A National, Post-Registration, Observational Study of the Longterm Safety and Health Outcome of Patients Treated With Defitelio®, Including Patients With Severe Hepatic VOD After HSCT. (DEFIFRANCE Registry)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  DEFITELIO

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Infants and toddlers (28 days – 23 months)
    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 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:
  The primary objective of this study is to evaluate the efficacy in patients treated with Defitelio® in terms of complete response (CR) of severe hepatic VOD at Day+100 post HSCT and survival at Day+100 post HSCT.
• Outcomes:
  The primary objective of this study is to evaluate the efficacy in patients treated with Defitelio® in terms of complete response (CR) of severe hepatic VOD at Day+100 post HSCT and survival at Day+100 post HSCT. -Describe the main characteristics of patients treated with Defitelio®-Evaluate the rate of SAEs of interest in patients treated with Defitelio®-Determine the rate of acute and chronic GvHD after HSCT-Determine the overall mortality and VOD-related mortality after treatment-Identify prognostic factors influencing CR and survival in patients treated with Defitelio® for severe hepatic VOD
• Data analysis plan:
  MedDRA coding will be used to classify SAEs. The frequency (in absolute values and as a percentage) of SAEs according to SOC (System Organ Class) and events considered individually in each of these classes will be indicated.