缺陷而有出血倾向增加的症状。VWD的治疗取决于VWD的类型、预先存在的因子VIII(FVIII)水平、VWF活性水平和临床参数/特征，患者围手术期需给予血管性血友病因子(VWF)浓缩液。考虑到反复注射1:1比例的VWF/FVIII凝血因子浓缩液(CFC)后FVIII积累的问题，这项研究主要探讨了恢复和FVIII积累随时间的变化。

图1：FVIII和VWF活性恢复的散点和线性回归斜率的95%CI:0.52~0.85

这项单中心研究对VWD患者进行围手术期1:1比例CFC输注研究。符合条件的参与者是在研究团队所在的血友病治疗中心治疗的所有类型的VWD的儿童和成人患者，他们从2019年12月至2022年10月期间接受了CFC补充手术。CFC的剂量根据体重和内源性VWF/FVIII活性定。在T0(基线)、T1(输液后15分钟)和T2-T6(24-120h)时监测FVIII和VWF活性。

这项研究一共纳入了125名患者，包括23名儿童，接受了276次CFC管理，进行了125次手术，在所有手术中，大手术63例(50.4%)，小手术62例(49.6%)。大手术患者输液183次(66.3%)，小手术患者输液93次(33.7%)。共纳入5例获得性血管性血友病综合征(VWS)患者。平均给药剂量为35.7IU/kgCFC时，FVIII和VWF的回收率分别为2.6IU/dL/IU/kg和2.4IU/dL/IU/kg。

图2：(A)125名接受一次或多次浓缩凝血因子输注的VWD患者随时间的平均FVIII和VWF活性水平。x轴是时间点，以小时为单位，每个时间点有多少病人。y轴上是IU/dL。(B)接受三次或三次以上浓缩凝血因子输注的患者随时间的平均FVIII和VWF。

T0时平均FVIII水平为62(SD51.9)，T1时平均FVIII水平为164(SD80.4)，T2时平均FVIII水平为155(SD62.8)，T3时平均FVIII水平为162(SD59.8)，T4和T5时分别为124(SD78.4)和120(SD65.3)IU/dL。T0时VWF平均活性水平为29(SD25.0)，T1为133(SD43.7)，T2何T3分别为92(SD37.2)和86(SD37.5)IU/dL。亚组分析在47例患者超过3次输液，显示没有积累平均FVIII水平。

这项研究集中于VWF/FVIII比值较低的VWF浓缩液(Wilate®)对接受手术的VWD患者的恢复和积累。研究证明，在所有患者中，单次输注Wilate®后，VWF和FVIII均有良好的恢复，仅输三次CFC就覆盖了大多数重大手术，且重复给药后FVIII无积累。该围手术期研究证明VWF浓缩液的比例为1:1在VWD患者中具有良好的FVIII和VWF恢复效果。

原始出处：

Kuppens GZL, Fischer K, van Galen KPM, et al. Efficacy of a 1:1 ratio VWF/FVIII concentrate in patients with von Willebrand disease. Haemophilia. Published online July 15, 2024. doi:10.1111/hae.15079

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2024-08-15 13013f32m56暂无昵称来自河北省

学习知识提升技能，学习学习

53 0
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2024-08-16 Dew· 来自新疆

感谢您的分享

48 0
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2024-08-15 yangchou 来自浙江省

好文章，谢谢分享。

47 0
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2024-08-15 梅斯管理员来自上海

#血液学# #凝血障碍# #血管性血友病# #VWF/FVIII 复合物#

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