Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.
Blood Research Institute, Versiti Blood Center of Wisconsin, Milwaukee, WI, USA.
Clin Appl Thromb Hemost. 2019 Jan-Dec;25:1076029619873976. doi: 10.1177/1076029619873976.
Patients with von Willebrand disease (VWD) often require treatment with supplemental von Willebrand factor (VWF) prior to procedures or to treat bleeding. Commercial VWF concentrates and more recently recombinant human VWF (rVWF) have replaced cryoprecipitate as the mainstay of therapy. In comparison with cryoprecipitate, the VWF content and multimer distribution under current manufacturing processes of these commercial products has not been reported. We measured the factor VIII (FVIII:C), VWF antigen (VWF:Ag), VWF collagen-binding activity (VWF:CB), VWF platelet-binding activity by GPIbM enzyme-linked immunosorbent assay (VWF:GPIbM), and percentage of high-molecular-weight (HMWM) VWF in 3 pools of group A and O cryoprecipitate, 3 vials of VWF concentrate (Humate-P), and 1 lot of rVWF (Vonvendi). We found that both group O and group A cryoprecipitate have significantly higher ratios of VWF:GPIbM activity and FVIII:C activity relative to VWF:Ag and have better preservation of HMWM than Humate-P. Although not compared statistically, rVWF appears to have more HMWM VWF and a higher ratio of VWF:GPIbM to VWF:Ag than Humate-P and cryoprecipitate. The estimated acquisition cost for our hospital for treating one major bleeding episode was more than 4-fold higher with Humate-P and 7- to 10-fold higher with rVWF than with cryoprecipitate.
患有血管性血友病(VWD)的患者在进行手术前或治疗出血时,通常需要额外补充血管性血友病因子(VWF)。商业 VWF 浓缩物和最近的重组人 VWF(rVWF)已经取代了冷沉淀作为主要的治疗方法。与冷沉淀相比,这些商业产品的当前制造工艺下的 VWF 含量和多聚体分布尚未报道。我们测量了 3 组 A 型和 O 型冷沉淀、3 瓶 VWF 浓缩物(Humate-P)和 1 批 rVWF(Vonvendi)中的 VIII 因子(FVIII:C)、VWF 抗原(VWF:Ag)、VWF 胶原结合活性(VWF:CB)、GPIbM 酶联免疫吸附试验(VWF:GPIbM)的 VWF 血小板结合活性以及高分子量(HMWM)VWF 的百分比。我们发现,O 型和 A 型冷沉淀的 VWF:GPIbM 活性和 FVIII:C 活性与 VWF:Ag 的比值显著更高,并且 HMWM 的保存情况优于 Humate-P。尽管没有进行统计学比较,但 rVWF 似乎比 Humate-P 和冷沉淀具有更多的 HMWM VWF 和更高的 VWF:GPIbM 与 VWF:Ag 的比值。我们医院治疗一次大出血事件的估计获得成本,使用 Humate-P 比使用冷沉淀高出 4 倍以上,使用 rVWF 比使用冷沉淀高出 7-10 倍。