Health-related quality of life in children with von Willebrand disease: results of the French real-life Willebrand Study Health-related Quality of Life study

Abstract

Background: Hemorrhagic episodes may have physical or psychological effects on children with von Willebrand disease (VWD) and on their families. These effects can be measured by health-related quality of life (HRQoL).

Objectives: The Willebrand Study Health-related Quality of Life study aimed at filing this knowledge gap, using generic or disease-specific patient-reported outcome (PRO) questionnaires in France.

Methods: This prospective observational study included 117 children (<18 years) with all VWD types (type 1 with basal von Willebrand factor [VWF]:Ag < 30%; HRQoL [DISABKIDS, VWD-QoL]; and treatment satisfaction, and family burden) were assessed at baseline and 24 months via child and/or parent questionnaires.

Results: The DISABKIDS questionnaire revealed age-specific patterns: preschoolers (4-7 years) showed predominant physical limitations, while older children (8-17 years) faced independence challenges. The VWD-QoL questionnaire identified early and persistent impacts on peer/family relationships. Parent-child concordance was observed in total DISABKIDS/VWD-QoL scores, although adolescents were more optimistic than parents in some domains, while children aged 4-7 years reported slightly poorer physical abilities. Gender differences emerged, with boys (8-17 years) reporting more school difficulties and younger girls (4-7 years) perceiving greater social/sports restrictions. Disease severity (VWF:Ristocetin co-factor <15 IU/dL in types 1/2) significantly worsened overall HRQoL. Parents reported some dissatisfaction regarding treatment ease and burden. Parents also reported significant disease-related impacts on both family dynamics and personal well-being, particularly in type 3 VWD families.

Conclusion: HRQoL was reduced in French VWD children and their families secondary to physical, mental, and social health impacts, especially (but not only) in adolescents and in children with severe VWD types.

Keywords: child; global disease burden; observational study; quality of life; von Willebrand disease.