A multi-centre, multinational, prospective observational registry to collect safety and outcome data in patients diagnosed with severe hepatic veno-occlusive disease (VOD) following haematopoietic stem cell transplantation (HSCT) and treated with Defitelio® (DF VOD-2013-03-REG)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Prospective observational study

Study drug and medical condition

• Medicinal product name:
  DEFITELIO
• Medical condition to be studied:
  Venoocclusive liver disease

Population studied

• Short description of the study population:
  The Study Population is defined as the population of patients with severe hepatic VOD postHSCT who are entered into the registry and treated with Defitelio. This population will be used for all summaries. The initial diagnosis of severe hepatic VOD will be performed by the treating physician according to clinical practice. The relevant sections of the e-form will be filled in accordingly.
• Age groups:
  • 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
• Estimated number of subjects:
  176

Study design details

• Main study objective:
  The main objective of the registry is to assess the incidence rate of specific SAEs of interest (including fatalities) in patients with severe hepatic VOD treated with Defitelio®.
• Outcomes:
  Incidence rate of specific SAEs of interest. -To describe the population treated with Defitelio®-To determine the incidence rate of multi-organ failure (MOF) and Graft versus hostdisease (GvHD) -To determine survival by Day+100 post-HSCT, overall mortality and mortality due toVOD-To determine the rate of VOD/MOF resolution any time after treatment initiation
• Data analysis plan:
  MedDRA coding will be used to classify and tabulate SAEs of interest. Frequencies (absolute and percentages) across SOC and for individual events within those classes will be provided for each treatment group. Safety data will be obtained by collecting the serious adverse events of interest, those listed as identified and potential risks in the Risk Management Plan, namely haemorrhage, coagulopathy, hypotension, immunogenicity (allergic and hypersensitivity reactions), injection site reactions, infection and septicaemia, thromboembolic events, pregnancy and lactation.Incidence rates will be obtained for each of the treatment groups and relative risks calculatedwhere appropriate together with 95% confidence intervals for both the incidence rates and the relative risk for the Defitelio® group and the control group for patients with severe VOD.