Ulcerative Colitis
Conditions
Keywords
Ulcerative Colitis, Tofacitinib, France, Real world data
Brief summary
This is an observational prospective study with two years of follow-up, designed to evaluate the effectiveness of tofacitinib in patients with moderate to severe ulcerative colitis in French clinical practice
Detailed description
TO FAst is a non-interventional study in France with primary objective to describe the clinical benefit of tofacitinib 1 year after its initiation for the treatment of moderate to severe UC in routine clinical practice. The study will also make it possible to report the clinical benefit 2 years after its initiation, to search for predictors of clinical benefit, improve our understanding of the efficacy of treatment in a real-life setting (in terms of response and speed of response), describe the characteristics of patients starting a treatment by tofacitinib, its real-life patterns of use as well as patient adherence to treatment.
Interventions
DRUGTofacitinib
Observational study
Sponsors
Pfizer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients of 18 years old or above * Patients with confirmed diagnosis of moderate to severe ulcerative colitis * Patients for whom gastroenterologist decides to initiate treatment with tofacitinib as per the French SmPC * Patients informed about the study procedures and receiving an information letter signed by the investigator
Exclusion criteria
* Patients who have already received tofacitinib treatment before baseline * Patients that fulfill any of the contrindications according to the latest version of the SmPC
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients with clinical benefit one year after initiation of tofacitinib treatment. | Week 52 | The definition of clinical benefit is independent of the discontinuation or not of tofacitinib treatment during the observation period. Clinical benefit at year is defined on the basis of symptomatic remission evaluated with the PRO2 score ≤1 (absence of rectal bleeding and a stool frequency score between 0 and 1)\*. Patients who died or who had a colectomy or used another biologic/anti-JAK/immunosuppressant will be considered to be non-responders, as well as patients who used oal corticosteroids for UC, (regardless of the treatment duration) during the 3 months preceding the end of the observation period. The clinical benefit of tofacitinib is independent of the administration or not of 5-ASA, or corticosteroids (not complying with the above definition) during the observation period (between 0 and 1 year). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients with clinical benefit of tofacitinib at 2 years | week 104 | — |
| Predictors of the clinical benefit at one year identified from the available baseline data | Week 52 | — |
| Proportion of patients in clinical remission and still receiving tofacitinib | Week 52 and Week 104 | Clinical remission is defined as partial Mayo score (PMS) \<2 |
| Proportion of patients in clinical remission without corticosteroids (oral or topical with systemic effects for UC) | Week 52 and week 104 | — |
| Proportion of patients with short-term clinical response for patients still treated with tofacitinib | Approximately week 8 and 16 | Clinical response is defined as a reduction in partial Mayo score ≥ 3 points and ≥ 30% with respect to baseline, with a concomitant reduction in rectal bleeding sub-score ≥ 1 point (absolute sub-score of 0 or 1). |
| Proportion of patients with biological response during the observation period | Week 52 and 104 | Biological response is defined as 50% reduction in the initial value of CRP or Fecal Calprotectine (FCP) |
| Proportion of patients with endoscopic improvement during the observation period | Week 52 and 104 | endoscopic improvement is defined as endoscopic subscore of 0 or 1 |
| Proportion of patients in sustained clinical remission | Week 52 and 104 | Clinical remission is defined as partial Mayo score (PMS) \<2 at 52 and 104 weeks |
| Proportion of patients with extraintestinal manifestations at each visit | Week 8, 16, 24, 72, 52, 104 | — |
| Proportion of patients with a colectomy during study follow-up and time of occurrence | Week 8, 16, 24, 72,52 and 104 | — |
| Characteristics of patients and UC, on the basis of all the data collected at baseline | Week 104 | — |
| Description of the changes in the rectal bleeding and stool frequency subscores during the first 2 weeks after initiation of tofacitinib therapy | 14 days | (self-assessment by patients) |
| Change in patient quality-of-life evaluated from the SIBDQ questionnaire between baseline and 1 year, baseline and 2 years, and between 1 and 2 years | Week 52, Week 52 to week 104 and week 104 | — |
| Change in adherence to tofacitinib treatment during each visit | Week 8, 16, 24, 72, 52, 104 | Using MARS questionnaire |
| Proportion of patients with serious and non-serious adverse events. | Week 8, 16, 24,72,52 and 104 | — |
| Time to loss of response to tofacitinib treatment in patients after dose reduction to 5 mg BID at the end of induction | Week 8, 16, 24, 72, 52 and 104 | The clinical loss of response is defined by a recrudescence of the symptoms that lead to a systemic therapeutic intervention (return to previous dose of tofacitinib or corticosteroid therapy, or an immunosuppressant or biologic/other anti-JAK) |
Other
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients with a clinical response* 1 year and 2 years after initiation of tofacitinib | Week 52 and 104 | Reduction in partial Mayo score ≥ 3 points and ≥ 30% with respect to baseline, with a concomitant reduction in rectal bleeding sub-score ≥ 1 point (absolute sub-score of 0 or 1). |
Countries
France
Outcome results
None listed