Desmopressin testing in von Willebrand disease: Lowering the burden

Jessica M. Heijdra, Ferdows Atiq, in collaboration with the OPTI-CLOT Study Group, Wala Al Arashi, Quincy Kieboom, Esmee Wuijster, K. Meijer, Marieke J.H.A. Kruip, Frank W.G. Leebeek, Marjon H. Cnossen*

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Abstract

Background: Individuals with von Willebrand disease (VWD) require desmopressin testing because of interindividual response differences. However, testing is burdensome, while not all patients may need extensive testing. Objectives: To provide von Willebrand factor (VWF) cutoffs that predict desmopressin nonresponse and thereby identify individuals who do not need extensive testing in a retrospective cohort. We validated these cutoffs in a prospective cohort. Patients and Methods: We included 376 patients (Type 1 VWD with VWF activity [VWF:Act] <0.30 IU/ml: n = 112; with VWF:Act 0.30–0.50 IU/ml: n = 206; Type 2 VWD: n = 58; ages, 5–76 years) from January 2000 to July 2020. We collected VWF:Act and factor VIII activity (FVIII:C) at baseline and several time points after desmopressin (T1–T6). We defined response as VWF:Act and FVIII:C 0.50 IU/ml or greater at T1 and T4. We compared VWF:Act and FVIII:C distribution (historically lowest level, baseline, and T1) between responders and nonresponders and determined cutoffs discriminating between these groups. Results were validated in a group of 30 individuals. Results: All individuals with Type 1 VWD and Type 2 VWD, respectively, with baseline VWF:Act 0.34 IU/ml or greater or 0.28 IU/ml or greater were responders. In individuals with T1 VWF:Act ≥0.89 IU/ml (Type 1 VWD) or T1 VWF:Act 1.10 IU/ml or greater (Type 2 VWD), response remained at T4. Conclusion: Desmopressin testing is not needed when lowest historical VWF:Act is 0.30 IU/ml or greater. In patients with Type 1 VWD who require testing, measurements after T1 are often not needed. In patients with Type 2 VWD who require testing, we advise performing T1 and T4 measurements.

Original languageEnglish
Article numbere12784
JournalResearch and Practice in Thrombosis and Haemostasis
Volume6
Issue number6
DOIs
Publication statusPublished - Aug 2022

Bibliographical note

FUNDING INFORMATION
This investigator-initiated research project was supported
by the Innovation Fund of Health Care Insurance Companies
(Innovatiefonds Zorgverzekeraars) in the Netherlands (project number 3216). The funder is not involved in the design or performance of
the study and does not get any benefits from the study.

Publisher Copyright:
© 2022 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH).

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