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.
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 VWDpatients 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 VWDchildren 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.
Authors: C Rae; W Furlong; J Horsman; E Pullenayegum; C Demers; J St-Louis; D Lillicrap; R Barr Journal: Haemophilia Date: 2012-09-21 Impact factor: 4.287
Authors: T C Abshire; A B Federici; M T Alvárez; J Bowen; M D Carcao; J Cox Gill; N S Key; P A Kouides; K Kurnik; A E Lail; F W G Leebeek; M Makris; P M Mannucci; R Winikoff; E Berntorp Journal: Haemophilia Date: 2012-07-23 Impact factor: 4.287
Authors: Ashley E Neal; Christian Stopp; David Wypij; David C Bellinger; Carolyn Dunbar-Masterson; David R DeMaso; Jane W Newburger Journal: J Pediatr Date: 2014-10-28 Impact factor: 4.406
Authors: F Atiq; J L Saes; M C Punt; K P M van Galen; R E G Schutgens; K Meijer; M H Cnossen; B A P Laros-Van Gorkom; M Peters; L Nieuwenhuizen; M J H A Kruip; J de Meris; J G van der Bom; F J M van der Meer; K Fijnvandraat; I C Kruis; W L van Heerde; H C J Eikenboom; Frank W G Leebeek; S E M Schols Journal: EClinicalMedicine Date: 2021-01-29