Galvanin, Federico;
Lee, Chung Fun;
Yuxuan, Yang;
(2025)
On the development of pharmacokinetic models for the characterisation and diagnosis of von Willebrand disease.
Physical Sciences Reviews
(In press).
![[thumbnail of Galvanin_articolo_final_rev_unmarked.pdf]](https://discovery.ucl.ac.uk/style/images/fileicons/text.png) |
Text
Galvanin_articolo_final_rev_unmarked.pdf - Accepted Version Access restricted to UCL open access staff until 22 September 2025. Download (859kB) |
Abstract
Von Willebrand disease (VWD) is a metabolic disease characterised by a qualitative and/or quantitative deficiency of von Willebrand factor (VWF) a multimeric glycoprotein that mediates platelet adhesion in haemostatic processes. Pharmacokinetic (PK) models have been developed and applied to characterize VWD metabolic pathways, and to achieve a model-based diagnosis based on clinical data. However, current PK models cannot be calibrated from infusion tests data, and their calibration requires stressful 24-hours long tests to be carried out on subjects to achieve a satisfactory estimation of the individual haemostatic parameters. The objectives of this review chapter are the following: i) to provide a review on physiological modelling of VWD starting from the analysis of basic VWF mechanisms in the body; ii) to describe methods and modelling tools used to provide a model-based diagnosis of VWD and, consequently, a classification of complex VWD types; iii) to illustrate how model-based design of experiments (MBDoE) techniques can be applied to maximise the information that can be obtained from advanced clinical tests (DDAVP) but also from infusion tests used in VWD treatment where blood analogues are administered through single or multiple injections. Results show how PK models calibrated from clinical data can be used to estimate key haemostatic parameters in the diagnosis of the disease. Promising results on the application of MBDoE to design infusion tests show that the overall duration of clinical tests for the identification of haemostatic parameters can significantly be reduced from 24 hours to 2.5 hours, with the potential to increase the acquired test information if multiple infusions can be managed.
| Type: | Article |
|---|---|
| Title: | On the development of pharmacokinetic models for the characterisation and diagnosis of von Willebrand disease |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > UCL BEAMS UCL > Provost and Vice Provost Offices > UCL BEAMS > Faculty of Engineering Science > Dept of Chemical Engineering |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10206425 |
Loading...
Loading...Loading...Loading...Archive Staff Only
 |
View Item |
