Srivastava A, Serban M, Werner S, Schwartz B A, Kessler C M
Department of Haematology, Christian Medical College, Vellore, India.
University Emergency Pediatric Hospital Louis Turcanu, Timisoara, Romania.
Haemophilia. 2017 Mar;23(2):264-272. doi: 10.1111/hae.13106. Epub 2016 Dec 27.
Surgical procedures in von Willebrand disease (VWD) patients may require prophylactic treatment with exogenous von Willebrand factor (VWF) and coagulation factor VIII (FVIII) to prevent excessive bleeding. Wilate is a plasma-derived, double virus-inactivated, highly purified, freeze-dried VWF/FVIII concentrate, containing both factors in a physiological activity ratio of 1:1.
To investigate the efficacy and safety of wilate in maintaining haemostasis in VWD patients undergoing surgical procedures.
This prospective, open-label multinational clinical study documents 28 individuals who underwent 30 surgical procedures managed with wilate . Twenty-one patients had VWD Type 3, and 21 surgeries were major. Efficacy was assessed intra- and postoperatively by the surgeon and investigator, respectively, and adjudicated by an Independent Data Monitoring Committee, using an objective scale based on blood loss, transfusion requirements and postoperative bleeding and oozing. Treatment success (primary endpoint) was determined using a composite assessment algorithm and was formally assessed.
Surgical prophylaxis with wilate was successful in 29 of 30 procedures. The overall rate of success was 96.7% (98.75% CI: 0.784, 1.000). All 21 surgeries in patients with VWD Type 3 were managed successfully. There was no accumulation of VWF or FVIII after multiple dosing, and no thromboembolic events or inhibitors to VWF or FVIII were observed.
Wilate demonstrated effective prevention and treatment of bleeding in inherited VWD patients undergoing surgery, with no clinically significant safety concerns.
血管性血友病（VWD）患者的外科手术可能需要使用外源性血管性血友病因子（VWF）和凝血因子VIII（FVIII）进行预防性治疗，以防止过度出血。Wilate是一种源自血浆、经过双重病毒灭活、高度纯化的冻干VWF/FVIII浓缩物，两种因子的生理活性比例为1:1。
研究Wilate在接受外科手术的VWD患者中维持止血的有效性和安全性。
这项前瞻性、开放标签的多国临床研究记录了28例接受30次使用Wilate治疗的外科手术的患者。21例患者为3型VWD，其中21例手术为大手术。分别由外科医生和研究者在术中及术后评估疗效，并由独立数据监测委员会根据失血量、输血需求以及术后出血和渗血情况使用客观量表进行判定。使用综合评估算法确定治疗成功（主要终点）并进行正式评估。
30例手术中有29例使用Wilate进行手术预防成功。总体成功率为96.7%（98.75%可信区间：0.784，1.000）。所有21例3型VWD患者的手术均成功完成。多次给药后未出现VWF或FVIII蓄积，未观察到血栓栓塞事件或针对VWF或FVIII的抑制剂。
Wilate在接受手术的遗传性VWD患者中显示出有效的出血预防和治疗效果，且无临床显著的安全问题。
