Registry of Pediatric Patients Treated with Vedrop® (Tocofersolan) in Europe for Vitamin E Deficiency due to Digestive Malabsorption in Congenital or Hereditary Chronic Cholestasis

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

  Effectiveness study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  VEDROP
• Medical condition to be studied:
  Vitamin E deficiency

Population studied

• Short description of the study population:
  Pediatric patients suffering from vitamin E deficiency due to chronic or hereditary chronic cholestasis leading to digestive malabsorption.
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Vitamin E deficient patients
• Estimated number of subjects:
  500

Study design details

• Main study objective:
  To collect data about the demographic profile of patients, the use of Vedrop® and its efficacy and safety profile.
• Data analysis plan:
  All data will be analyzed in a descriptive manner, no formal hypotheses will be tested. Continuous variables will be summarised with descriptive statistics and categorical variables will be displayed in frequency tables.