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OC-030 Effective Stratification Of Hepatocellular Carcinoma Risk In Primary Biliary Cirrhosis: Results Of A Multi-centre International Study
  1. PJ Trivedi1,
  2. W Lammers2,
  3. H van Buuren2,
  4. H Janssen3,
  5. P Invernizzi4,
  6. PM Battezzati5,
  7. A Floreani6,
  8. A Pares7,
  9. C Ponsioen8,
  10. C Corpechot9,
  11. R Poupon10,
  12. M Mayo11,
  13. J Talwalkar12,
  14. A Burroughs13,
  15. F Nevens14,
  16. A Mason15,
  17. T Bruns16,
  18. K-K Li1,
  19. K Kowdley17,
  20. T Kumagi18,
  21. A Cheung18,
  22. A Lleo19,
  23. N Cazagon20,
  24. I Franceschet21,
  25. L Caballería22,
  26. K Boonstra23,
  27. E de Vries24,
  28. M Imam12,
  29. G Pieri13,
  30. P Kanwar25,
  31. K Lindor12,26,
  32. B Hansen2,
  33. G Hirschfield1
  34. on behalf of Global PBC Study Group
  1. 1NIHR Biomedical Research Unit and Centre for Liver Research, University of Birmingham, Birmingham, UK
  2. 2Department of Gastroenterology and Hepatology, Erasmus Medical Centre, Rotterdam, The Netherlands
  3. 3Liver Clinic, Toronto Western and General Hospital, University Health Network, Toronto, Canada
  4. 4Liver Unit and Center for Autoimmune Liver Diseases, Humanitas Clinical and Research Center, Rozzano, Italy
  5. 5Department of Health Sciences, Università Degli Studi Di Milano, Milano, Italy
  6. 6Department of Surgical, Oncological and Gastroenterological, University of Padua, Padua, Italy
  7. 7Liver Unit, Hospital Clínic, CIBERehd, IDIBAPS, University of Barcelona, Barcelona, Spain
  8. 8Department of Gastroenterology and Hepatology, Academic Medical Center, Amsterdam, The Netherlands
  9. 9Centre de Référence Des Maladies Inflammatoires Des Voies Biliaires, Hôpital Saint-Antoine, APHP, Paris, France
  10. 10Centre de Référence Des Maladies Inflammatoires Des Voies Biliaires, Hôpital Saint-Antoine, Paris, France
  11. 11Digestive and Liver Diseases, UT Southwestern Medical Center, Dallas, USA
  12. 12Department Gastroenterology and Hepatology, Mayo Clinic, Rochester, USA
  13. 13The Sheila Sherlock Liver Centre, The Royal Free Hospital, London, UK
  14. 14Department of Hepatology, University Hospitals Leuven, Leuven, Belgium
  15. 15Divison of Gastroenterology and Hepatology, University of Alberta, Edmonton, Canada
  16. 16Department of Internal Medicine IV, Jena University Hospital, Friedrich Schiller University Jena, Jena, Germany
  17. 17Liver Center of Excellence, Digestive Disease Institute, Virginia Mason Medical Center, Seattle, USA
  18. 18Liver Clinic, Toronto Western and General Hospital, Toronto, Canada
  19. 19Liver Unit and Center for Autoimmune Liver Diseases, Humanitas Clinical and Research Center, Rozzano, Italy
  20. 20Dipartimento Di Scienze Chirurgiche Oncologiche E Gastroenterologiche, Università Degli Studi Di Milano, Milano, Italy
  21. 21Department of Surgical, Oncological and Gastroenterological, University of Padua, Padua, Padua, Italy
  22. 22Primary Healthcare Centre Premià de Mar, Catalan Health Institute, Premià de Mar, Spain
  23. 23Department of Gastroenterology and Hepatology, Academic Medical Center, Amsterdam, Netherlands
  24. 24Department of Gastroenterology and Hepatology, Academic Medical Center, Amsterdam, Netherlands
  25. 25Liver Center of Excellence, Digestive Disease Institute, Seattle, USA
  26. 26Arizona State University, Phoenix, USA

Abstract

Introduction Hepatocellular carcinoma (HCC) is an important but infrequent outcome in primary biliary cirrhosis (PBC). Improved risk evaluation is an important goal for stratified surveillance.

Methods Risk-factor analysis of the ‘Global PBC Study Group’ comprising 15 centres across North America and Europe spanning >40-years follow-up was performed using Cox proportional hazards model, logistic regression and Kaplan-Meier estimates (SPSSv21).

Results Of 3546 patients with PBC (med. follow-up 8.6 yrs; IQR 4.4–14.1), 131 developed HCC. Excluding those who developed HCC within 12 months of PBC diagnosis (n = 23), median time to HCC was 12.7 yrs (6.9–16.8) and subsequent survival 1.1 yrs. (0.2–2.7). At diagnosis, factors associated with HCC development were male gender (adj. HR: 4.4;1.4–12.2, p = 0.014) and thrombocytopenia (adj. HR: 4.5;1.4–14.8, p = 0.012). Use of ursodeoxycholic acid per-se was not associated with future risk of HCC, but stratification of risk by biochemical response at 12 months was effective by Rotterdam (adj. HR: 8.9;2.1–37.3, P = 0.003), Paris-I (adj. HR: 7.6;2.0–29.0, p = 0.003) or Toronto criteria (HR: 5.6;1.6–18.8, P = 0.006). Five (4.6 vs. 0.2%) and 10-year (13%vs.1.9%) HCC incidence was significantly increased for biochemical non-responders (p = 2.2 × 10–9), and by multivariate analysis non-response remained the only significant risk factor.

Conclusion Our uniquely powered cohort allows robust demonstration that 12-month biochemical non-response is associated with an increased risk of developing HCC in PBC. Routine surveillance in those achieving biochemical response is unlikely cost-effective.

Disclosure of Interest None Declared.

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