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

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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.

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Authors and Affiliations

  1. Specialty Pharmacy Services, Atrium Health, 4400 Golf Acres Drive, Charlotte, NC, 28208, USA

    Thuy Tran & Justin Arnall

  2. Levine Cancer Institute, Atrium Health, 100 Medical Park Drive, Concord, NC, 28025, USA

    Donald C. Moore

  3. Department of Pharmacy, University of Virginia Health System, PO Box 800674, Charlottesville, VA, 22908, USA

    Leslie Ward

  4. Department of Pharmacy, University of Colorado Hospital, 1665 Aurora Court, Aurora, CO, 80045, USA

    Surabhi Palkimas

  5. Division of Hematology/Oncology, University of Virginia Health System, PO Box 800716, Charlottesville, VA, 22908, USA

    Louise Man

Authors
  1. Thuy Tran
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  2. Justin Arnall
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  3. Donald C. Moore
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  4. Leslie Ward
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  5. Surabhi Palkimas
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  6. Louise Man
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Correspondence to Thuy Tran.

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Tran, T., Arnall, J., Moore, D.C. et al. Vonicog alfa for the management of von Willebrand disease: a comprehensive review and single-center experience. J Thromb Thrombolysis 49, 431–440 (2020). https://doi.org/10.1007/s11239-019-02018-1

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  • DOI: https://doi.org/10.1007/s11239-019-02018-1

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