Non-interventional study for long term documentation of treatment with Vihuma in patients with hemophilia A (Biotest NIS-019)

Study type

• Study topic:
  Disease /health condition

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

  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prospective, single arm study

Study drug and medical condition

• Name of medicine:
  VIHUMA
• Medical condition to be studied:
  Factor VIII deficiency

Population studied

• Short description of the study population:
  Patients with hemophilia A treated with Vihuma recombinant factor VIII (FVIII) at home and, in some exceptional cases, in the clinic or a local doctor's practice.; Patients with following criteria were included:; 1. Treatment in accordance with the SPC for Vihuma; 2. Children of all ages and adult patients with FVIII deficiency (previously treated and previously untreated patients); 3. Written informed consent to allow data collection and data transfer to third party
• 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)

  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:
  Immunocompromised
• Estimated number of subjects:
  60

Study design details

• Main study objective:
  With this NIS long-term data for the effectiveness in bleeding prevention and on QoL will be generated.What is the dose and frequency of Vihuma in prophylaxis?Is it possible to reduce the dose or extend the frequency of applications compared to previously used factor VIII products?What are the factors influencing the risk of bleeding over the time of treatment?
• Outcomes:
  Annual bleeding rate defined as episodes per year in patients with Vihuma treatment, differentiated by prophylaxis and on demand treatmentDose and frequency of Vihuma applications in comparison to previously used factor VIII products, AE and subsequent suspected ADR (AE assessed as causally related with Vihuma treatment)AE with bleeding = AE of special interest (AESI) with extended bleeding documentation, for e.g. if the duration and severity of the bleeding is within the situation as expected or unexpectedOccurrence and characterization of FVIII inhibitors to Vihuma
• Data analysis plan:
  All analyses will be performed in an exploratory sense. Data will be analyzed using descriptive statistics. For continuous variables, mean, standard deviation, minimum, maximum, median, 25% and 75% percentiles will be presented. Qualitative and categorical variables will be presented by means of absolute and relative frequencies. A medical evaluation of the findings will be performed. Details of analysis will be described in a statistical analysis plan.