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Multicenter Study
. 2025 Dec;13(10):1955-1963.
doi: 10.1002/ueg2.70134. Epub 2025 Oct 22.

Trends in Primary Biliary Cholangitis: Prospective Cohort Study From the European Reference Network Registry (R-LIVER)

A Gerussi  1   2 , E Nofit  1   2 , D P Bernasconi  2 , C Kroll  3   4 , F Groß  3   4 , I Schregel  3   4 , A Marini  2 , L Cristoferi  1 , F Malinverno  1 , M Papp  4   5 , G Dalekos  4   6 , E Rigopoulou  4   6 , M K Janik  4   7 , P Milkiewitz  4   7 , H Ytting  4   8   9 , A Leburgue  4   10 , F Braun  3   4 , C Casar  4   11 , M Sebode  3   4 , A Lohse  3   4 , B Hansen  12 , M Carbone  1   13 , C Schramm  3   4   11 , P Invernizzi  1   2
Affiliations
Multicenter Study

Trends in Primary Biliary Cholangitis: Prospective Cohort Study From the European Reference Network Registry (R-LIVER)

A Gerussi et al. United European Gastroenterol J. 2025 Dec.

Abstract

Background and aims: The European Reference Network on Hepatological Diseases (ERN RARE-LIVER) is a Europe-wide network for centers of excellence in the management of rare liver diseases. We aimed to evaluate the current diagnostic and therapeutic trends of primary biliary cholangitis (PBC).

Methods: Prospective data of PBC cases diagnosed from 2017 to March 2024 were extracted from the R-LIVER registry of ERN-RARE LIVER. Cases without two follow-ups within 24 months were excluded from the treatment analysis. Biochemical response according to Toronto criteria and normalization of alkaline phosphatase (ALP) values after 12 months of Ursodeoxycholic Acid (UDCA) were evaluated.

Results: This study included 327 incident cases from six centers. Median age was 56 years, 89.3% were female. At the time of diagnosis, median values of ALP were 1.37 x ULN, and median bilirubin was 0.49 x ULN. Transient elastography (TE) was performed in 230 patients (70.3%) at baseline; median liver stiffness was 6.2 kPa. Out of 316 subjects, treatment with UDCA was started in 312 patients (98.7%); 246 (85.1%) achieved ALP values < 1.67 x ULN at 12 months. Normalization of ALP values occurred in 143 subjects (49.5%) at 12 months. Among 43 patients with inadequate response, 18 (41.9%) were treated with second-line therapies, and had worse liver biochemistry at baseline.

Conclusion: In the current era, patients with PBC are diagnosed at an early stage using non-invasive methods and are almost all treated with UDCA. The biochemical response rate is 85.1%, but the use of second-line therapies for inadequate responders remains suboptimal.

Keywords: UDCA; autoimmunity; cholestasis; epidemiology; liver.

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Conflict of interest statement

G.D. has received research grants from Gilead and Ipsen, has served as advisor and/or lecturer for Ipsen, Gilead, Genesis, Pfizer, Sanofi, and Sobi and as PI for Amyndas Pharmaceuticals, Intercept Pharma, CymaBay Therapeutics, Genkyotex, Novo Nordisk, Pfizer, Regulus Therapeutics, Sobi, and Tiziana Life Sciences. C.S. has received lecture fees and travel grants from Falk, and consulting fees from Pliant, Chemomab, Agomab and Moonlake. M.S. has received lecture fees and travel grants from Falk. M.C. has received consulting fees from Advanz, Alentis, Albireo/Ipsen, Alfasigma, Amgen, Calliditas, Echosens, Genetic spa, GSK, Intercept, Kowa, Mayoly, Mirum, Perspectum, Zydus, and is on the scientific advisory board of Advanz/Intercept, Cymabay/Gilead, Falk, GSK, Kowa, Ipsen, Mirum, Moderna. P.I. is on the scientific advisory board of Ipsen, Gilead, Zydus, Mirum, Advanz. AWL has received lecture fees from Falk, and consulting fees from Servier and Kezar Biosciences. A.G. has received lecture fees from Ipsen and Advanz, has received consulting fees from Ipsen, Gilead, Astrazeneca, Signanthealth, CAMP4 therapeutics and has received travel grants from Ipsen, Advanz and Zydus, has received unrestricted grant support from Ipsen and is in the scientific advisory board of Ipsen. L.C. has received lecture fees from Ipsen and Advanz, has received consulting fees from Falk, Amgen, Ipsen, Signanthealth, is in the scientific advisory board of Falk, Amgen, Ipsen and has received travel grant from Ipsen.

Figures

FIGURE 1
FIGURE 1
Flowchart of study design and participant selection. After excluding patients without adequate follow‐up, those suspected of variant syndrome due to concomitant use of immunosuppressive drugs for liver disease and centers contributing less than five cases, 327 patients were included in the analysis on diagnostic trends (baseline factors). Eleven patients died during the interval of time under study and were excluded from the analysis of therapeutic trends.
FIGURE 2
FIGURE 2
Sankey plot of therapeutic changes from diagnosis to 12‐month and 24‐month follow‐ups. Beza, bezafibrate; Feno, fenofibrate, OCA, obeticholic acid, UDCA, ursodeoxycholic acid.
See this image and copyright information in PMC

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