Non-interventional study of long term treatment with Haemoctin SDH (Biotest NIS-016)

Study type

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

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other

Study drug and medical condition

• Medicinal product name, other:
  Haemoctin SDH
• Medical condition to be studied:
  Factor VIII deficiency

Population studied

• Age groups:
  • Adolescents (12 to < 18 years)
  • Children (2 to < 12 years)
  • Infants and toddlers (28 days – 23 months)
  • Term newborn infants (0 – 27 days)
  • 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:
  150

Study design details

• Main study objective:
  With this NIS long-term data for the effectiveness in bleedingprevention and on QoL will be generated.The following questions will be examined:· What are the factors influencing the risk of bleeding over thetime of treatment?· What are the factors influencing the risk to develop FVIIIinhibitors during treatment with Haemoctin SDH?· Can these inhibitors be further characterized?
• Outcomes:
  Annual bleeding rate defined as episodes per year in patients with Haemoctin SDH treatment, differentiated by prophylaxis and on demand Treatment. · AE and subsequent suspected ADR (AE assessed as causally related with Haemoctin 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 unexpected · characterization of FVIII inhibitors to Haemoctin SDH · QoL determined with the EQ-5D
• 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.