Study on Clinical and Pathological Features of Primary Biliary Cholangitis and Risk Factors Related to Disease Progression

NCT07733882 | Not Yet Recruiting

• 1 other identifier: ZZLX2026030
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 1

Brief Summary

Primary biliary cholangitis (PBC) is a chronic autoimmune intrahepatic cholestatic liver disease characterized by progressive, non-suppurative, destructive cholangitis, potentially leading to fibrosis, cirrhosis, and liver failure. It predominantly affects middle-aged and elderly women, with highly variable progression rates: some patients remain stable long-term, while others rapidly develop portal hypertension and decompensation. Early risk factor identification and accurate risk stratification are essential for improving prognosis. Large-scale, multi-dimensional (clinical-pathological-laboratory) studies on PBC progression risk factors in the Chinese population remain scarce. The associations of histological stage, autoantibody profiles, and biochemical response with prognosis require further clarification. This retrospective observational study will enroll PBC patients with histologically confirmed diagnosis via liver biopsy at Beijing Ditan Hospital, Capital Medical University, from January 2015 to June 2026. We will systematically analyze clinical, laboratory, autoantibody, and pathological features. Univariate and multivariate logistic/Cox regression will be used to identify independent risk factors, aiming to establish a progression risk prediction model tailored to Chinese PBC patients. This model will support early identification of high-risk individuals and guide personalized treatment and follow-up strategies in clinical practice.

Conditions

PBC

Keywords

Primary Biliary Cholangitis (PBC); Disease progression; Risk factors

Outcome Measures

Primary Outcomes (1)

• Disease progression

  Composite endpoint defined as occurrence of any of the following: (1) histological stage progression by at least 1 stage according to the Ludwig staging system on repeat liver biopsy; (2) decompensated cirrhosis events including ascites (confirmed by imaging), esophageal/gastric variceal bleeding (confirmed by endoscopy or clinically), or hepatic encephalopathy; (3) liver transplantation; or (4) liver-related death

  From date of diagnosis (baseline liver biopsy) to date of first endpoint event or last clinical follow-up, assessed up to 10 years

Study Arms (2)

Progressive Group

PBC patients who experienced disease progression during follow-up, defined as histological stage progression (≥1 stage by Ludwig system), decompensated cirrhosis events (ascites, variceal bleeding, or hepatic encephalopathy), liver transplantation, or liver-related death.

Non-progressive Group

PBC patients who did not experience any of the above events during follow-up and remained alive without liver transplantation at the last follow-up visit.

Eligibility Criteria

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

Study Population

All PBC patients diagnosed by liver biopsy at the Second Department of Hepatology, Beijing Ditan Hospital, Capital Medical University, between January 1, 2015, and June 31, 2026. All data were retrospectively collected from the hospital's HIS and LIS systems, including inpatient and outpatient medical records, laboratory reports, imaging reports, and pathological reports. This is a retrospective observational study with no additional interventions or biological sample collection.

You may qualify if:

• Met the diagnostic criteria for PBC according to the Guidelines for the Diagnosis and Treatment of Primary Biliary Cholangitis (2021) and the Guidelines for the Diagnosis and Treatment of Primary Biliary Cholangitis (2025 Edition), satisfying at least two of the following three criteria: ① biochemical evidence of cholestasis (elevated ALP and/or GGT) with imaging excluding extrahepatic or intrahepatic large bile duct obstruction; ② positive AMAs/AMA-M2 or positivity for other PBC-specific autoantibodies (anti-gp210 or anti-sp100); ③ histologic evidence of non-suppurative destructive cholangitis and small bile duct destruction. (2) Underwent liver biopsy with a complete pathological report. (3) Had complete clinical data with missing values for key variables not exceeding 20%.

You may not qualify if:

• Concomitant other liver diseases, such as chronic hepatitis B, hepatitis C, hepatitis D, hepatitis E, alcoholic liver disease, non-alcoholic fatty liver disease, drug-induced liver injury, autoimmune hepatitis, primary hemochromatosis, or Wilson's disease. (2) Concurrent non-hepatotropic viral infections causing liver injury, including Epstein-Barr virus, cytomegalovirus, and human immunodeficiency virus. (3) Concurrent liver malignancy. (4) Age \< 18 years. (5) Severely incomplete clinical data with missing values for key variables exceeding 20%.

Sponsors & Collaborators

• Beijing Municipal Administration of Hospitals: lead

Study Sites (1)

Beijing Ditan Hospital, Capital Medical University

Beijing, Beijing Municipality, 100015, China

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary; Disease Progression

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Disease Attributes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Department

Study Record Dates

• First Submitted: July 24, 2026
• First Posted: July 29, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: July 29, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)