Cost-effectiveness of prophylaxis with recombinant vs plasma-derived VWF in severe von Willebrand disease in the United States

Christina Waldron; Satoko Ito; Daniel Wang; Cecily Allen; Giri Viswanathan; Robert D Bona; Adam Cuker; George Goshua

doi:10.1182/blood.2024024209

Cost-effectiveness of prophylaxis with recombinant vs plasma-derived VWF in severe von Willebrand disease in the United States

Blood. 2024 May 30;143(22):2332-2335. doi: 10.1182/blood.2024024209.

Authors

Christina Waldron¹, Satoko Ito², Daniel Wang¹, Cecily Allen³, Giri Viswanathan⁴, Robert D Bona², Adam Cuker^{5

6}, George Goshua^{2

7}

Affiliations

¹ Yale School of Medicine, New Haven, CT.
² Section of Hematology, Department of Internal Medicine, Yale School of Medicine, New Haven, CT.
³ Division of Hematology, Department of Medicine, Johns Hopkins Hospital, Baltimore, MD.
⁴ Yale College, New Haven, CT.
⁵ Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
⁶ Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
⁷ Center for Outcomes Research and Evaluation, Yale New Haven Hospital, New Haven, CT.

PMID: 38635764
DOI: 10.1182/blood.2024024209

Abstract

We evaluated the cost-effectiveness of prophylaxis with recombinant von Willebrand factor (rVWF) vs with plasma-derived von Willebrand factor (pdVWF) for patients with severe Von Willebrand disease. We found that rVWF is a cost-saving factor replacement compared with pdVWF across all willingness-to-pay thresholds in the United States.

Publication types

Comparative Study
Research Support, Non-U.S. Gov't

MeSH terms

Cost-Benefit Analysis*
Female
Humans
Male
Recombinant Proteins* / economics
Recombinant Proteins* / therapeutic use
United States
von Willebrand Diseases* / economics
von Willebrand Factor* / therapeutic use

Substances

von Willebrand Factor
Recombinant Proteins

Grants and funding

T35 HL007649/HL/NHLBI NIH HHS/United States