Juvenile arthritis Methotrexate/Biologics long-term Observation (JuMBO)

Study type

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

  Effectiveness study (incl. comparative)

  Other
• If ‘other’, further details on the scope of the study:
  Cost of illness

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (L04AB01) etanercept

  etanercept

  (L04AB04) adalimumab

  adalimumab

  (L04AC07) tocilizumab

  tocilizumab

  (L04AX03) methotrexate

  methotrexate
• Medical condition to be studied:
  Juvenile idiopathic arthritis

Population studied

• Age groups:
  • Adolescents (12 to < 18 years)
  • Adults (18 to < 46 years)
• Estimated number of subjects:
  2000

Study design details

• Main study objective:
  The aim of JuMBO is to describe the long-term safety, effectiveness, and cost of biologic drugs, especially of etanercept, adalimumab, and tocilizumab, in JIA in comparison to a conventional DMARD therapy (MTX). In order to achieve this goal, the patients already enrolled in BiKeR are to be observed after reaching adult age and/or leaving the pediatric rheumatology care.
• Outcomes:
  1. To study the long-term safety of nonbiologic (nb) DMARDs and biologic (b) DMARDS in young adults with JIA by recording the frequency, kind, severity and consequences of serious and non-serious adverse events.2. To evaluate the long-term effectiveness outcomes of nbDMARDs and bDMARDS by assessing disease activity, function, pain, final height, quality of life and duration under therapy. 1. To determine cost effectiveness of etanercept, adalimumab, and tocilizumab by analyzing direct and indirect costs as well as treatment outcomes including quality of life.2. To provide DNA samples for a joint pharmacogenetic study with the study center Sankt Augustin and the University of Calgary aiming at the identification of genetic predictors influencing efficacy and toxicity of drugs.
• Data analysis plan:
  The following statistical principles will be applied in JuMBO for safety and effectiveness analyses: (i) propensity score methods (confounding by indication), (ii) Cox regression (confounder adjustment, changing risks), (iii) Generalized regression models for survival data (confounder adjustment, changing risks, recurrent AEs), (iv) Generalized estimation equations (confounder adjustment, changing risks, recurrent AEs) and (v) Missing data models (missing data). In case patients are lost to follow-up, the reasons for study non-completion are determined and comparisons of drop-outs and non-drop outs are performed. Furthermore, survival analysis methods will be used to study treatment adherence of biologic drugs and the reasons for discontinuation. All subjects who received at least one dose of a specific biologic DMARD will be included in the safety analysis.