Emicizumab Use in Pediatric Patients in the Real World: an Analysis of the PedNet Registry

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

  Effectiveness study (incl. comparative)

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

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  Hemlibra
• Study drug International non-proprietary name (INN) or common name:
  EMICIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (B02BX06) emicizumab
• Medical condition to be studied:
  Haemophilia A without inhibitors

  Haemophilia A with anti factor VIII

Population studied

• Age groups:
  Paediatric Population (< 18 years)

  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:
  215

Study design details

• Main study objective:
  The primary objective for this study is to evaluate the overall safety and tolerability of emicizumab administration in all pediatric patients with haemophilia A in real-world conditions, and in subgroups determined by age and inhibitor status, as well as by severity for patients without inhibitors
• Setting:
  PedNet Registry is the largest registry in the world for pediatric patients with hemophilia. Currently, at the time of protocol alendment, 19 European countries plus Canada with approximately 32 treatment centers participate in the registry. The registry includes data for children and adolescents up to age 18 years, and with any disease severity (FVIII <25 IU/dL), which provides an adequate representation of the pediatric patient population.; ; The following criteria describe the population eligible for this study, which is a subset of the overall population participating in the PedNet Registry.; Inclusion criteria for inclusion in the PedNet Registry:; - Diagnosis of hemophilia A; - Factor VIII activity < 25 IU/dL; - Treated in one of the participating centers; Additional inclusion for emicizumab-specific analysis:; - Received prophylactic treatment with emicizumab; ; Exclusion criteria for the PedNet registry:; - Referral to a participating HTC after development of inhibitors; - Informed consent for participation in the PedNet Registry not obtained; Exclusion criteria for emicizumab-specific analysis:; - Inherited or acquired bleeding disorder other than hemophilia A
• Outcomes:
  - Frequency and incidence of thromboembolic events (TEs), thrombotic microangiopathy (TMA), and anaphylaxis (including terms of systemic hypersensitivity, anaphylaxis, and anaphylactoid events), overall and in subgroups determined by age and inhibitor status, as well as by severity for patients without inhibitors. ; ; Safety endpoints:; - Frequency and incidence of any adverse events (overall, by age, and inhibitor status).; ; Effectiveness endpoints (overall, by age, and inhibitor status):; - Annual bleeding rate for treated joint and major bleeds; ; - Number of treated bleeds, including soft tissue and minor bleeds; ; - Percentage of patients with zero treated bleeds.
• Data analysis plan:
  The Marketing Authorisation Holder (MAH) will receive aggregate level data of patients treated with emicizumab from the PedNet Registry on an annual basis. Based on the number of patients, number of adverse events (AEs), and exposure to emicizumab provided by the PedNet Registry, the MAH will perform analyses of frequencies/incidence of AEs overall and grouped by age and inhibitor status, as well as by severity for patients without inhibitors. The youngest age group will be newborns (birth to 28 days). Other age groups include: 29 days to less than(<)6 months, 6 months-<2 years, 2 years-<6 years, 6 years-<12 years, and 12 years-18 years. The MAH will report annual bleeding rate (ABR) for treated bleeds, percentage of zero bleeds, ABR for joint bleeds and major bleeds overall and grouped by age and inhibitor status, as well as by severity for patients without inhibitors, as sent by the PedNet Registry. ABRs for soft tissue bleeds and minor bleeds is not calculated by PedNet and therefore only the number of bleeds will be presented.

Documents

Study report

Interim_CSR,_Study_MO40685_(PEDNET),_PASS_Annual_Report_30Sep2020,_Published_Output-1_Redacted

MO40685 (PEDNET)-Interim CSR Synopsis 2, PASS Annual Report_30Sep2021_Redacted

MO40685 (PEDNET)-Interim CSR Synopsis 3_PASS Annual Report_28Sep2022_Redacted

MO40685 (PEDNET)-Interim CSR Synopsis 4_Annual Report_29-Sep-2023_Redacted.pdf