Vonicog alfa for the management of von Willebrand disease: a comprehensive review and single-center experience

Authors: Tran, T., Arnall, J., Moore, D. C., Ward, L., Palkimas, S., and Man, L.

Published: Journal of Thrombosis and Thrombolysis;

Affiliations: Atrium Hlth, Specialty Pharm Serv, 4400 Golf Acres Dr, Charlotte, NC 28208 USA ; Atrium Hlth, Levine Canc Inst, 100 Med Pk Dr, Concord, NC 28025 USA ; Univ Virginia Hlth Syst, Dept Pharm, POB 800674, Charlottesville, VA 22908 USA ; Univ Colorado Hosp, Dept Pharm, 1665 Aurora Court, Aurora, CO 80045 USA ; Univ Virginia Hlth Syst, Div Hematol Oncol, POB 800716, Charlottesville, VA 22908 USA.

Abstract: Von Willebrand Disease (VWD) is characterized by a qualitative or quantitative defect in von Willebrand factor that results in prolonged bleeding due to the inability to form a stable platelet plug. VWD is the most common inherited bleeding disorder. The mainstay of treatment of VWD includes desmopressin; with plasma-derived von Willebrand Factor concentrates reserved for patients with severe VWD or those with desmopressin intolerability. Although efficacious, plasma-derived factor concentrates can have risks associated with them including minimal risk of pathogenic transmission, potential to contain extraneous plasma proteins and cause severe allergic reactions, and a supply limited by plasma donor availability. Vonicog alfa is a recombinant von Willebrand Factor product. Two phase III trials evaluated the safety and efficacy of vonicog alfa in preventing perioperative bleeding and treating acute bleeding in patients with VWD. Beyond the clinical trials, there has been little real-world experience published regarding experiences with this medication. This article comprehensively reviews the efficacy, safety, pharmacokinetics, and pharmacodynamics of vonicog alfa. These points will be discussed using institutional experiential data from the University of Virginia (UVA) Health System in relation to the clinical studies. The goal of this review article is to offer insights to clinical directions, discuss operational challenges, and offer guidance for future studies and formulary decisions.