Management of von Willebrand Disease With a Factor VIII-Poor von Willebrand Factor Concentrate: Results From the Paediatric Cohort of a Prospective Observational Post-Marketing Study.

ABSTRACT:

Introduction

Although clinical experience of a triple-secured, plasma-derived, von Willebrand factor (pdVWF), almost devoid of Factor VIII (FVIII) in adults with von Willebrand disease (VWD), is widely reported, its use in children is less documented.

Aim

To explore the safety and efficacy of this concentrate in real-life, in children <12 years old.

Methods

Data from 30 paediatric patients enrolled in a prospective, 3-year observational, post-marketing study in France were analysed. Efficacy and safety were assessed in two cohorts: 0-6 and 6-11 years old.

Results

The population included 14 children <6 years of age and 16 children aged 6-11 years. Most patients (80%) were severely affected (von Willebrand factor ristocetin cofactor activity [VWF:RCo] ≤ 15 IU/dL)

SUBMITTER: Goudemand J 

PROVIDER: S-EPMC12612368 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature