New and Emerging Therapies for Women, Girls and People with the Potential to Menstruate with VWD.

Innovation in therapies for von Willebrand disease (VWD) patients has lagged far behind hemophilia, creating inequity in the bleeding disorder community. Although currently existing treatments of antifibrinolytics, desmopressin and plasma-derived von Willebrand factor (pdVWF) replacement are considered effective, multiple studies report poor quality of life in VWD patients, 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 VWF (rVWF) is the most recent new therapy for VWD; the data specific to women is 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 towards pivotal clinical trials for these elegant and innovative treatments.