An Observational Post-authorization Safety Study of Ustekinumab in the Treatment of Pediatric Patients Aged 6 Years and Older With Moderate to Severe Plaque Psoriasis

Study type

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

  Effectiveness study (incl. comparative)

Non-interventional study

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

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  USTEKINUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AC05) ustekinumab

  ustekinumab
• Medical condition to be studied:
  Psoriasis

Population studied

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

    • Children (2 to < 12 years)
    • Adolescents (12 to < 18 years)
• Estimated number of subjects:
  135

Study design details

• Main study objective:
  To monitor the long-term safety of ustekinumab in pediatric patients with moderate to severe plaque psoriasis, through monitoring for the following adverse events potentially related to immune modulation: serious infections, malignancies and autoimmunity. To monitor the long-term effects of ustekinumab on growth (height, weight, BMI) and development (sexual maturity based on the Tanner scale).
• Outcomes:
  Long-term safety of ustekinumab in pediatric patients with moderate to severe plaque psoriasis, through monitoring for the following adverse events potentially related to immune modulation: serious infections, malignancies and autoimmunity. Long-term effects of ustekinumab on growth (height, weight, BMI) and development (sexual maturity based on the Tanner scale). Clinical outcomes (PASI, PGA, BSA), quality of life CDLQI, and comorbidities in pediatric patients with moderate to severe plaque psoriasis treated with ustekinumab.
• Data analysis plan:
  Data from the study will be evaluated using a longitudinal observational inception cohort to monitor safety and tolerability, clinical outcomes, quality of life, comorbidities, and treatment regimens. The clinical outcomes of patients with different burdens of disease and/or treatment modalities will also be evaluated. No formal hypothesis testing is planned. Appropriate descriptive statistics will be used to summarize data including medical history, disease characteristics, prior and concomitant psoriasis therapies, and clinical outcomes. Incidences of adverse events in patients who have received ustekinumab will be collected and analyzed. The primary analysis will include all ustekinumab-exposed patients. Subgroup analyses will be performed by age category on patients with age at start of data collection (initiation of ustekinumab treatment) ≥12 years to <18 years, and on patients with age at start of data collection ≥6 years to <12 years.