Is pharmacokinetic-guided dosing of desmopressin and von Willebrand factor-containing concentrates in individuals with von Willebrand disease or low von Willebrand factor reliable and feasible? A protocol for a multicentre, non-randomised, open label cohort trial, the OPTI-CLOT: to WiN study

What Was Tested

This clinical study paper looks at Desmopressin.

Primary aim of this study is to assess the predictive performance (the difference between predicted and measured von VWF activity and FVIII levels) of Bayesian forecasting using the developed population PK models in four different situations: (A) desmopressin testing (n≥30); (B) medical procedures (n=70; 30 receiving desmopressin, 30 receiving VWF-containing concentrate and 10 receiving a combination of both); (C) bleeding episodes (n=20; 10 receiving desmopressin and 10 receiving VWF-containing concentrate) and (D) prophylaxis with a VWF-containing concentrate (n=3 to 5).

Why It Was Studied

Von Willebrand disease (VWD) is a bleeding disorder, caused by a deficiency or defect of von Willebrand factor (VWF).

However, in many cases these factor levels are outside the targeted range.

How the Study Was Run

In design terms this was a multicenter study.

More than one site took part, so the participants came from several places.

What Was Measured

However, in many cases these factor levels are outside the targeted range.

These models enable calculation of individual PK parameters by Bayesian analysis, based on an individual desmopressin test or PK profile with a VWF-containing concentrate.

PubMed files the paper under topics including Bayes Theorem, Deamino Arginine Vasopressin, Reproducibility of Results, von Willebrand Diseases and von Willebrand Factor.

Author keywords: bleeding disorders & coagulopathies, clinical trials and protocols & guidelines.

What This Means in Plain Language

The paper is a multicenter study of Desmopressin.

The publication is from 2022 (BMJ open), a journal based in England.

More context: the Desmopressin compound page, all human trials.