Observational Study To Assess The Effectiveness and Treatment Adherence Of Tofacitinib of Ulcerative Colitis In Clinical Practice In Sweden

Study Overview
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Brief Summary
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This is a prospective observational study using data from an existing, ongoing National Swedish registry (SWIBREG). This study is designed to assess the effectiveness and treatment adherence of tofacitinib on clinical disease activity parameters in patients with ulcerative colitis in Swedish clinical practice. The study will also assess treatment adherence of tofacitinib using the Swedish Prescribed Drug Register.

Official Title
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Observational Study of Tofacitinib in Ulcerative Colitis in Sweden (ODEN)

Conditions
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Ulcerative Colitis

Intervention / Treatment
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* Drug: tofacitinib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The assignment of the patient to tofacitinib is not decided in advance by the protocol but falls within clinical practice and the prescription of the medicine is done according to the SmPC and is clearly separated from the decision to include the patient in the study. The patient must sign the informed consent before enrollment in the study. The informed consent permits extraction of data from SWIBREG at baseline and during the duration of the study. For patients not registered in SWIBREG, they must complete all SWIBREG consents and registration at the time of treatment initiation. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study. Patients, male or female, must be 18 years old or above. The patient must have active disease as confirmed by fecal calprotectin >250 mg/kg or endoscopic assessment corresponding to a Mayo endoscopic subscore ≥2 not more than 4 weeks prior onset to the initiation of tofacitinib treatment. This inclusion criteria applies also to patients that have already been enrolled in the study. Exclusion Criteria: The patient is enrolled in a clinical trial in which the treatment of ulcerative colitis is dictated by a study protocol. If the patient is participating in another ongoing observational study (non-interventional), the patient may be included in this observational study. Patients that fulfill any of the contraindications according to the latest version of the SmPC. Any SmPC label updates will be communicated to all study sites. For whatever reason the physician feels the patient unsuitable to participate in the study.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients prescribed tofacitinib<br>Patients with a confirmed diagnosis of ulcerative colitis with confirmed active disease (biomarker or endoscopy) initiating tofacitinib as per the Swedish summary of product characteristics (SmPC). | Drug: tofacitinib<br>* Observational study<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of Participants in Remission as Measured by Partial Mayo Score | Clinical Remission is defined as a partial score of <2 points with 0 points regarding rectal bleeding. | Week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of Participants who are taking tofacitinib |  | Baseline, Weeks 8, 16, 52, 104 | 
| Proportion of Participants in Clinical Remission Based on Total Mayo Score | Clinical Remission is defined as a total Mayo score of ≤2 points with no individual subscore exceeding 1 point, with 0 points regarding rectal bleeding. | Weeks 8, 16, 52, and 104 | 
| Proportion of Participants in Clinical Response Based on Total Mayo Score | Clinical Response is defined as a total Mayo score decrease of ≥3 points and a decrease of ≥30% from baseline, with a decrease of ≥1 point on the rectal bleeding subscore or an absolute rectal bleeding score of ≤1 | Weeks 8, 16, 52, and 104 | 
| Proportion of Participants in Clinical Remission Based on Partial Mayo Score | Clinical Remission is defined as a partial Mayo score <2 points with 0 points regarding rectal bleeding. | Weeks 8, 16, 52 and 104 | 
| Proportion of Participants in Clinical Response Based on Partial Mayo Score | Clinical Response is defined as a partial Mayo score decrease of ≥2 points and reduction of at least 25% in partial Mayo (pMayo) score from baseline with an accompanying decrease in rectal bleeding subscore of ≥1 point or absolute rectal bleeding subscore of ≤1 point | Weeks 8, 16, 52 and 104 | 
| Proportion of Participants in Steroid-Free Clinical Remission | Steroid-Free Clinical Remission is defined by a total Mayo Score who did not require any corticosteroid treatment during the period ≥4 weeks prior to the visit (for all patients and for those treated with corticosteroids at baseline) | Weeks 16, 52, and 104 | 
| Change from Baseline in Total Mayo Score |  | Baseline, Weeks 8, 16, 52, and 104 | 
| Change From Baseline In Partial Mayo Score |  | Baseline, Weeks 8, 16, 52 and 104 | 
| Change From Baseline In Level of Fecal Calprotectin (f-calprotectin) | Fecal calprotectin is an inflammatory marker for the gastrointestinal tract and considered as a measurement of neutrophil migration to the gastrointestinal tract. Higher values indicate more serious inflammation. | Baseline, Weeks 8, 16, 52 and 104 | 
| Proportion of Responders defined by a Fecal Calprotectin (f-calprotectin) Reduction of ≥50%, ≥75% or ≥90% | Fecal calprotectin is an inflammatory marker for the gastrointestinal tract and considered as a measurement of neutrophil migration to the gastrointestinal tract. Higher values indicate more serious inflammation. | Weeks 8, 16, 52, and 104 | 
| Change from Baseline of C-Reactive Protein (CRP) |  | Baseline, Weeks 8, 16, 52, and 104 | 
| Proportion of Participants In Sustained Remission (Partial Mayo score) |  | Week 8 To Weeks 16, 52 and 104 | 
| Proportion of Participants In Sustained Remission (Partial Mayo score) |  | Week 16 To Weeks 52 and 104 | 
| Proportion of Participants in Sustained Steroid Free Remission (Partial Mayo Score) (for all patients and for those treated with corticosteroids at baseline) |  | Weeks 16 through 52 and at Week 104 | 
| Proportion of Participants in Endoscopic Remission, Mucosal Healing or Endoscopic Response | Endoscopic remission is defined as a subscore of 0. Mucosal healing is defined as a subscore of 0-1. Endoscopic response is defined as a subscore reduction from baseline of ≥1. | Week 8, 16, 52 and 104 | 
| Proportion of Participants in Sustained Endoscopic Remission, Mucosal Healing or Endoscopic Response | Endoscopic remission is defined as a subscore of 0. Mucosal healing is defined as a subscore of 0-1. Endoscopic response is defined as a subscore reduction from baseline of ≥1. | Week 8 to Week 16, 52 and 104 | 
| Proportion of Participants in Sustained Endoscopic Remission, Mucosal Healing or Endoscopic Response | Endoscopic remission is defined as a subscore of 0. Mucosal healing is defined as a subscore of 0-1. Endoscopic response is defined as a subscore reduction from baseline of ≥1. | Week 16 to 52 and at Week 104 | 
| Proportion of Participants in Sustained Steroid Free Remission (Partial Mayo Score) (For All Participants and for Those Treated With Corticosteroids at Baseline) and Endoscopic Remission, Mucosal Healing or Endoscopic Response | Endoscopic remission is defined as a subscore of 0. Mucosal healing is defined as a subscore of 0-1. Endoscopic response is defined as a subscore reduction from baseline of ≥1. | Week 16 to 52 and 104 | 
| Change From Baseline In Inflammatory Bowel Disease Fatigue (IBD-F) Score |  | Baseline, Weeks 8, 16, 52 and 104 | 
| Change From Baseline In EuroQol 5 Dimensions 5 Levels (EQ5D-5L) |  | Baseline, Weeks 8, 16, 52, and 104 | 
| Change From Baseline In Short Health Scale (SHS) |  | Baseline, Weeks 8, 16, 52 and 104 | 
| Proportion of Participants Who Had a Colectomy |  | Weeks 8, 16, 52, and 104 | 
| Comparison of Response and Remission (Partial Mayo Score) Based on the Extent of Ulcerative Colitis According To the Montreal Classification |  | Weeks 8, 16, 52 and 104 | 
| Proportion of Participants In Sustained Remission (Total Mayo score) |  | Week 16 To Weeks 52 and 104 | 
| Proportion of Participants In Sustained Remission (Total Mayo score) |  | Week 8 To Weeks 16, 52 and 104 | 
| Proportion of Participants in Sustained Steroid Free Remission (Total Mayo Score) (For All Participants and for Those Treated With Corticosteroids at Baseline) and Endoscopic Remission, Mucosal Healing or Endoscopic Response | Endoscopic remission is defined as a subscore of 0. Mucosal healing is defined as a subscore of 0-1. Endoscopic response is defined as a subscore reduction from baseline of ≥1. | Week 16 to 52 and 104 | 
| Proportion of Participants in Sustained Steroid Free Remission (Total Mayo Score) (for all patients and for those treated with corticosteroids at baseline) |  | Weeks 16 through 52 and at Week 104 | 
| Comparison of Response and Remission (Total Mayo Score) Based on the Extent of Ulcerative Colitis According To the Montreal Classification |  | Weeks 8, 16, 52 and 104 | 
| Proportion of Participants in Steroid-Free Clinical Remission | Steroid-Free Clinical Remission is defined by a partial Mayo Score who did not require any corticosteroid treatment during the period ≥4 weeks prior to the visit (for all patients and for those treated with corticosteroids at baseline) | Weeks 16, 52, and 104 | 
| Proportion of participants reaching f-calprotectin below 250 mg/kg of those that had f-calprotectin above 250 mg/kg at baseline | Fecal calprotectin is an inflammatory marker for the gastrointestinal tract and considered as a measurement of neutrophil migration to the gastrointestinal tract. Higher values indicate more serious inflammation. | Baseline, week 8, 16, 52 and 104 | 
| Portion of participants with dose change of tofacitinib |  | Week 8, 16, 52 and 104 | 
| Portion of participants with a termination of tofacitinib |  | Week 8, 16, 52 and 104 | 
| Portion of participants with dose and dose changes of tofacitinib and corticosteroids |  | Week 8, 16, 52 and 104 | 
| Proportion of particpants with changes in rectal bleeding and stool frequency | Proportion of patients with improvement in rectal bleeding and stool frequency with a change in baseline sub score 1 | Baseline, Week 2 | 
| Proportion of participants with changes in EQ5D and SHS |  | Baseline, Week 2 | 
| Proportion of particpants with rectal bleeding and stool frequency sub score indicative of mild disease |  | Baseline, Week 2 | 
| Proportion of participants with stool frequency and rectal bleeding subscore of 0 |  | Baseline, Week 2 | 
| Proportion of participants with mild abdominal pain |  | Baseline, Week 2 | 
| Proportion of participants with no abdominal pain |  | Baseline, Week 2 | 
| Proportion of participants with no bowel urgency |  | Baseline, Week 2 | 
| Proportion of participants with reduction of ≥ 1 point from baseline rectal bleeding and stool frequency sub score |  | Baseline, Week 2 |