Post Authorization Rixubis Study (PARIXS)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD04) coagulation factor IX

  coagulation factor IX
• Medical condition to be studied:
  Haemophilia

Population studied

• Short description of the study population:
  All patients with congenital hemophilia B receiving RIXUBIS in routine clinical practice.
• Age groups:
  • Paediatric Population (< 18 years)

    • Neonate

      • Term newborn infants (0 – 27 days)
    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Congenital haemophilia B patients
• Estimated number of subjects:
  80

Study design details

• Main study objective:
  To assess hemostatic effectiveness in the prevention of bleeding events in subjects with hemophilia Breceiving rFIX (RIXUBIS) using any therapeutic regimen in routine clinical practice.
• Outcomes:
  To assess hemostatic effectiveness in the prevention of bleeding events in subjects with hemophilia Breceiving rFIX (RIXUBIS) using any therapeutic regimen in routine clinical practice. To describe the safety and immunogenicity in subjects with hemophilia B receiving rFIX(RIXUBIS).To describe joint health outcomes in subjects receiving rFIX(RIXUBIS).Health-Related Quality of Life (HR-QoL) objectives:To describe acute and chronic pain associated with hemophilia, physical activity, hemophilia related comorbidity and healthcare recource use in subjects receiving rFIX (RIXUBIS)
• Data analysis plan:
  Descriptive statistics of all endpoints will include specifically but not exclusively,arithmetic mean, medians, standard deviations, minimum, maximum, proportions,frequency counts, 25th and 75th percentiles, and 95% confidence intervals of select pointestimates. Figures will be prepared to illustrate the patterns of data over time whereappropriate. The number of subjects included in each analysis set will be reported.