Université Paris-Saclay, INSERM, Hémostase Inflammation Thrombose HITH U1176, Le Kremlin-Bicêtre, France.
Department of Medicine, Queen's University, Kingston, ON, Canada.
Blood Adv. 2023 Dec 26;7(24):7501-7505. doi: 10.1182/bloodadvances.2023010716.
Innovation in therapies for patients with von Willebrand disease (VWD) has lagged far behind that for hemophilia, creating inequity in the bleeding disorder community. Although currently existing treatments of antifibrinolytics, desmopressin, and plasma-derived von Willebrand factor replacement are considered effective, multiple studies report poor quality of life in patients with VWD, especially those with heavy menstrual bleeding (HMB). This disconnect underscores the need for novel therapies that are safe and effective and that consider a patient's specific contraceptive and reproductive needs. Recombinant von Willebrand factor is the most recent new therapy for VWD; the data specific to women are reviewed. We also present emerging data on emicizumab for the treatment of VWD, BT200 (rondoraptivon pegol), generalized hemostatic therapies (VGA039 and HMB-011), as well as treatments based on nanotechnology (platelet-inspired nanoparticles and KB-V13A12). We are optimistic as we move toward pivotal clinical trials for these elegant and innovative treatments.
治疗血管性血友病 (VWD) 的创新疗法远远落后于血友病，这在出血性疾病患者群体中造成了不平等。尽管目前存在的抗纤维蛋白溶解剂、去氨加压素和血浆源性血管性血友病因子替代疗法被认为是有效的，但多项研究报告称 VWD 患者的生活质量较差，尤其是那些月经过多（HMB）的患者。这种脱节凸显了需要新型安全有效的治疗方法，同时考虑到患者特定的避孕和生殖需求。重组血管性血友病因子是治疗 VWD 的最新新型疗法；本文回顾了专门针对女性的相关数据。我们还介绍了emicizumab 治疗 VWD 的新数据，以及 BT200（rondoraptivon pegol）、全身性止血疗法（VGA039 和 HMB-011），以及基于纳米技术的治疗方法（血小板启发型纳米颗粒和 KB-V13A12）。我们对这些优雅创新的治疗方法即将进行关键性临床试验感到乐观。
