Federici A B, Baudo F, Caracciolo C, Mancuso G, Mazzucconi M G, Musso R, Schinco P C, Targhetta R, Mannuccio Mannucci P
Department of Internal Medicine, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ospedale Maggiore, University of Milan, Italy.
Haemophilia. 2002 Nov;8(6):761-7. doi: 10.1046/j.1365-2516.2002.00688.x.
The goal of therapy in patients with von Willebrand disease (vWD) is to correct the dual defect of primary haemostasis and intrinsic coagulation reflected by low levels of von Willebrand factor (vWF) and factor VIII coagulant activity (FVIII:C). Factor VIII/von Willebrand factor (FVIII/vWF) concentrates are currently the treatment of choice in vWD patients unresponsive to desmopressin (DDAVP). However, only few studies on their clinical use are available so far. The main objective of this study was to retrospectively evaluate the clinical efficacy of a highly purified, doubly virus-inactivated FVIII/vWF concentrate with a high content of FVIII/vWF (Fanhdi). Twenty-two patients with congenital vWD have been treated from 1999 to 2001 at eight specialized centres belonging to the Italian Association of Hemophilia Centers (AICE). Ten males and 12 females, median age 28.5 years, range 5-70 years) had type 3 vWD (six cases), DDAVP-unresponsive type 1 (nine cases) and type 2B (seven cases). The study drug was given to stop or prevent 12 bleeding episodes or to prevent excessive bleeding during 14 surgical or invasive procedures. Overall, replacement therapy with the concentrate showed an excellent to good clinical efficacy in 92% of bleeding episodes and in 93% of surgical procedures. No adverse events occurred during 1,601 infusions, accounting for a total of 304,500 IU of FVIII:C administered. These results confirm the efficacy and safety of this concentrate in the management of bleeding episodes and in the prevention of excessive bleeding during major and minor surgery.
血管性血友病（vWD）患者的治疗目标是纠正因血管性血友病因子（vWF）水平低和凝血因子 VIII 促凝活性（FVIII:C）低所反映的原发性止血和内源性凝血双重缺陷。目前，VIII 因子/血管性血友病因子（FVIII/vWF）浓缩物是对去氨加压素（DDAVP）无反应的 vWD 患者的首选治疗方法。然而，迄今为止，关于其临床应用的研究很少。本研究的主要目的是回顾性评估一种高纯度、双重病毒灭活的高含量 FVIII/vWF 浓缩物（Fanhdi）的临床疗效。1999 年至 2001 年期间，意大利血友病中心协会（AICE）下属的八个专业中心对 22 例先天性 vWD 患者进行了治疗。其中 10 名男性和 12 名女性，中位年龄 28.5 岁，年龄范围 5 - 70 岁，患有 3 型 vWD（6 例）、对 DDAVP 无反应的 1 型（9 例）和 2B 型（7 例）。使用研究药物来停止或预防 12 次出血发作，或在 14 次手术或侵入性操作期间预防过度出血。总体而言，浓缩物替代疗法在 92%的出血发作和 93%的手术中显示出优异至良好的临床疗效。在 1601 次输注期间未发生不良事件，共输注了 304,500 IU 的 FVIII:C。这些结果证实了该浓缩物在治疗出血发作以及预防大、小手术期间过度出血方面的有效性和安全性。
