Influence of blood group and von Willebrand factor on population pharmacokinetics and dose individualization of recombinant factor VIII in Taiwanese patients with haemophilia A

Abstract

Introduction: The large interpatient variability in the pharmacokinetic (PK) parameters of recombinant Factor VIII (rFVIII) observed in haemophilia A hinders efficient and cost-beneficial prophylactic regimen initiation. Identification of factors influencing the PK of rFVIII may shed more light on personalised treatment. Aim: This study aimed to develop a population PK model in the Taiwanese haemophilia A and evaluate the current national health insurance (NHI) reimbursement guidelines of Taiwan for haemophilia treatment. Methods: A population PK analysis was established based on 69 Taiwanese with moderate or severe haemophilia A. A nonlinear mixed-effects modelling (NONMEM ® ) was 230

Correlation Analysis of DNA Methylation in the von Willebrand Factor Promoter Region and the Risk of Unexplained Recurrent Hemophilia: Systematic Review and Meta-Analysis

“…The vWF gene is located at the top of the short arm of chromosome 12 (12p13.3), and the cDNA of vWF generates a primary transcript of 2813 amino acids containing a 22-amino acid signal peptide, a 741-amino acid propeptide (vWFAg: II), and a mature unit of 2050 amino acids. The types of mutations in vWF genes include insertion or deletion mutations, nonsense mutations, splice site mutations, missense mutations, and promoter mutations, with missense mutations being the most common [ 8 ]. The main pathogenesis of vWF is vWF gene mutation, and vWF gene detection can help vWF typing and differential diagnosis.…”