This Study is to Describe and Evaluate Patients in Finland Treated With Tofacitinib for the Treatment of Ulcerative Colitis Using Real World Data.

Retrospective Non-interventional Multicenter Patient Chart Data Study on Tofacitinib Realworld Experience in Ulcerative Colitis in Finland (FinTofUC)

The aim of this study is to describe and evaluate clinical outcomes, treatment lines, and to identify the key characteristics of the patients treated with tofacitinib.

Study Overview

• Status: Completed
• Conditions: Ulcerative Colitis

• Study Type: Observational
• Enrollment (Actual): 1

Contacts and Locations

Study Locations

• Finland
  • Helsinki, Finland
    • Pfizer

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

This is a retrospective non-interventional multicenter patient chart review study, collecting real world data from 21 Finnish hospital district databases and for whom data is available.

Description

Inclusion Criteria:

• Xeljanz (tofacitinib) usage for ulcerative colitis
• Diagnosis of ulcerative colitis (ICD-10: K51.0, K51.1, K51.2, K51.3, K51.5, K51.8, K51.9) between January 2010 and December 2021 (incident or prevalent).

Exclusion Criteria:

• Age < 18 years at the start of tofacitinib use
• Use of tofacitinib before reimbursement (1.3.2019)
• < 8 weeks of treatment with tofacitinib at the start of data mining
• History of panproctocolectomy, IPAA or ileostomy.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Patients treated with Tofacitinib; Patients treated with tofacitinib for ulcerative colitis in Finland.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Participant demographics at tofacitinib treatment initiation	Age, gender, weight, height, smoking status, body mass index (BMI), treating hospital	Baseline
Disease characteristics at tofacitinib treatment initiation	Age at diagnosis, duration of disease and extent of colonic involvement according to the Montreal classification: E1 (ulcerative proctitis), E2 (left sided, distal colitis), E3 (pancolitis)	Baseline
Disease severity at tofacitinib treatment initiation	Assessed by Mayo score and fecal calprotectin (f-calprotectin)	Baseline
Laboratory results for biochemical inflammatory markers at tofacitinib treatment initiation	Plasma C-reactive protein (P-CRP), blood thrombocytes (B-thromb), plasma albumin (P-alb), blood leukocytes (B-leuk), blood lymphocytes (B-ly), blood neutrophiles (B-neutr), blood hemoglobin (B-hb) and f-calprotectin	Baseline
Endoscopic findings including histology at tofacitinib treatment initiation		Baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients who are taking tofacitinib		Weeks 8, 16, 24, 52
Rate of clinical remission based on full Mayo score	A clinical remission is defined as a full Mayo score of ≤2 points with no individual sub score exceeding 1 point, with rectal bleeding sub-score of 0	Weeks 8, 16, 24, 52
Rate of clinical remission based on partial Mayo score	A clinical remission is defined as a partial Mayo score <2 points with rectal bleeding sub-score of 0	Weeks 8, 16, 24, 52
Rate of clinical response based on full Mayo score	A clinical response is defined as a full Mayo score decrease of ≥3 points and a decrease of ≥30% from baseline, with a decrease of ≥1 point on the rectal bleeding sub score or an absolute rectal bleeding score of ≤1	Weeks 8, 16, 24, 52
Rate of clinical response based on partial Mayo score	A clinical response is defined as a partial Mayo score decrease of ≥2 points and reduction of at least 25% in partial Mayo score from baseline with an accompanying decrease in rectal bleeding sub score of ≥1 point or absolute rectal bleeding sub score of ≤1	Weeks 8, 16, 24, 52
Proportion of participants in steroid-free clinical remission	Steroid-free clinical remission is defined by full or partial Mayo who did not require any corticosteroid treatment during the period ≥4 weeks prior to the visit	Weeks 8, 16, 24, 52
Proportion of participants reaching clinical response based on full Mayo score	A clinical response is defined as a full Mayo score decrease of ≥3 points and a decrease of ≥30% from baseline, with a decrease of ≥1 point on the rectal bleeding sub score or an absolute rectal bleeding score of ≤1.	Weeks 8, 16, 24, 52
Proportion of participants reaching clinical response based on partial Mayo score	A clinical response is defined as a partial Mayo score decrease of ≥2 points and reduction of at least 25% in partial Mayo score from baseline with an accompanying decrease in rectal bleeding sub score of ≥1 point or absolute rectal bleeding sub score of ≤1	Weeks 8, 16, 24, 52
Time to response as assessed by a decrease based on full Mayo score.		Weeks 8, 16, 24, 52
Time to response as assessed by a decrease based on partial Mayo score.		Weeks 8, 16, 24, 52
Proportion of participants that had f-calprotectin above 250 mg/kg		Baseline
Change from baseline in fecal calprotectin		Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching f-calprotectin below 250 mg/kg of those with active disease based on f-calprotectin at baseline	Active disease defined as fecal calprotectin (f-calprotectin) >250mg/kg.	Weeks 8, 16, 24, 52
Proportion of participants in sustained remission (full Mayo score)		Week 8 to week 16, 24 and 52
Proportion of participants in sustained remission (full Mayo score)		Week 16 to week 24 and 52
Proportion of participants in sustained remission (partial Mayo score)		Week 8 to week 16, 24 and 52
Proportion of participants in sustained remission (partial Mayo score)		Week 16 to week 24 and 52
Proportion of participants in sustained steroid free remission (full Mayo score) (for all patients and for those treated with corticosteroids at baseline).		Week 16 to 24 and 52
Proportion of participants in sustained steroid free remission (partial Mayo score) (for all patients and for those treated with corticosteroids at baseline).		Week 16 to 24 and 52
Change in full Mayo score		Baseline, Weeks 8, 16, 24, 52
Change in partial Mayo score		Baseline, Weeks 8, 16, 24, 52
Proportion of participants in sustained endoscopic remission, mucosal healing or endoscopic response	Endoscopic remission is defined as a sub score = 0. Mucosal healing is defined as a sub score 0-1. Endoscopic response is defined as a sub score reduction from baseline of ≥1.	Baseline, Week 8 to week 16, 24 and 52
Proportion of participants in physician assessed histological remission determined as inactive disease, or normal histology, and change from baseline in histology assessment	Active disease is defined as an endoscopic Mayo sub-score of ≥2 or fecal-calprotectin (f-calprotectin) >250mg/kg. Histology is assessed as subscore 0= normal histology, 1= inactive disease and 2 = active disease.	Baseline, Weeks 8, 16, 24, 52
Proportion of participants in sustained steroid free remission (partial Mayo score) (for all patients and for those treated with corticosteroids at baseline) and endoscopic remission, mucosal healing or endoscopic response	Endoscopic remission is defined as a sub score = 0. Mucosal healing is defined as a sub score 0-1. Endoscopic response is defined as a sub score reduction from baseline of ≥1.	Baseline, Week 8 to week 16, 24 and 52
Proportion of participants in sustained steroid free remission (full Mayo score) (for all patients and for those treated with corticosteroids at baseline) and endoscopic remission, mucosal healing or endoscopic response	Endoscopic remission is defined as a sub score = 0. Mucosal healing is defined as a sub score 0-1. Endoscopic response is defined as a sub score reduction from baseline of ≥1.	Baseline, Week 8 to week 16, 24 and 52
Comparison of response and remission (full Mayo score) based on the extent of colonic involvement according to the Montreal classification		Baseline, Weeks 8, 16, 24, 52
Comparison of response and remission (partial Mayo score) based on the extent of colonic involvement according to the Montreal classification		Baseline, Weeks 8, 16, 24, 52
Proportion of participants with corticosteroid tapering and their tapering rates and doses		Baseline, Weeks 8, 16, 24, 52
Proportion of participants with improvement in stool frequency and change from baseline in stool frequency sub score	Improvement in stool frequency defined as sub score improvement of 1 or more points	Baseline, Weeks 8, 16, 24, 52
Proportion of patients with improvement in rectal bleeding and change from baseline in rectal bleeding sub score	Improvement inrectal bleeding defined as sub score improvement of 1 or more points	Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching normal plasma C-reactive protein (P-CRP) levels and change from baseline	Normal P-CRP levels defined as below 4mg/L	Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching normal blood hemoglobin (B-hb) levels and change from baseline	Normal B-hb levels defined as men=134-167 g/L, women=117-155 g/L	Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching normal blood leukocyte (B-leuk) levels and change from baseline	Normal B-leuk levels defined as 3.4-8.2 x 109/L	Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching normal blood thrombocytes (B-Thromb) levels and change from baseline	Normal B-Thromb levels defined as 150-360 x 109/L	Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching normal blood lymphocyte (B-ly) levels and change from baseline	Normal B-ly levels defined as 1.3-3.6 x 109/L	Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching normal blood neutrophile (B-neutr) levels and change from baseline	Normal B-neutr levels defined as 1.5-6.7 x 109/L	Baseline, Weeks 8, 16, 24, 52
Proportion of participants reaching normal plasma albumin (P-alb) levels and change from baseline	Normal P-alb levels defined as 18-39 years=36-48 g/L, 40-69 years=36-45 g/L, 70 years and over=34-45 g/L	Baseline, Weeks 8, 16, 24, 52
Proportion of participants with extended tofacitinib induction dose	Participants with induction dose after 8 weeks	Baseline, Weeks 8, 16, 24, 52
Real-world dosing of tofacitinib		Baseline, Weeks 8, 16, 24, 52
Survival without drug discontinuation, colectomy or UC-related hospitalization		Baseline, Weeks 8, 16, 24, 52
To Assess Treatment Lines Prior to Tofacitinib Treatment.	Number and type of previous UC treatments.	Baseline
Proportion of responders defined by a fecal calprotectin (f-calprotectin) reduction of ≥50%, ≥75% or ≥90% compared to 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, Weeks 8, 16, 24, 52

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start (Actual): May 30, 2022
• Primary Completion (Actual): November 1, 2022
• Study Completion (Actual): November 1, 2022

Study Registration Dates

• First Submitted: October 5, 2021
• First Submitted That Met QC Criteria: October 5, 2021
• First Posted (Actual): October 19, 2021

Study Record Updates

• Last Update Posted (Actual): March 31, 2023
• Last Update Submitted That Met QC Criteria: March 30, 2023
• Last Verified: March 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Intestinal Diseases; Inflammatory Bowel Diseases; Ulcer; Colitis; Colitis, Ulcerative
• Other Study ID Numbers: A3921390; FinTofUC (Other Identifier: Alias Study Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.