A Comparative Retrospective Observational Study of the Effectiveness and Safety of Upadacitinib as First-Line and Rescue Therapy in Acute Severe Ulcerative Colitis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 81
- 试验地点
- 1
- 主要终点
- Clinical-endoscopic remission rate at 12 weeks
研究概览
简要总结
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.
详细描述
This is a retrospective observational cohort study. All interventions described are part of routine clinical care for acute severe ulcerative colitis (ASUC). Treatment decisions were made by treating clinicians per standard of care; the investigator did not prospectively assign, modify, or control any therapeutic interventions. Participants did not receive any intervention specifically for the purpose of this study. The study only involves retrospective analysis of existing medical records to compare real-world outcomes between different treatment exposure patterns.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 16 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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;
- •Any other circumstances which, in the opinion of the investigator, would render the subject unfit for study inclusion.
研究组 & 干预措施
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.
干预措施: Upadacitinib (Drug)
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.
干预措施: Upadacitinib (Drug)
结局指标
主要结局
Clinical-endoscopic remission rate at 12 weeks
时间窗: 12 weeks after upadacitinib initiation
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.
Colectomy-free rate within 90 days
时间窗: 90 days after upadacitinib initiation
The proportion of ASUC patients who did not undergo colectomy within 90 days after the initiation of upadacitinib treatment
次要结局
- 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)
- Endoscopic remission rate at week 12(12 weeks after upadacitinib initiation)
- Adverse events (AEs) rate(From upadacitinib initiation to study completion)
研究者
Yongquan Shi
Professor
Xijing Hospital of Digestive Diseases