Observational Study Evaluating Effectiveness and Safety of Real-World Treatment with Damoctocog alfa pegol in Previously Treated Patients with Hemophilia A (HEM-POWR)

Study type

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

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  Jivi
• Medical condition to be studied:
  Haemophilia A without inhibitors

Population studied

• Age groups:
  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)
• Estimated number of subjects:
  200

Study design details

• Main study objective:
  To assess the effectiveness of prophylaxis with damoctocog alfa pegol in the real-world setting through the collection of total bleeding events and analysis of ABR in the different prophylaxis regimens (following approved local label), or any other regimen prescribed by the physician as part of normal clinical practice in patients with hemophilia A.
• Outcomes:
  Mean/median annualized number of reported total bleeds in patients with hemophilia A. The study will capture patterns of switching in damoctocog alfa pegol dose and dosing regimen, reasons for choice of treatment regimen, damoctocog alfa pegol consumption, adverse events, pharmacokinetics (if part of routine practice), as well as patient treatment satisfaction, work productivity and activity impairment.
• Data analysis plan:
  Statistical analyses will be of an explorative and descriptive nature. All variables will be analyzed descriptively with appropriate statistical methods: categorical variables by frequency tables (absolute and relative frequencies) and continuous variables by sample statistics (i.e. mean, standard deviation SD, minimum, median, quartiles, and maximum). Continuous variables will be described by absolute value and as change from baseline per analysis time point, if applicable. All analyses will be performed for the total study population (overall analysis). Separate analyses for individual participating countries or regions will be provided if required for local reasons when sufficient data is available. Annual analyses are planned to provide annual safety reports and preliminary results to support publications. The final analysis will be performed after end of the study, which is the date the analytical dataset is completely available.