Favaloro Emmanuel J
Diagnostic Haemostasis Laboratory, Department of Haematology, Institute of Clinical Pathology and Medical Research, Westmead Hospital, Westmead, New South Wales, Australia.
Semin Thromb Hemost. 2006 Sep;32(6):566-76. doi: 10.1055/s-2006-949662.
Clinical management of von Willebrand disease (or von Willebrand disorder [vWD]) often involves factor replacement or desmopressin acetate (DDAVP) therapy to control (potential) bleeding. Laboratory monitoring involves testing patient samples prior to therapy and at discreet time points after therapy. Classical testing generally comprises assays for factor VIII:coagulant activity, von Willebrand factor (vWF):antigen and vWF:ristocetin cofactor activity. The PFA-100 (platelet function analyser) is a relatively new tool for the investigation of primary hemostasis, and studies have shown its potential utility in identifying both vWD and platelet disorders, and in monitoring DDAVP therapy in these patients. However, the PFA-100 has limited utility in monitoring factor replacement therapy. The collagen-binding activity (vWF:CB) assay is a relatively new functional vWF assay and studies have also shown its utility in identifying vWD, and in monitoring both DDAVP and factor replacement therapy in these patients. This review assesses the laboratory monitoring of therapy for vWD with a special focus on the combined potential utility of the PFA-100 and a vWF:CB assay sensitive for the presence or absence of large vWF multimers. This review should be of value to both hemostasis scientists and clinical specialists.
血管性血友病（或血管性血友病紊乱症[vWD]）的临床管理通常涉及因子替代疗法或醋酸去氨加压素（DDAVP）治疗，以控制（潜在的）出血情况。实验室监测包括在治疗前以及治疗后的特定时间点对患者样本进行检测。传统检测通常包括对凝血因子VIII：凝血活性、血管性血友病因子（vWF）：抗原以及vWF：瑞斯托霉素辅因子活性的测定。PFA - 100（血小板功能分析仪）是一种用于原发性止血研究的相对较新的工具，研究表明其在识别vWD和血小板疾病以及监测这些患者的DDAVP治疗方面具有潜在用途。然而，PFA - 100在监测因子替代疗法方面的用途有限。胶原结合活性（vWF：CB）测定是一种相对较新的功能性vWF测定方法，研究也表明其在识别vWD以及监测这些患者的DDAVP和因子替代疗法方面的用途。本综述评估了vWD治疗的实验室监测，特别关注PFA - 100和对大vWF多聚体的存在与否敏感的vWF：CB测定的联合潜在用途。本综述对止血科学家和临床专家均应具有价值。
