Author information

• ¹Toronto Centre for Liver Disease, Toronto General Hospital, University Health Network, Toronto, Ontario, Canada.
• ²Gastroenterology and Hepatology, Erasmus University Medical Center, Rotterdam, the Netherlands.
• ³Division of Gastroenterology and Center for Autoimmune Liver Diseases, Department of Medicine and Surgery, University of Milano-Bicocca, San Gerardo Hospital, Monza, Italy.
• ⁴European Reference Network on Hepatological Diseases (ERN RARE-LIVER).
• ⁵National Institute for Health Research Birmingham Biomedical Research Centre and Centre for Liver and Gastrointestinal Research, Institute of Immunology and Immunotherapy, University of Birmingham, Birmingham, UK.
• ⁶Reference Center for Inflammatory Biliary Diseases and Autoimmune Hepatitis, Saint-Antoine Hospital, APHP, Sorbonne University, Paris, France.
• ⁷Department of Health Sciences, Universita[Combining Grave Accent] degli Studi di Milano, Milan.
• ⁸Department of Surgery, Oncology and Gastroenterology, University of Padua, Padua, Italy.
• ⁹Liver Unit, Hospital Cli[Combining Acute Accent]nic, CIBERehd, IDIBAPS, University of Barcelona, Barcelona, Spain.
• ¹⁰Department of Hepatology, University Hospitals Leuven, KU Leuven, Leuven, Belgium.
• ¹¹Division of Internal Medicine and Hepatology, Humanitas Clinical Research Center IRCSS, Humanitas University, Rozzano, Milan, Italy.
• ¹²Digestive and Liver Diseases Clinic, UT Southwestern Medical Center, Dallas, Texas.
• ¹³Liver Care Network and Organ Care Research, Swedish Medical Center, Seattle, Washington, USA.
• ¹⁴Department of Gastroenterology and Hepatology, Academic Medical Center, Amsterdam, the Netherlands.
• ¹⁵Department of Medicine and Research Laboratory of Internal Medicine, National Expertise Center of Greece in Autoimmune Liver Diseases, General University Hospital of Larissa, Larissa, Greece.
• ¹⁶The Sheila Sherlock Liver Centre, The Royal Free Hospital, London, UK.
• ¹⁷Divison of Gastroenterology and Hepatology, University of Alberta, Edmonton, Alberta, Canada.
• ¹⁸Department of Gastroenterology and Hepatology, Ghent University Hospital, Ghent, Belgium.
• ¹⁹Department of Internal Medicine IV, Jena University Hospital, Friedrich Schiller University, Jena, Department of Internal Medicine III, University Hospital RWTH Aachen, Aachen, Germany.
• ²⁰College of Health Solutions, Arizona State University, Phoenix, Arizona, USA.
• ²¹Institute of Health Policy, Management and Evaluation, University of Toronto, Toronto, Ontario, Canada.

Abstract

Background: Opportunity to redefine the care journeys for those living with primary biliary cholangitis (PBC) includes facilitating access to enhanced (PBC-dedicated) programmes by nonspecialist risk 'flagging' of patients.

Objective: To develop a nonexpert PBC stratification tool to help care pathway choices (standard vs. enhanced) choices in PBC.

Methods: We included ursodeoxycholic acid-treated patients with PBC from the Global PBC Study Group. The performance of baseline and 1-year clinical markers with transplant-free survival was assessed to develop the 'ABA' tool using Age (A), Bilirubin (B), and Alkaline phosphatase (A). Added value of fibrosis estimation was assessed.

Results: 'ABA' classification mapped three risk groups (n = 2226): low [Age > 50 years, bilirubin ≤ 1 × ULN, alkaline phosphatase (ALP) ≤ 3 × ULN], high (Age ≤ 50 years, bilirubin > 1 × ULN, ALP > 3 × ULN), and intermediate (other). Transplant-free survival at 10 years in the low-, intermediate-, and high-risk groups were 89, 77, and 59% at baseline and 86, 76, and 40% at 1 year, respectively. We propose that high-risk patients at baseline be directly triaged to enhanced (PBC-dedicated) care and the remaining be reassessed at 1 year. Modelling showed after 1 year 46% patients were proposed to enhanced care and 54% to standard care. The 'ABA' mapped pathways facilitated identification of patients at risk based on a young age, as compared to traditional liver biochemical stratification. In patients proposed to standard care, estimated fibrosis stage had ongoing prognostic value.

Conclusion: Nonspecialist use of the 'ABA' risk tool could prioritize care journey choices for patients with PBC.