A Longitudinal, Retrospective, Multi-centre Observational Study to Evaluate Effectiveness, Persistence, Treatment Patterns and Safety of Australian Patients Receiving Early Access to Tofacitinib

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  A longitudinal, retrospective, non-interventional. multi-center observational study

Study drug and medical condition

• Name of medicine:
  XELJANZ
• Study drug International non-proprietary name (INN) or common name:
  TOFACITINIB
• Anatomical Therapeutic Chemical (ATC) code:
  (L04AF01) tofacitinib

  tofacitinib
• Medical condition to be studied:
  Colitis ulcerative

Population studied

• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  160

Study design details

• Main study objective:
  Examine the disease characteristics and outcomes in ulcerative colitis patients granted access to tofacitinib in Australia
• Outcomes:
  - Clinical response rate (decrease in the partial Mayo score by 2 or partial Mayo score <2) at end of induction (week 8-12) and week 16, ; - Remission rate at end of induction (week 8-12) / week 16 ; - Proportion of patients on tofacitinib completing induction therapy (8 or 16 weeks) ; - Persistence of response up to week 52 Resolution of rectal bleeding at end of induction ; - Endoscopic healing Cessation of steroids ; - Steroid free remission at end of induction and at week 48 ; - Change in CRP & cholesterol (LDL/HDL) levels ; - Colectomy rate
• Data analysis plan:
  Descriptive summary statistics for continuous variables will be used to describe the study population including number of participants, mean, standard deviation, median and range. ; Categorical variables will be described with frequency counts and percentages. ; Pre-specified groups for statistical analysis include remitters, responders, and non-responders at the specified time points. ; P values will be calculated from chi-square for categorical variables and t-tests for continuous variables with normal distribution and non-parametric testing for non-normally distributed data. ; Kaplan-Meier survival analyses will be used to analyse individual predictors and display survival curves. Cox proportional hazards regression will be used to analyse multivariable survival data.