Surveillance Of Emicizumab-Treated Patients: An Analysis of the EUHASS Pharmacovigilance Registry (Emicizumab Pharmacovigilance: EUHASS registry)

Study type

• Study topic:
  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:
  Cohort surveillance

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

  emicizumab
• Medical condition to be studied:
  Haemophilia A with anti factor VIII

  Haemophilia A without inhibitors

Population studied

• Short description of the study population:
  The study population will consist of patients with Hemophilia A treated with emicizumab at centers participating in the EUHASS registry. Depending on the local approval/reimbursement decisions in the participating countries, the study population may include patients with any level of disease severity without FVIII inhibitors.
• 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:
  Hepatic impaired

  Immunocompromised

  Pregnant women

  Renal impaired
• Estimated number of subjects:
  680

Study design details

• Main study objective:
  The primary objective for this study is to estimate the incidence of TE, TMA, and anaphylaxis under real-world conditions in patients exposed to emicizumab, with or without coagulation factor products.
• Data analysis plan:
  Descriptive statistics on endpoints, including number and percentage of patients, will be provided. Continuous variables will be summarized by mean, median, minimum, maximum, and interquartile range (IQR). Categorical variables will be summarized by counts, percentages, and corresponding 95% confidence intervals (CIs). Data generated within the first 7 calendar years post-marketing authorization throughout the European Union (2018-2024) will be analyzed annually. Proportions of each adverse event, along with corresponding 95% CIs, will be calculated annually for all patients exposed to emicizumab. In addition, proportions of TE and TMA will be calculated annually for patients exposed to emicizumab and each of the following agents: aPCC, rFVII, and FVIII.