@article{85c4ee8776ab405cbaa07b0a4ce49e97,
title = "Desmopressin testing in von Willebrand disease: Lowering the burden",
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.",
keywords = "desmopressin, factor VIII, humans, von Willebrand disease, von Willebrand factor",
author = "Heijdra, {Jessica M.} and Ferdows Atiq and {Al Arashi}, Wala and Quincy Kieboom and Esmee Wuijster and Karina Meijer and Kruip, {Marieke J.H.A.} and Leebeek, {Frank W.G.} and Cnossen, {Marjon H.} and K. Fijnvandraat and Math{\^o}t, {R. A.A.} and S. Polinder and M. Coppens and Tamminga, {R. Y.J.} and K. Meijer and {Laros-van Gorkom}, {B. A.P.} and P. Brons and Schols, {S. E.M.} and {van der Meer}, {F. J.M.} and Eikenboom, {H. C.J.} and Schutgens, {R. E.G.} and K. Fischer and F. Heubel-Moenen and L. Nieuwenhuizen and P. Ypma and Driessens, {M. H.E.} and Zwaan, {C. M.} and {van Vliet}, I. and Collins, {P. W.} and R. Liesner and P. Chowdary and D. Keeling and J. Lock and Hazendonk, {H. C.A.M.} and {van Moort}, I. and T. Preijers and {de Jager}, {N. C.B.} and Goedhart, {M. C.H.J.} and Bukkems, {L. H.} and Cloesmeijer, {M. E.} and A. Janssen",
note = "Funding Information: JH received the CSL Behring‐professor Heimburger Award 2018. FA received the CSL‐Behring‐professor Heimburger Award 2018 and a travel grant from Sobi. KM received research grants from Bayer, Pfizer, and Sanquin; speaker fees from Aspen, Bayer, BMS, Boehringer Ingelheidm, and Sanquin; and consulting fees from Uniqure. MK received grants from governmental research institutes, such as the Dutch Research Institute (ZonMW/NWO), Dutch Thrombosis Foundation, and Innovation fund; unrestricted grants from Bayer, Pfizer, Daiichi Sankyo, Sobi, and Boehringer Ingelheim and speaker's fee from Bayer. FL received research support from CSL Behring and Shire/Takeda for performing the Willebrand in the Netherlands (WiN) study, and from uniQure and Sobi for other studies. He is a consultant for uniQure, Novo Nordisk, and Shire/Takeda, of which the fees go to the institution, and has received a travel grant from Sobi. He is also a DSMB member for a study by Roche. MC has received grants from governmental research institutes, such as the Dutch Research institute (NWO), ZonMW, Innovation fund, an NWO‐NWA grant, and unrestricted investigator‐initiated research grants as well as educational and travel funding from various companies over the years (Pfizer, Baxter/Baxalta/Shire, Bayer Schering Pharma, CSL Behring, Sobi Biogen, Novo Nordisk, Novartis, and Nordic Pharma), and has served as a member on steering boards of Roche, Bayer, and Octapharma. All grants, awards, and fees go to the institution. The other authors declare no potential conflicts of interest. Publisher Copyright: {\textcopyright} 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).",
year = "2022",
month = aug,
doi = "10.1002/rth2.12784",
language = "English",
volume = "6",
number = "6",
}