# 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

## TL;DR

A study shows that a specific von Willebrand factor concentrate is safe and effective for treating children with von Willebrand disease, including those with severe cases.

## Contribution

The study provides new evidence on the safety and efficacy of an FVIII-poor von Willebrand factor concentrate in young pediatric patients.

## Key findings

- The concentrate was rated excellent or good for all major bleeds or surgeries in children.
- Breakthrough bleeding occurred in only 1.6% of infusions for children on long-term prophylaxis.
- No safety concerns were observed in the treatment of children with von Willebrand disease.

## Abstract

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.

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

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.

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), including 30% with Type 3 VWD. Children received pdVWF for 16 major bleeds, 138 minor bleeds, 7 major surgeries, 8 minor surgeries and 12 invasive procedures. Efficacy was rated excellent or good in 100% of major bleeds or surgeries. The dose per infusion was approximately 50 IU/kg. By age group, median doses per infusion varied from 49 to 73 IU/kg for patients <6 years and from 46 to 59 IU/kg for patients 6–11 years, according to clinical situation. FVIII coadministration/correction was more frequent in Type 3 VWD, regardless of patient's age. Of five children receiving long‐term prophylaxis, breakthrough bleeding occurred in 1.6% of infusions and median annualised bleeding rate was 0.8. No safety concerns were raised.

This analysis enlarges clinical experience of an FVIII‐poor pdVWF in the paediatric population. This concentrate offers safe and effective treatment regardless of VWD severity.