Long-term observational safety study to evaluate the nature and incidence of adverse effects of deferiprone treatment in patients with beta-thalassaemia major aged from 1 month to less than 18 years. (DEEP-3)

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:
  FERRIPROX
• Name of medicine, other:
  Kelfer
• Study drug International non-proprietary name (INN) or common name:
  DEFERIPRONE
• Anatomical Therapeutic Chemical (ATC) code:
  (V03AC02) deferiprone

  deferiprone
• Medical condition to be studied:
  Thalassaemia beta

Population studied

• Age groups:
  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  330

Study design details

• Main study objective:
  The main objective of the study is to investigate serious adverse reactions related to deferiprone treatment in children aged 1 month to less than 18 years diagnosed with beta-thalassaemia major.
• Outcomes:
  Primary outcome is the incidence of serious adverse drug reactions. Secondary outcome is the incidence of non-serious adverse drug reactions.
• Data analysis plan:
  Descriptive analysis will be carried out on demographics, co-prescribing and co-morbidities as well as adverse drug reactions (ADRs).Data will be stratified by covariates such as age (e.g. <10 years, >10 years), gender and country. Cumulative ADR incidence and ADR incidence rate will be calculated Logistic regression will be used to investigate the risk factors for ADRs, Kaplan-Meier Survival and Cox regression analyses will be used to analyse time to withdrawal from deferiprone treatment in order to identify the risk factors for withdrawal. All estimates will be presented with 95% confidence intervals.