A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Severe Von Willebrand Disease (VWD) (ATHN 9: Severe VWD Natural History Study)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Other
• If ‘other’, further details on the scope of the study:
  To assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Von Willebrand's disease

Population studied

• Age groups:
  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:
  130

Study design details

• Main study objective:
  To assess the safety of various Von Willebrand Factor (VWF) regimens for different indications (on-demand, surgery and prophylaxis) in adult and pediatric participants with clinically severe congenital VWD.
• Outcomes:
  Safety will be measured by the number of reported events as defined by the European Haemophilia Safety Surveillance (EUHASS) program. -standardized diagnostic battery using an VWF assay, and genetic sequence analysis of VWF coding regions and adjacent non-coding regions.-establish a platform for sub-studies -evaluate the use of factor replacement as prophylaxis -describe bleeding events and annualized bleeding rate -describe effectiveness of VWD treatment as measured by:Health care utilization and Quality of Life
• Data analysis plan:
  Descriptive statistics will be calculated to analyze the primary and secondary outcomes. Most of the study outcome variables are discrete in nature, such as mortality, newly developed inhibitor, bleeding rate, etc. Some outcome measurements will be treated as continuous, like health-related quality of life. For each categorical variable, its frequency and percentage will be reported. In terms of a continuous measurement, its mean, median, standard deviation, interquartile range, minimum, and maximum values will be disclosed. During the course of the study, the Steering Committee will evaluate the appropriateness of various statistical approaches based on the amount and quality of data collected.