Background: Von Willebrand disease (VWD) is the most frequent inherited bleeding disorder. Whether VWD is associated with health-related quality of life (HR-QoL) in children is unknown. Objectives: This nationwide cross-sectional study measured HR-QoL in children with moderate or severe VWD. Our primary aim was to compare HR-QoL of VWD patients with that of reference populations. Additionally, we studied the impact of bleeding phenotype and VWD type on HR-QoL. Methods: HR-QoL was assessed with the Infant/Toddler QoL Questionnaire (0-5 years) and Child Health Questionnaire (6-15 years), and compared with reference population scores. Multivariate analysis was used to evaluate the influence of type of VWD and bleeding phenotype on HR-QoL scores. Results: Preschool children (0-5 years, n=46) with VWD had lower HR-QoL scores for general health perceptions and parental time than reference populations. School children (6-15 years, n=87) with VWD had lower scores for physical functioning, role functioning - emotional/behavioral, general health perceptions, and physical summary. Type of VWD was associated with HR-QoL in school children for bodily pain, general health perceptions, parental emotion, family activities, and physical summary. Scores of children with type 3 VWD were, on average, 15 points lower than those of the reference population on the above-mentioned scales. A more severe bleeding phenotype was associated with a lower score on 11/15 physical, emotional and social scales. Conclusion: HR-QoL is lower in VWD children than in reference populations, in particular in school children. The negative impact of VWD is sensitive to type of VWD and bleeding phenotype; as well as physical scales, emotional and social scales are affected.

Bleeding, Health-related quality of life, Pediatrics, Von Willebrand disease
dx.doi.org/10.1111/j.1538-7836.2010.04175.x, hdl.handle.net/1765/23850
Journal of Thrombosis and Haemostasis
Erasmus MC: University Medical Center Rotterdam

de Wee, E.M, Fijnvandraat, K, de Goede-Bolder, A, Mauser-Bunschoten, E.P, Eikenboom, J.C.J, Brons, P.P, … Leebeek, F.W.G. (2011). Impact of von Willebrand disease on health-related quality of life in a pediatric population. Journal of Thrombosis and Haemostasis, 9(3), 502–509. doi:10.1111/j.1538-7836.2010.04175.x