Real World Evidence of Safety and Dosing of Mircera in Children with Chronic Kidney Disease

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

  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Non-interventional study secondary data use (NIS SDU) and voluntary post-authorization safety study (PASS) of pediatric patients from the International Pediatric Peritoneal Network (IPPN) and International Pediatric Hemodialysis Network (IPHN)

Study drug and medical condition

• Name of medicine:
  MIRCERA
• Medical condition to be studied:
  Chronic kidney disease

Population studied

• Short description of the study population:
  Pediatric patients aged from 0 months to less than 18 years old on chronic peritoneal dialysis (PD) or HD, with at least one observation while treated with Mircera, who are included in two international, prospective, multicenter registries (IPPN and IPHN).
• Age groups:
  • Paediatric Population (< 18 years)

    • Adolescents (12 to < 18 years)
    • Children (2 to < 12 years)
    • Infants and toddlers (28 days – 23 months)
    • Neonate

      • Term newborn infants (0 – 27 days)
• Special population of interest:
  Renal impaired
• Estimated number of subjects:
  148

Study design details

• Main study objective:
  The objectives for the study are to describe the safety profile of Mircera by aggregate assessment of safety data (number and causes of deaths and hospitalizations) and to assess the relationship between Mircera dosing and Hb concentrations using patient level data.
• Outcomes:
  Descriptive analyses of aggregate safety data for the cumulative number and leading cause of hospitalizations and deaths, over the period of observation (patients on Mircera treatment in the registry) and until 6 months after the last observation. Descriptive analysis of the relationship between Mircera dose and Hb concentrations using patient level data at the first and subsequent observations. An external validation of the Modeling and Simulation Framework will be performed and compared to observed data from the present study. The dose conversions from previous ESA treatment to Mircera will be evaluated.
• Data analysis plan:
  The primary objectives will be addressed by conducting descriptive analyses for aggregate safety data, including cumulative number and cause of hospitalizations and deaths over the observation period and until 6 months after the last observation on Mircera and for patient level data on Mircera dose and Hb concentration at the first and subsequent observation(s) under Mircera treatment. Patient characteristics to be evaluated include demographics, clinical characteristics, specific treatments, and laboratory measures within each registry (IPPN and IPHN) for each age group. The secondary objectives will be addressed by conducting an external validation of the Modeling and Simulation Framework for Mircera that has been developed on Phase II and III adult data and the first pediatric study DOLPHIN (NH19707, Fischbach et al. 2018). In addition, the dose conversions from previous ESA treatment to Mircera that were determined/tested in studies NH19707 (IV) and NH19708 (SC) will be evaluated.