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

Authors: Goudemand, J; Borel-Derlon, A; Claeyssens, S; Chambost, H; Mourey, G; Harroche, A; Meunier, S; Henriet, C; Leroi, T; Susen, S; Repesse, Y

Affiliations: Department of Haemostasis and Transfusion, Lille University Hospital, Lille, France. Haemophilia Treatment Centre, University Hospital of Caen, Caen, France. Haemophilia Treatment Center, Purpan Hospital, Toulouse, France. AP-HM, Department of Paediatric Haematology Oncology, Institut National De La Santé Et De La Recherche médicale, Institut national De La Recherche Agronomique, Centre Recherche En CardioVasculaire et Nutrition, Children Hospital La Timone & Aix Marseille University, Marseille, France. Medical Biology Laboratory, Biological Haemostasis Department, CHU Besançon, Besançon, France. CHU Besançon, SINERGIES (UR 4662), Université Marie et Louis Pasteur, Besançon, France. AP-HP, Haemophilia Treatment Centre, Hospital Necker, Paris, France. Hospices Civils de Lyon, French Reference Center For Haemophilia, Clinical Haemostasis Unit Lyon, Louis Pradel Hospital, Bron, France. Clinical Development, Laboratoire Français Du Fractionnement et Des Biotechnologies (LFB), Les Ulis, France. Medical Operation France, Laboratoire Français Du Fractionnement et Des Biotechnologies (LFB), Les Ulis, France. Department of Haemostasis and Transfusion, Lille University Hospital, Lille, France. Haemophilia Treatment Centre, University Hospital of Caen, Caen, France.

Publication: Haemophilia; 2025. e70129

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 analyzed. 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), 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 annualized bleeding rate was 0.8. No safety concerns were raised. CONCLUSION 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.