Management of inherited von Willebrand disease in 2006
Management of inherited von Willebrand disease in 2006
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DOI：
10.1055/s-2006-949666
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发表时间：
2006-09-01
影响因子：
5.7
通讯作者：
Federici, Augusto B.
中科院分区：
文献类型：
--
作者：
Federici, Augusto B.
Von Willebrand病(VWD)的治疗目的是纠正止血的双重缺陷(即von Willebrand因子[VWF]减少和/或功能障碍导致的血小板粘附性异常和低水平的凝血因子VIII型凝血功能异常)。醋酸去氨加压素(DDAVP)是治疗I型VWD的首选药物，因为它可以诱导正常的VWF从细胞室释放。关于DDAVP对1型和2型VWD的生物学反应和临床疗效的前瞻性研究正在进行中，以探索其作为一种治疗选择的益处和局限性。在3型和严重的1型和2型VWD中，DDAVP无效，对这些患者来说，含有VWF和FVIII的血浆病毒灭活浓缩物是主要的治疗方法。有几种中纯度和高纯度的VWF/FVIII浓缩液可供选择，并已被证明在临床实践(出血和手术)中有效。新的VWF产品几乎不含FVIII，目前正在临床实践中进行评估。尽管反复输注浓缩液的VWD患者很少发生血栓事件，但有人担心持续的高FVIII水平可能会增加术后静脉血栓形成的风险。VWF/FVIII给药的剂量和时间应计划为将FVIII水平保持在50至150U/dL之间。适当的剂量和重复输液的时机对接受二次长期预防复发出血的患者也非常重要。
The aim of treatment of von Willebrand disease (VWD) is to correct the dual defect of hemostasis (i.e., the abnormal platelet adhesion due to reduced and/or dysfunctional von Willebrand factor [VWF] and the abnormal coagulation expressed by low levels of factor [F] VIII). Desmopressin acetate (DDAVP) is the treatment of choice for type I VWD because it can induce release of normal VWF from cellular compartments. Prospective studies on biological response versus clinical efficacy of DDAVP in VWD types 1 and 2 are in progress to explore its benefits and limits as a therapeutic option. In type 3 and in severe forms of type 1 and 2 VWD, DDAVP is not effective, and for these patients plasma virally inactivated concentrates containing VWF and FVIII are the mainstay of treatment. Several intermediate- and high-purity VWF/FVIII concentrates are available and have been shown to be effective in clinical practice (bleeding and surgery). New VWF products almost devoid of FVIII are now under evaluation in clinical practice. Although thrombotic events are rare in VWD patients receiving repeated infusions of concentrates, there is some concern that sustained high FVIII levels may increase risk of postoperative venous thromboembolism. Dosage and timing of VWF/FVIII administrations should be planned to keep the FVIII level between 50 and 150 U/dL. Appropriate dosage and timing in repeated infusions are also very important in patients exposed to secondary long-term prophylaxis for recurrent bleedings.