Safety of potential paediatric patients treated by idarucizumab: a worldwide non-interventional chart review study (Pediatric NIS)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Non-interventional study based on medical chart review

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  IDARUCIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (V03AB37) idarucizumab

  idarucizumab
• Medical condition to be studied:
  Brief resolved unexplained event

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)
• Estimated number of subjects:
  10

Study design details

• Main study objective:
  • To characterize potential paediatric patients administered with idarucizumab; • To further collect safety data among the paediatric patients until hospital discharge:; o Incidence of thromboembolic events after administrationo ; o Incidence of hypersensitivity / anaphylactic reactionso ; o Incidence of Adverse Events (AE), Serious Adverse Ev
• Outcomes:
  Based on observed events until observation discharge:; - thromboembolic events (incidence rate); - hypersensitivity / anaphylactic reactions (incidence rate); - AE, SAE, ADR, SADR reporting (incidence rates); - in-hospital death (incidence rate, cause of death), ; Comparison of patient characteristics of paediatric patients with and without primary outcome events.
• Data analysis plan:
  Patients will be analysed separately based on the methods by which the idarucizumab usage was identified (eg. Idarucizumab drug administration surveillance program, spontaneous reporting). As this is a descriptive study, no hypotheses will be tested, all variables will be presented using descriptive statistics, incidences rates for the outcome events and 95% confidence intervals. Patients’ characteristics will be described for patients with and without outcome events.