Disease characteristics and outcomes of pulmonary arterial hypertension in children and adolescents in real-world clinical settings: Sytematic Review of prospective, observational registries (Pediatric Systematic Review)

Study type

• Study topic:
  Disease /health condition

  Other
• Study topic, other:
  Disease/Epidemiology study
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Systematic review and meta-analysis

Study drug and medical condition

• Medical condition to be studied:
  Pulmonary arterial hypertension

Population studied

• Short description of the study population:
  Children and adolescents in real-world clinical settings with pulmonary arterial hypertension (PAH).
• Age groups:
  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)
• Estimated number of subjects:
  500

Study design details

• Main study objective:
  For each registries, the objectives are to describe: -Patient demographics and disease characteristics -Outcomes of PAH in all pediatric patients (including patients on Tracleer). The main areas of interest are general development (growth and sexual maturation), clinical worsening, hospitalization and death-Safety experience in Tracleer-treated pediatric patients
• Outcomes:
  -Outcomes of general development (weight, height, sexual maturation)-Outcomes of PAH such as clinical worsening -Outcomes of safety relevant experience in the sub-group of Tracleer treated patients.
• Data analysis plan:
  Data analysis will be exploratory, and will be performed for each registry separately by the data owner using similar statistical methods based on a common SAP. Two groups – ‘All patients’ and ‘All Tracleer patients’ – will be described within each study.