Auerswald G, Kreuz W
Center for Thrombosis- and Haemostasis, Prof.-Hess-Kinderklinik, Klinikum Bremen-Mitte, Bremen, Germany.
Haemophilia. 2008 Nov;14 Suppl 5:39-46. doi: 10.1111/j.1365-2516.2008.01850.x.
von Willebrand disease (VWD) is a heterogeneous bleeding disorder with symptoms in affected patients ranging from mild effects to potentially devastating haemorrhagic events. Desmopressin (DDAVP) and von Willebrand factor/factor VIII (VWF/FVIII) concentrates are the principal treatments. Haemate P/Humate-P is an intermediate-purity VWF/FVIII concentrate with extensive clinical experience in VWD. This concentrate has been shown to correct haemostatic defects of VWD, with efficacy ratings of good/excellent in nearly all patients treated for bleeding or surgical events. Haemate P/Humate-P has a high content of the high molecular weight (HMW) VWF multimer fraction, which has been shown to be very effective in achieving haemostasis. The HMW VWF multimer pattern in Haemate P/Humate-P is more similar to that of normal human plasma (94% for Haemate P/Humate-P vs. 100% for normal human plasma) than that of other VWF/FVIII concentrates and correlates with functional VWF activities including ristocetin cofactor activity (VWF:RCo) and collagen-binding activity. The recommended dosing of Haemate P/Humate-P is based preferentially on VWF:RCo activity, which is approximately twice that of FVIII:C (2.4:1). Haemate P/Humate-P has been shown to be safe; no serious adverse events or cases of thrombosis have been observed in clinical trials and no documented cases of viral transmission in nearly three decades of clinical use. While DDAVP is effective in a large proportion of VWD patients, it may not provide adequate haemostasis in all situations. In such cases, Haemate P/Humate-P is an effective replacement concentrate for all types of VWD in both adult and paediatric patients.
血管性血友病(VWD)是一种异质性出血性疾病,受影响患者的症状从轻微影响到潜在的毁灭性出血事件不等。去氨加压素(DDAVP)和血管性血友病因子/凝血因子VIII(VWF/FVIII)浓缩物是主要治疗方法。海莫莱士/人凝血因子复合物(Haemate P/Humate-P)是一种中等纯度的VWF/FVIII浓缩物,在VWD方面有广泛的临床经验。这种浓缩物已被证明可纠正VWD的止血缺陷,在几乎所有因出血或手术事件接受治疗的患者中,疗效评级为良好/优秀。海莫莱士/人凝血因子复合物含有高比例的高分子量(HMW)VWF多聚体部分,已被证明在实现止血方面非常有效。海莫莱士/人凝血因子复合物中的HMW VWF多聚体模式比其他VWF/FVIII浓缩物更类似于正常人血浆(海莫莱士/人凝血因子复合物为94%,正常人血浆为100%),并且与包括瑞斯托霉素辅因子活性(VWF:RCo)和胶原结合活性在内的功能性VWF活性相关。海莫莱士/人凝血因子复合物的推荐剂量优先基于VWF:RCo活性,其约为FVIII:C活性的两倍(2.4:1)。海莫莱士/人凝血因子复合物已被证明是安全的;在临床试验中未观察到严重不良事件或血栓形成病例,在近三十年的临床使用中也没有病毒传播的记录病例。虽然DDAVP在很大比例的VWD患者中有效,但在所有情况下可能无法提供足够的止血效果。在这种情况下,海莫莱士/人凝血因子复合物是成人和儿童患者所有类型VWD的有效替代浓缩物。