A model-based support for diagnosing von Willebrand disease

Von Willebrand disease (VWD) is the most common inherited bleeding disease and is caused by deficiency or dysfunction of a multimeric glycoprotein, namely the von Willebrand factor (VWF). The disease is present in numerous subtypes, making the diagnosis through the classic 24-h DDAVP sampling protocol a difficult task. In this simulation study, a new simplified pharmacokinetic model is proposed with the aims of demonstrating that a shorter DDAVP clinical test can be devised and that indices, such as clearance, total amount of VWF released and multimeric ratio at basal state can be exploited with multivariate classification methods in order to help practitioners to reach a correct diagnosis.