An observational, multicenter study to evaluate the safety and tolerability of deferasirox in the treatment of pediatric patients with non-transfusion-dependent iron overload (NESO)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Non Interventional Safety

Study drug and medical condition

• Name of medicine:
  EXJADE
• Study drug International non-proprietary name (INN) or common name:
  DEFERASIROX
• Anatomical Therapeutic Chemical (ATC) code:
  (V03AC03) deferasirox

  deferasirox
• Medical condition to be studied:
  Iron overload

Population studied

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

    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
• Estimated number of subjects:
  40

Study design details

• Main study objective:
  The primary objective of this study was to characterize the long-term safety profile of deferasirox in pediatric patients with Non-transfusion-dependent thalassemia (NTDT) with exposure up to 5 years.
• Data analysis plan:
  All data summaries and analyses were descriptive only. Categorical data (e.g., gender, race, etc.) were summarized by means of contingency tables; a “missing” category was included as applicable.; Percentages were calculated using the number of patients in the relevant population or subgroup as the denominator. Quantitative data (e.g., age, body weight, etc.) was summarized by appropriate descriptive statistics (i.e. mean, standard deviation, median, minimum and maximum).