Key Drivers of Coagulation Factor Use in Von Willebrand Disease During Hospitalization: An Overview of the French BERHLINGO Cohort

Key Drivers of Coagulation Factor Use in Von Willebrand Disease During Hospitalization: An Overview of the French BERHLINGO Cohort

Background Von Willebrand disease (VWD) is the most common inherited bleeding disorder. However, studies of hospitalisation patterns with replacement treatment are scarce. Objectives The aim of this study was to investigate the current therapeutic management of VWD and determine the key drivers of c...

Full description

Saved in:
Bibliographic Details
Published in:Clinical drug investigation Vol. 44; no. 1; pp. 35 - 49
Main Authors: Horvais, Valérie, Beurrier, Philippe, Cussac, Vincent, Pan-Petesch, Brigitte, Schirr-Bonnans, Solène, Rose, Johann, Bayart, Sophie, Ternisien, Catherine, Fouassier, Marc, Sigaud, Marianne, Babuty, Antoine, Drillaud, Nicolas, Guillet, Benoît, Trossaërt, Marc
Format: Journal Article
Language:English
Published: Cham Springer International Publishing 01-01-2024
Springer Nature B.V
Springer Verlag
Series:Clinical Drug Investigation
Subjects:
Antigens
Disease
Gender
Health care
Hospitalization
Hospitals
Internal Medicine
Life Sciences
Medicine
Medicine & Public Health
NCT
NCT03875924
Original Research Article
Patients
Pediatrics
Pharmacology/Toxicology
Pharmacotherapy
Software
Variance analysis
France
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:Background Von Willebrand disease (VWD) is the most common inherited bleeding disorder. However, studies of hospitalisation patterns with replacement treatment are scarce. Objectives The aim of this study was to investigate the current therapeutic management of VWD and determine the key drivers of coagulation factor uses in patients during hospitalisation. Methods Hopscotch-WILL was a multi-centric retrospective study conducted over a 48-month period in any patients with VWD. The data were collected from the BERHLINGO Research Database and the French Hospital database. Results A total of 988 patients were included; 153 patients (15%) were hospitalised during 293 stays requiring treatment with von Willebrand factor (VWF) concentrates—pure or in association with Factor VIII (FVIII). Their median basal concentrations of VWF and FVIII were significantly lower than in untreated patients: VWF antigen < 30 IU/dL, VWF activity < 20 IU/dL and FVIII:C < 40 IU/dL. The median (interquartile range) concentrate consumption was similar between highly purified VWF or VWF combined with FVIII (72 [110] vs 57 [89] IU/kg/stay, p = 0.154). The use of VWF was highly heterogeneous by VWD type; type 3 had a particularly high impact on VWF consumption in non-surgical situations. The main admissions were for ear/nose/throat, hepato-gastroenterology, and trauma/orthopaedic conditions, besides gynaecological-obstetric causes in women. Conclusions The use of VWF concentrates is mostly influenced by low basal levels of VWF and FVIII, but also by VWD type or the cause for hospitalisation. These results could inform future studies of newly released recombinant VWF.
Bibliography:ObjectType-Article-1
SourceType-Scholarly Journals-1
ObjectType-Feature-2
content type line 23
ISSN:1173-2563
1179-1918
DOI:10.1007/s40261-023-01323-1