Detection Program for Patients With Primary Biliary Cholangitis Lost in the System

NCT ID: NCT05919433

Last Updated: 2023-06-26

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 500 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-05-01
• Study Completion Date: 2028-12-31

Brief Summary

Primary biliary cholangitis (PBC) has been considered a rare disease and its management has been limited by the lack of therapeutic alternatives. PBC is a slowly progressing chronic liver disease characterized by an immune-mediated destruction of the intrahepatic bile ducts, which leads to cholestasis, portal inflammation, and ultimately liver cirrhosis and its associated complications (ascites, portal hypertension, etc), if not treated effectively. Thus, early diagnosis and close management of these patients with PBC is essential. First-line treatment with ursodeoxycholic acid (UDCA) improves liver biochemical parameters, delays histological progression, and increases liver transplant-free survival and overall survival. However, up to 40% of patients are non-responders to UDCA. Obeticholic acid (OCA) is recommended as second-line therapy in combination with UDCA for patients with an inadequate response to UDCA or as monotherapy in cases of UDCA intolerance.

According to current clinical guidelines, the diagnosis of PBC includes a combination of elevated alkaline phosphatase (ALP) levels and the presence of anti-mitochondrial antibodies (AMA) (titer >1:40) and/or anti-nuclear antibodies (ANA) anti-gp210 or anti-sp100. AMA are highly sensitive and specific for PBC and are detected in nearly 95% of PBC patients. A liver biopsy is not necessary unless there is an elevation of ALP without the presence of specific AMA and/or anti-gp210 or anti-sp100 ANA or if coexistence with other liver diseases is suspected (autoimmune hepatitis, hepatic steatosis).

The incidence of PBC has increased in recent years due to an increase in the diagnosis of cases in the initial phases, better awareness in the medical community and the development of more sensitive diagnostic tests. However, up to 31% of patients with PBC are lost without follow-up. The correct identification of patients with PBC is essential so that they can benefit from an adequate treatment and modify disease progression. To date, two studies (one Spanish and one Portuguese) showed that 27% and 45.5% of the patients lost with PBC presented advanced fibrosis, respectively.

The objective of this study is to identify, through computerized data, patients with PBC who may be lost in the system and evaluate their clinical, analytical and demographic characteristics, and in a second phase, provide access to follow-up in specialized consultations.

Conditions

Primary Biliary Cholangitis

Study Design

• Observational Model Type: CASE_ONLY
• Study Time Perspective: OTHER

Study Groups

Patients with positive AMA and/or ANA anti-gp210/sp100 identified in the hospital database

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Patients with positive AMA and/or with positive ANA anti-gp210 or anti-sp100 identified in the computer databases of each of the participating centers.
• Adults (≥18 years)

Exclusion Criteria

• Patients with Overlap Syndrome (PBC overlap with Autoimmune Hepatitis (AIH))

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hospital Mutua de Terrassa

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hospital Universitari MútuaTerrassa

Terrassa, Barcelona, Spain

Site Status: RECRUITING

Countries

Spain

Central Contacts

Diana Horta, MD, PhD

Role: CONTACT

diana.horta.s@gmail.com

Facility Contacts

Diana Horta, MD, PhD

Role: primary

diana.horta.s@gmail.com

References

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Reference Type: BACKGROUND

PMID: 26364546 (View on PubMed)

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Reference Type: BACKGROUND

PMID: 16177252 (View on PubMed)

European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. J Hepatol. 2017 Jul;67(1):145-172. doi: 10.1016/j.jhep.2017.03.022. Epub 2017 Apr 18.

Reference Type: BACKGROUND

PMID: 28427765 (View on PubMed)

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Reference Type: BACKGROUND

PMID: 35152430 (View on PubMed)

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Reference Type: BACKGROUND

PMID: 34663072 (View on PubMed)

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Reference Type: BACKGROUND

PMID: 36270719 (View on PubMed)

Olveira-Martin A, Yebra-Carmona J, Amaral-Gonzalez C, Tejedor M, Eiras P, Hernandez-Perez M, Suarez-Cabredo C, Spigarelli-de Rabago I, Suarez-Ferrer C, Morales-Arraez D, Chico I, Diaz-Flores F, Rodriguez R, Llorente S, Molina-Perez E, Hernandez-Guerra de Aguilar MN. Retrieval and treatment of patients with primary biliary cholangitis who are lost in the health system. Rev Esp Enferm Dig. 2021 Nov;113(11):776-779. doi: 10.17235/reed.2021.8174/2021.

Reference Type: BACKGROUND

PMID: 34470449 (View on PubMed)

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Reference Type: BACKGROUND

PMID: 34402472 (View on PubMed)

Other Identifiers

RESCAT

Identifier Type: -

Identifier Source: org_study_id