OBSERVATIONAL SAFETY AND EFFECTIVENESS STUDY OF PATIENTS WITH POLYARTICULAR JUVENILE IDIOPATHIC ARTHRITIS TREATED WITH TOCILIZUMAB

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Other

  Safety study (incl. comparative)
• If ‘other’, further details on the scope of the study:
  Observational safety and effectiveness study as post-marketing commitment to FDA and EMA
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  ROACTEMRA
• Study drug International non-proprietary name (INN) or common name:
  TOCILIZUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AC07) tocilizumab

  tocilizumab
• Medical condition to be studied:
  Juvenile idiopathic arthritis
• Additional medical condition(s):
  Polyarticular juvenile idiopathic arthritis

Population studied

• Short description of the study population:
  Patients enrolled in the feeder registries must meet all of the following criteria for ; inclusion in this study:; - Diagnosis of polyarticular juvenile idiopathic arthritis (pJIA), defined according to ILAR classification (Petty et al. 2004), and in line with the licensed indications in the United States and the European Union:; Patients with rheumatoid factor (RF)-positive pJIA; Patients with RF-negative pJIA; Patients with oligoarticular juvenile idiopathic arthritis (eoJIA); - Initiation of treatment with TCZ (IV or SC) or a comparator biologic. The TCZ group will include patients with no previous exposure to TCZ (IV or SC). The comparator biologic group will include patients with no previous ; exposure to that specific comparator biologic.; - Age <=17 years at the time of initiation of treatment with TCZ (IV or SC) or a comparator biologic
• Age groups:
  • Children (2 to < 12 years)
  • Adolescents (12 to < 18 years)
• Estimated number of subjects:
  600

Study design details

• Study design:
  This is an international, multicenter, prospective, observational-cohort study to examine long-term safety and effectiveness data from patients with pJIA.
• Main study objective:
  To assess the long-term safety and effectiveness of tocilizumab in relation to comparator biologics in the treatment of pJIA in a real-world setting for 5 years.
• Setting:
  This was an international, multicenter, prospective, observational-cohort study, designed to examine long-term safety and effectiveness data in a real-world setting. Data was obtained from patients enrolled in active national disease or treatment registries (feeder registries) located in the United States and the Germany.
• Comparators:
  Patients receiving a comparator biologic
• Outcomes:
  - Rate of serious adverse events ; - Rates of serious adverse events in the following categories of special interest: * Infections * Cardiovascular events * Malignancies * Gastrointestinal perforations ; - Rate and treatment outcome of uveitis - Growth patterns - Development patterns; - Juvenile Arthritis Disease Activity Score in 10 joints (JADAS-10)
• Data analysis plan:
  Descriptive summary analyses will be used to characterize baseline demographics, medical history, medications, growth and development. ; Incidence rates, with 95% confidence intervals, will be provided for serious adverse events. Height standard deviation scores will be summarized descriptively over time by treatment group. The data for development patterns will be summarized by gender for each treatment group. ; The rate of uveitis and description of treatment outcome will be summarized. JADAS-10 will be summarized over time.
• Summary results:
  This 5-year real-world study confirms a favorable long-term benefit-risk profile for tocilizumab (TCZ) in pediatric patients with pJIA, aligning with current labeling.; ; Safety and AESIs: No increased risk of atherosclerosis, malignancies, or GI perforations was observed. Serious infection rates were very low and identical to the comparator group. TCZ demonstrated a lower incidence of uveitis despite a more refractory baseline population with no serious cases and observations failing to support a direct causal link.; ; Growth and Development: Continuous TCZ treatment does not adversely impact physical or pubertal development; rather, it facilitates the normalization of growth (height Standard Deviation Score [SDS]) by Year 5 through effective inflammation control.; ; Efficacy: The 10 mg/kg IV Q4W (and SC) regimen provides durable disease control (Juvenile Arthritis Disease Activity Score in 10 joints [JADAS-10]), demonstrating effectiveness even in highly treatment-refractory patients who previously failed TNF-inhibitors, regardless of RF status.; ; The clinical benefits of TCZ observed in controlled trials translate effectively to real-world practice with no new safety signals identified over long-term exposure.