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

Brief summary (registry, in English)

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.

What you commit to

• Study type: Observational
• Planned participants: 500
• From 1 May 2023 to the primary completion date, 31 Dec 2023
• 1 site in 1 country
• Visits and tests: ask the trial team, the registry does not list them

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Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

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