Effectiveness and Safety of Upadacitinib for Acute Severe Ulcerative Colitis

NCT07472309 | Completed

• 1 other identifier: KY20252556-F-1
• Study Type: observational
• Target: 81
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this retrospective observational study is to investigate and compare the real-world effectiveness and safety of upadacitinib when used as first-line exposure versus rescue exposure in patients with acute severe ulcerative colitis (ASUC). The key questions to be addressed are: In patients with ASUC, does upadacitinib administered as first-line induction exposure result in higher rates of colectomy-free survival, clinical remission, and endoscopic healing compared with its use as rescue exposure following failure of conventional or biologic therapies? What are the differences in the incidence, type, and severity of adverse events between these two real-world treatment exposure patterns? The researchers will conduct a retrospective analysis of medical records and electronic health data from patients diagnosed with ASUC who received upadacitinib either as part of routine first-line clinical care or routine rescue clinical care. All treatment decisions were made by treating clinicians per standard of care; the investigator did not assign or modify any therapeutic interventions. Data will be collected during a defined follow-up period to compare the real-world effectiveness and safety profiles of the two treatment exposure strategies.

Conditions

Ulcerative Colitis (UC); Acute Severe Ulcerative Colitis; Upadacitinib

Outcome Measures

Primary Outcomes (2)

• Clinical-endoscopic remission rate at 12 weeks

  Clinical remission is defined as a total Mayo score ≤ 2 with no subscore \> 1 and a rectal bleeding subscore of 0; endoscopic remission is defined as a Mayo endoscopic subscore ≤ 1 without mucosal friability.Clinical-endoscopic remission requires achievement of both clinical and endoscopic remission, assessed at 12weeks after upadacitinib initiation.

  12 weeks after upadacitinib initiation

• Colectomy-free rate within 90 days

  The proportion of ASUC patients who did not undergo colectomy within 90 days after the initiation of upadacitinib treatment

  90 days after upadacitinib initiation

Secondary Outcomes (7)

• Patient-Reported Outcomes-2 (PRO2) score at week 1

  1 week after upadacitinib initiation

• Clinical response rate at weeks 8 and 12

  8 weeks and 12 weeks after upadacitinib initiation

• Clinical remission rate at weeks 8 and 12

  8 weeks and 12 weeks after upadacitinib initiation

• Corticosteroid-free clinical remission rate at weeks 8 and 12

  8 weeks and 12 weeks after upadacitinib initiation

• Endoscopic response rate at week 12

  12 weeks after upadacitinib initiation

Study Arms (2)

ASUC Patients with First-Line Upadacitinib Exposure

This retrospective observational cohort includes patients diagnosed with acute severe ulcerative colitis (ASUC) who received upadacitinib as first-line therapy in routine clinical practice. First-line upadacitinib was defined as administration in ASUC patients who had not previously received corticosteroids or biologics during this episode. All treatment decisions were made by treating clinicians per standard of care; the investigator did not assign or modify any therapeutic interventions, and only retrospectively collected and analyzed clinical outcomes from medical records.

Drug: Upadacitinib

ASUC Patients with Rescue Upadacitinib Exposure

This retrospective observational cohort includes patients diagnosed with acute severe ulcerative colitis (ASUC) who received upadacitinib as part of routine rescue therapy (second- and third-line treatment). Second-line rescue therapy was defined as upadacitinib initiation after 3-5 days of intravenous methylprednisolone (40-60 mg/day) with inadequate response. Third-line rescue therapy was defined as upadacitinib use following failure of both intravenous methylprednisolone and infliximab during the same ASUC episode. All treatment decisions were made by treating clinicians in accordance with standard of care; the investigator did not assign, modify, or control any therapeutic interventions, and only retrospectively collected and analyzed clinical outcomes from medical records.

Drug: Upadacitinib

Interventions

Upadacitinib DRUG

Upadacitinib was administered orally as part of routine clinical care for acute severe ulcerative colitis (ASUC), in accordance with standard clinical guidelines. The induction dose was 45 mg once daily for up to 12 weeks (8 weeks for most patients, extended to 12 weeks for a subset with severe disease), followed by a maintenance dose of 30 mg once daily. All dosing decisions were made by treating clinicians; the investigator did not assign, modify, or control any dosing regimen for research purposes.

ASUC Patients with First-Line Upadacitinib Exposure; ASUC Patients with Rescue Upadacitinib Exposure

Eligibility Criteria

• Age: 16 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This is a multicenter, retrospective cohort study conducted at 11 tertiary inflammatory bowel disease (IBD) centers in China from June 2023 to December 2025. A total of 81 patients diagnosed with ASUC according to the modified Truelove and Witts criteria were enrolled. All patients received upadacitinib as first-line or rescue therapy. The study population was categorized into two groups: the first-line therapy group (upadacitinib administered in ASUC patients who had not previously received corticosteroids or biologics during this episode) and the rescue therapy group (including second-line and third-line therapy, defined as upadacitinib initiation after failure of intravenous methylprednisolone or both intravenous methylprednisolone and infliximab).

You may qualify if:

• Patients diagnosed with ASUC according to the modified Truelove and Witts criteria;
• Patients treated with upadacitinib at 11 tertiary inflammatory bowel disease (IBD) centers in China from June 2023 to December 2025；
• Patients with complete and available clinical, endoscopic, and follow-up data；
• Patients for whom the study was approved by the Institutional Research 5.Ethics Committee and conducted in accordance with the Declaration of Helsinki.

You may not qualify if:

• Patients with hemodynamic instability;
• Patients with obvious liver and kidney function injury: bilirubin,aminotransferase (ALT, AST) exceeded the upper limit of normal by 2 times; eGFR \< 60ml/min or dialysis patients;
• Patients allergic to upadacitinib or its excipients;
• Patients whose primary disease was gastrointestinal malignancy;
• Patients currently suffering from serious or uncontrolled underlying diseases of the blood, digestive tract, metabolism, endocrine, lung, heart, nervous system, mental system, etc.;
• Female patients during pregnancy and breastfeeding (including patients with reproductive needs)；
• Patients with current infection with infectious diseases (hepatitis B, hepatitis C, syphilis, AIDS, tuberculosis, etc.);
• Patients with missing key data for outcome assessment;

Sponsors & Collaborators

• Xijing Hospital of Digestive Diseases: lead

Study Sites (1)

Xijing Hospital,Fourth Military Medical University

Xi'an, Shaanxi, 710032, China

Location

Related Publications (2)

• Gisbert JP, Chaparro M. Acute severe ulcerative colitis: State of the art treatment. Best Pract Res Clin Gastroenterol. 2018 Feb-Apr;32-33:59-69. doi: 10.1016/j.bpg.2018.05.007. Epub 2018 May 12.

  PMID: 30060940 BACKGROUND

• Gisbert JP, Chaparro M. Janus kinase inhibitors in the management of acute severe ulcerative colitis: a comprehensive review. J Crohns Colitis. 2025 Feb 4;19(2):jjaf021. doi: 10.1093/ecco-jcc/jjaf021.

  PMID: 39886994 BACKGROUND

MeSH Terms

Conditions

Colitis, Ulcerative

Interventions

upadacitinib

Condition Hierarchy (Ancestors)

Colitis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Inflammatory Bowel Diseases; Colonic Diseases; Intestinal Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 4, 2026
• First Posted: March 16, 2026
• Study Start: June 6, 2023
• Primary Completion: March 3, 2026
• Study Completion: March 3, 2026
• Last Updated: March 16, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)