Etude de la qualité de vie et de l’impact médico-économique de la maladie de Willebrand en France (WiSH-QoL)

Study type

• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Other

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD10) von Willebrand factor

  von Willebrand factor

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  350

Study design details

• Main study objective:
  The main objective is the analysis of the quality of life of patients with Von Willebrand Disease Whatever their age, sex and phenotype.
• Outcomes:
  Analyse the quality of life of patients with Von Willebrand Disease using generic and Von Willebrand Disease-specific quality of life questionnaires. 1. Survey and identify therapeutic practices in Von Willebrand Disease 2. Evaluate the impact of treatment strategies on Von Willebrand 3. Investigate/analyse the impact of Von Willebrand Disease on the clinical status, treatments and complications in subgroups of the population
• Data analysis plan:
  Statistical analyses were mainly descriptive on each outcome. The parameters collected were described for all analyzable patients. The quality-of-life information was analyzed by "scoring" several dimensions of each questionnaire used for adults and for children and/or their parents: generic quality of life questionnaire, Von Willebrand Disease-specific questionnaire including treatment satisfaction and FABEL questionnaire about family impact due to chronic pathology. The quantitative variables were expressed as a mean ± standard deviation, median, quartiles and range, and the qualitative variables as a frequency table. The descriptive analysis was carried out in following sub-groups: age classes, phenotypes (type 1, type 2, type 3, type undetermined) and treatment strategy (on-demand, prophylaxis).