Privigen® use and haemolytic anaemia in adults and children and the Privigen® safety profile in children with CIDP - an observational hospital-based cohort study in the US (Privigen PASS)

Study type

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medical condition to be studied:
  Chronic inflammatory demyelinating polyradiculoneuropathy

  Demyelinating polyneuropathy

  Primary immunodeficiency syndrome

  Congenital hypogammaglobulinaemia

  Guillain-Barre syndrome

  Kawasaki's disease

  Secondary immunodeficiency

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)
• Special population of interest:
  Immunocompromised
• Estimated number of subjects:
  25000

Study design details

• Main study objective:
  Risk assessment of known and potential risks with use of Privigen®
• Outcomes:
  Incidence of haemolytic anemia, occurrence of aseptic meningitis, acute renal failure, thromboembolic events and anaphylactic reactions in pediatric patients with CIDP
• Data analysis plan:
  For Part 1 results will include descriptive statistics of the standardized incidence of first-time occurrence of HA during the different calendar-time periods. Cox Proportional hazards modelling will be used to compare the hazard rate of the first-time occurrence of HA following the implementation of each of the two risk minimisation measures to the baseline hazard of HA (before the measures were introduced). For Part 2 results will include the incidence rates of AEs for the paediatric population with CIDP during the cumulative study period.