Upadacitinib for Refractory IBD in Asian Children and Elderly: Age-Stratified Analysis (UPA-AGE)

Age-Stratified Efficacy of Upadacitinib in Refractory Inflammatory Bowel Disease Among Asian Populations: Pediatric Crohn's Disease Versus Elderly Ulcerative Colitis Cohort Analysis

This single-center, retrospective study aims to evaluate the effectiveness and safety of upadacitinib (UPA) in Asian pediatric patients with refractory Crohn's disease (CD) and elderly patients with refractory ulcerative colitis (UC). The study will analyze data from 21 patients (11 children aged 9-17 with CD and 10 adults aged 60 and older with UC) treated at the Sixth Affiliated Hospital of Sun Yat-sen University between January 2023 and December 2024. The primary objective is to assess the clinical remission rate and endoscopic response rate in both groups. Secondary objectives include comparing treatment outcomes between the two age groups, exploring the impact of immunosenescence on UPA efficacy in elderly UC patients, and analyzing the correlation between laboratory markers (CRP, albumin) and clinical outcomes. This study is the first in Asia to investigate the age-stratified efficacy of upadacitinib in pediatric CD and elderly UC patients. The findings will provide crucial real-world evidence to inform individualized treatment strategies for these specific populations. Patients included in this study will have previously failed at least two biological therapies. Data collected will include demographics, disease characteristics, prior treatment history, UPA dosage and duration, clinical and endoscopic scores (PCDAI/Mayo, SES-CD/UCEIS), imaging results, laboratory values, and adverse events. The study is registered on ClinicalTrials.gov (NCT06274996). Ethical considerations will be addressed through data anonymization and a waiver of informed consent due to the retrospective nature of the study.

Study Overview

• Status: Completed
• Conditions: Inflammatory Bowel Disease (IBD); Pediatric Crohn's Disease; Ulcerative Colitis (UC); Crohn's Disease (CD); Refractory Crohn's Disease; Refractory Ulcerative Colitis; Geriatric Ulcerative Colitis
• Intervention / Treatment: Drug: Upadacitinib

• Study Type: Observational
• Enrollment (Actual): 21

Contacts and Locations

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 515000
      • The Sixth Affiliated Hospital of Sun Yat-Sen University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Asian pediatric patients (ages 9-17) with refractory Crohn's disease and elderly patients (60 years and older) with refractory ulcerative colitis.

Description

Inclusion Criteria:

• Diagnosis of IBD according to the "Consensus on Diagnosis and Treatment of Inflammatory Bowel Disease (2023)"; Failure of at least two prior biological therapies (anti-TNF/anti-integrin/IL-12/23 inhibitors); Complete baseline and follow-up data (at least 12 weeks))

Exclusion Criteria:

• Active infection, malignancy, severe cardiovascular disease, or hepatic/renal insufficiency; Pregnancy or breastfeeding; Use of other JAK inhibitors within 30 days prior to baseline)

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pediatric Crohn's Disease (CD) Group; Patients aged 9-17 years diagnosed with refractory Crohn's disease who received upadacitinib treatment.	Drug: Upadacitinib; A selective Janus kinase (JAK) 1 inhibitor used for the treatment of refractory inflammatory bowel disease.
Elderly Ulcerative Colitis (UC) Group; Patients aged 60 years or older diagnosed with refractory ulcerative colitis who received upadacitinib treatment.	Drug: Upadacitinib; A selective Janus kinase (JAK) 1 inhibitor used for the treatment of refractory inflammatory bowel disease.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Remission Rate at Week 12	he proportion of patients achieving clinical remission at 12 weeks, as defined by [Specify the definition of clinical remission, e.g., "a Mayo score ≤ 2 with no individual subscore > 1" for UC or "a PCDAI score < 10" for CD].	Week 12
Endoscopic Response Rate at Week 12	The proportion of patients achieving endoscopic response at 12 weeks, as defined by [Specify the definition of endoscopic response, e.g., "a decrease of ≥50% from baseline in the SES-CD score" for CD or "a decrease of ≥ 1 point from baseline in the UCEIS" for UC].	Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Response Rate at Week 12	The proportion of patients achieving clinical response at 12 weeks, as defined by [Specify the definition of clinical response, e.g., "a decrease of ≥ 3 points from baseline in the Mayo score" for UC or "a decrease of ≥ 20 points from baseline in the PCDAI score" for CD].	Week 12
Change in CRP from Baseline to Week 12	The change in C-reactive protein (CRP) levels from baseline to 12 weeks after treatment initiation.	Baseline to Week 12

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events	The number and type of adverse events occurring during the 12-week treatment period.	Week 0 to Week 12

Collaborators and Investigators

• Sponsor: Sixth Affiliated Hospital, Sun Yat-sen University

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2023
• Primary Completion (Actual): December 31, 2024
• Study Completion (Actual): March 1, 2025

Study Registration Dates

• First Submitted: April 3, 2025
• First Submitted That Met QC Criteria: April 3, 2025
• First Posted (Actual): April 10, 2025

Study Record Updates

• Last Update Posted (Actual): April 10, 2025
• Last Update Submitted That Met QC Criteria: April 3, 2025
• Last Verified: April 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Gastroenteritis; Colitis; Colitis, Ulcerative; Ulcer; Intestinal Diseases; Inflammatory Bowel Diseases; Janus Kinase Inhibitors; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antirheumatic Agents; Protein Kinase Inhibitors; Upadacitinib
• Other Study ID Numbers: 2025ZSLYEC-149

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No