Announcement
Thank you for attending THSNA 2026. The virtual meeting is now closed.
Thank you for attending THSNA 2026. The virtual meeting is now closed.
Presentation Details
| Investigating the Effect of Population Pharmacokinetic-Guided pdVWF/FVIII (1:1) Prophylaxis for Severe VWD in a Real-World Setting: The PopPK-WILPROPHY Study Robert F.Sidonio, Jr.1, Ron A.A.Mathôt2. 1Professor of Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.2Department of Hospital Pharmacy & Clinical Pharmacology, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands |
Abstract
Background: Prophylaxis with a von Willebrand factor (VWF)-containing concentrate is recommended for persons with von Willebrand disease (VWD) experiencing severe and frequent bleeds. The efficacy and safety of prophylaxis with a plasma-derived VWF/factor VIII concentrate (pdVWF/FVIII 1:1, wilate®) has been demonstrated across ages and VWD types. Tailoring of prophylaxis to individual patients has been shown to increase the net clinical benefit of treatments in persons with hemophilia and is the standard of care. Despite decades of experience, insufficient research has been conducted into pharmacokinetic (PK)-guided prophylaxis in persons with VWD and personalized prophylaxis has not yet been implemented in clinical practice. Objectives: To investigate the impact of pharmacokinetic (PK)-guided prophylaxis with a pdVWF/FVIII 1:1 concentrate on clinical outcomes in persons with clinically severe VWD during routine clinical practice. Methods: PopPK-WILPROPHY will be a prospective, observational, single-arm, multicenter study enrolling 70 participants with severe VWD of any age (in accordance with locally approved prescribing information) who intend to start pdVWF/FVIII 1:1 prophylaxis. Participants will either be a) receiving any VWF concentrate on-demand, b) receiving prophylaxis with another VWF treatment or c) already receiving pdVWF/FVIII 1:1 prophylaxis. Participants will be treated in accordance with standard clinical practice at the discretion of the treating physician. Following a screening visit, participants will receive pdVWF/FVIII 1:1 prophylaxis for 6 months using standard dosing. During this period, participants will undergo a PK assessment, and the data will be analyzed using a population PK (PopPK) model developed by the Amsterdam University Medical Centre in the Netherlands. The model will be used to propose tailored dosing regimens for subsequent PopPK-guided prophylaxis over 12 months, optimized according to patient characteristics. The adjusted regimen will be applied by the treating physician, taking into account each participant’s individual clinical condition and activity level. Total study duration per patient will be 18 months (Figure 1). The primary endpoint will be the annualized bleeding rate (ABR) during PopPK-guided prophylaxis compared with the ABR during standard dosing. Secondary endpoints will include quality of life, efficacy in treatment of bleeding episodes, efficacy of surgical prophylaxis, pdVWF/FVIII 1:1 consumption, and adverse events, including those of special interest (e.g. thrombotic events, inhibitor development) (Figure 2). Results: This study is planned to start in Q2 2026, enrolling participants across approximately 20 centers in the USA, Canada, Europe, Asia and Latin America. Conclusions: The PopPK-WILPROPHY study will provide important evidence on the clinical utility of PopPK-guided prophylaxis with pdVWF/FVIII 1:1, supporting future implementation of personalized treatment strategies for individuals with severe VWD.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.