title: Impact of von Willebrand disease on health-related quality of life in a pediatric population.
abstract: 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.
  
  
  OBJECTIVE
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Hematology, Erasmus University Medical Center, Rotterdam, the Netherlands.
authors.forename: E M
authors.initials: EM
authors.lastname: de Wee
authors.forename: K
authors.initials: K
authors.lastname: Fijnvandraat
authors.forename: A
authors.initials: A
authors.lastname: de Goede-Bolder
authors.forename: E P
authors.initials: EP
authors.lastname: Mauser-Bunschoten
authors.forename: J C J
authors.initials: JC
authors.lastname: Eikenboom
authors.forename: P P
authors.initials: PP
authors.lastname: Brons
authors.forename: F J
authors.initials: FJ
authors.lastname: Smiers
authors.forename: R
authors.initials: R
authors.lastname: Tamminga
authors.forename: R
authors.initials: R
authors.lastname: Oostenbrink
authors.forename: H
authors.initials: H
authors.lastname: Raat
authors.forename: J G
authors.initials: JG
authors.lastname: van der Bom
authors.forename: F W G
authors.initials: FW
authors.lastname: Leebeek
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D003430:Cross-Sectional Studies; D004644:Emotions; D005260:Female; D006470:Hemorrhage / Q000097:blood; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D009426:Netherlands; D017063:Outcome Assessment, Health Care; D010641:Phenotype; D011788:Quality of Life; D012961:Sociology; D011795:Surveys and Questionnaires; D014842:von Willebrand Diseases / Q000097:blood / Q000145:classification / Q000503:physiopathology* / Q000523:psychology*