PBC Long-term Outcomes Study
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Primary Biliary Cholangitis (PBC), Long-term Outcomes, Prognosis, Multicenter Prospective Study. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multi-center Prospective-retrospective Study of the Clinical Characteristics, Biochemical Response, and Long-term Outcomes in Patients With Primary Biliary Cholangitis
Overview
Primary biliary cholangitis (PBC) is an autoimmune liver disease predominantly affecting middle-aged women. While historically it was deemed rare, advancements in specific auto-antibody tests have led to increased recognition of PBC. The long-term survival of PBC patients in China is not yet fully understood. Several studies have investigated the prognosis of PBC in China. While these studies provide valuable insights into the disease characteristics and prognostic factors of PBC in China, they are all single-center studies with limitations. They lack consideration of the impact of symptoms, varying disease stages, and second-line treatments on prognosis. Therefore, there is a pressing need for multicenter and large-scale studies to further elucidate the characteristics and long-term survival of PBC patients in China. Biochemical response to ursodeoxycholic acid (UDCA) is an independent factor associated with long-term survival. Unfortunately, approximately 30-40% of PBC patients demonstrate insufficient biochemical response to UDCA. In our cohort, we reported a higher nonresponse rate, with about 44% according to the Paris I criteria. These patients remain at risk for disease progression to advanced stages and may benefit from additional second-line drug therapies. With significant advancements in the development of new drugs for PBC, a comprehensive understanding of patients with suboptimal responses to UDCA-including long-term prognosis, the distribution of different disease stages, and the prevalence of pruritus-will provide a basis for individualized treatment strategies. Additionally, fatigue and pruritus are prevalent symptoms for PBC patients and fluctuates independently of disease activity or stage, which significantly diminishing health-related quality of life. However, the incidence and impact of fatigue and pruritus on long-term outcomes for PBC patients in China need further clarification. Therefore, we would like to conduct this multicenter study to estimate the prevalence of PBC in China and to evaluate the symptomatic burden, treatment, and long-term outcomes among PBC patients in China.
Detailed description
This is a retrospective-prospective, multicenter cohort study that plans to enroll approximately 4,000 patients with PBC from ten centers across multiple provinces and cities nationwide of China.
Primary outcome measures
- death and/or liver transplantation. [Time frame: From January 2000 to December 2029]
Secondary outcome measures (1)
- Liver-related adverse events [Time frame: From January 2000 to December 2025]
Eligibility criteria
Inclusion criteria
- Diagnosis consistent with PBC.
- Willingness to participate, with informed consent obtained from the patient or legal representative (if the patient is deceased or cognitively impaired), and ability to adhere to follow-up.
Exclusion criteria
- Co-existing chronic hepatitis B or C, drug-induced liver injury, autoimmune hepatitis, primary sclerosing cholangitis, or hereditary/metabolic liver diseases.
- Co-existingMalignancy or severe cardiac, pulmonary, renal, cerebral, or hematologic disorders expected to substantially shorten life expectancy.
- Incomplete baseline medical records or laboratory data.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
China · 1 center
- Liver Research Center, Beijing Friendship Hospital, Capital Medical University — Beijing
Publications
- Chen S, Li MQ, Duan WJ, Li BE, Li SX, Lv TT, Ma L, Jia JD. Concomitant extrahepatic autoimmune diseases do not compromise the long-term outcomes of primary biliary cholangitis. Hepatobiliary Pancreat Dis Int. 2022 Dec;21(6):577-582. doi: 10.1016/j.hbpd.2022.05.009. Epub 2022 May 24. PMID 35668014
- Zeng N, Duan W, Chen S, Wu S, Ma H, Ou X, You H, Kong Y, Jia J. Epidemiology and clinical course of primary biliary cholangitis in the Asia-Pacific region: a systematic review and meta-analysis. Hepatol Int. 2019 Nov;13(6):788-799. doi: 10.1007/s12072-019-09984-x. Epub 2019 Sep 25. PMID 31552558
- You H, Ma X, Efe C, Wang G, Jeong SH, Abe K, Duan W, Chen S, Kong Y, Zhang D, Wei L, Wang FS, Lin HC, Yang JM, Tanwandee T, Gani RA, Payawal DA, Sharma BC, Hou J, Yokosuka O, Dokmeci AK, Crawford D, Kao JH, Piratvisuth T, Suh DJ, Lesmana LA, Sollano J, Lau G, Sarin SK, Omata M, Tanaka A, Jia J. APASL clinical practice guidance: the diagnosis and management of patients with primary biliary cholangi PMID 35119627
Identifiers
NCT: NCT07149675 · 2025-P2-173-01