Recombinant von Willebrand factor for von Willebrand disease: mechanism of action and clinical application.

Von Willebrand disease (VWD) is an inherited bleeding disorder resulting from a deficiency in von Willebrand factor (VWF), either quantitative or qualitative. Recombinant VWF (rVWF) presents a novel therapeutic option for patients with VWD. Produced in Chinese hamster ovary cells, rVWF is free of animal or human plasma proteins, thus eliminating the risk of pathogen transmission. It preserves the full range of VWF multimers, including ultra-large multimers, which are essential for hemostasis. Research indicates that rVWF demonstrates superior pharmacokinetics and pharmacodynamics compared to plasma-derived VWF, offering a longer terminal half-life, enhanced platelet adhesion and aggregation, and more robust factor VIII stabilization. These properties contribute to rVWF's increased hemostatic efficacy in managing bleeding episodes and perioperative surgical bleeding in adults and children with VWD, as well as the routine prophylaxis for adults to reduce the frequency of bleeding episodes. Furthermore, rVWF is well-tolerated with a low thrombotic risk, making it a promising treatment option and addressing a significant clinical need globally.