12- and 18-Month Outcomes and Long- Term Survival of Tofacitinib in Ulcerative Colitis

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Retrospective study

Study drug and medical condition

• Name of medicine:
  XELJANZ
• Medical condition to be studied:
  Colitis ulcerative

Population studied

• Short description of the study population:
  Patients with ulcerative colitis aged 18 years or older received tofacitinib therapy after 1 May 2018 identified in the Mass general Brigham (MGB) health system. ; Inclusion criteria: ; 1. Age 18 years or older.; 2. Initiation of tofacitinib therapy for ulcerative colitis on or after May 1, 2018.; 3. Patients within the MGB health system.; ; Exclusion criteria:; 1. History of prior colectomy.; 2. Primary indication of tofacitinib therapy is not ulcerative colitis.; 3. Diagnosis of Crohn's disease or intermediate colitis.; 4. Combination biologic therapy (eg., tofacitinib and vedolizumab simultaneously).
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with ulcerative colitis
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  To perform a retrospective cohort study to assess long-term clinical outcomes at 52 and 78 weeks of tofacitinib therapy for Ulcerative Colitis.
• Outcomes:
  Assess proportions of clinical remission (Simple Clinical Colitis Activity Index (SCCAI) or Mayo less than or equal to 2 or Physician global assessment (PGA)) and corticosteroid-free clinical remission (with no use of corticosteroids within 30 days preceding assessment) at week 52 and 78 after tofacitinib induction in a real-world cohort of patients with Ulcerative Colitis. Univariable and multivariable logistic regression to identify baseline predictors of corticosteroid-free clinical remission (with no use of corticosteroids within 30 days preceding assessment) at weeks 52 and 78.
• Data analysis plan:
  Descriptive statistics will be presented to describe patient characteristics. Categorical covariates will be described by frequency distribution while continuous covariates expressed in terms of their mean and standard deviation or median and interquartile range (IQR) as appropriate. Univariate and multivariable logistic regression models will be used to identify predictors (among all independent variables) of corticosteroid-free remission at Week 52 and week 78(2 separate models). Variables from the univariable analysis that are statistically significant at p<0.10 will be included in the final multivariable model. Adjusted odds ratios with 95% confidence intervals will be calculated using logistic regression models and reported in the final models. Patients who die prior to assessment of endpoints will be excluded from the logistic regression analysis.