Fressinaud E, Veyradier A, Sigaud M, Boyer-Neumann C, Le Boterff C, Meyer D
Laboratoire d'Hématologie, CHU Hôtel Dieu, Nantes.
Br J Haematol. 1999 Sep;106(3):777-83. doi: 10.1046/j.1365-2141.1999.01604.x.
We have evaluated the position of the Platelet Function Analyzer PFA-100TM in the management of 41 patients with von Willebrand disease (VWD) receiving either desmopressin (23 patients with type 1, five with type 2M, three with type 2A and three with type 2B) or von Willebrand factor (VWF) concentrates (four patients with type 3, two with type 2M 'type B', two with type 2A and one type 1 'platelet low'). In all patients the following were studied before and 30 min after infusion of desmopressin and/or VWF concentrates: VWF ristocetin cofactor activity (VWFRCo), bleeding time (BT) and closure time with the PFA-100 using ADP (CT-ADP) as well as epinephrine (CT-Epi) cartridges. After the infusion of desmopressin, the CT was modified in the same way as the VWFRCo levels, being always normalized in patients with type 1 and not constantly corrected in those with type 2. Thus, our results indicated that the measurement of the CT enabled a quick and accurate evaluation of the response to desmopressin which, in fact, measured the releasable VWF cellular compartment containing the highly multimerized forms of VWF. For patients with type 2 or 3 VWD who were non-responsive to desmopressin, VWF concentrates corrected the VWFRCo defect but not the CT as none of these patients had a normal platelet VWF content and the VWF concentrates did not contain the ultralarge VWF multimers. In conclusion, the very high shear conditions in the PFA-100 make it very sensitive to the contribution of platelet VWF and to the ultralarge VWF multimers, indicating that the evaluation of the CT is a very simple and rapid tool to discriminate between good and non-responders to desmopressin.
我们评估了血小板功能分析仪PFA-100TM在41例接受去氨加压素（23例1型、5例2M型、3例2A型和3例2B型）或血管性血友病因子（VWF）浓缩物（4例3型、2例2M型“B型”、2例2A型和1例1型“血小板低”）治疗的血管性血友病（VWD）患者管理中的作用。在所有患者中，在输注去氨加压素和/或VWF浓缩物之前和之后30分钟研究了以下指标：VWF瑞斯托霉素辅因子活性（VWFRCo）、出血时间（BT）以及使用ADP（CT-ADP）和肾上腺素（CT-Epi）检测卡通过PFA-100测定的封闭时间。输注去氨加压素后，CT的变化方式与VWFRCo水平相同，1型患者的CT总是恢复正常，而2型患者则并非总是得到纠正。因此，我们的结果表明，CT的测量能够快速准确地评估对去氨加压素的反应，实际上，去氨加压素测量的是含有高度多聚化形式VWF的可释放VWF细胞部分。对于对去氨加压素无反应的2型或3型VWD患者，VWF浓缩物纠正了VWFRCo缺陷，但未纠正CT，因为这些患者均无正常的血小板VWF含量，且VWF浓缩物不含超大VWF多聚体。总之，PFA-100中的超高剪切条件使其对血小板VWF的贡献和超大VWF多聚体非常敏感，这表明CT评估是区分对去氨加压素反应良好和无反应者的非常简单快速的工具。
