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Z Gastroenterol 2025; 63(12): 1260-1267
DOI: 10.1055/a-2721-1160
Original Manuscript

Clinical characteristics of newly diagnosed patients with primary biliary cholangitis (PBC) indicate the need for better awareness on timely diagnosis and adequate UDCA therapy

Klinische Charakteristika neu diagnostizierter Patienten mit Primär Biliärer Cholangitis weisen auf die Notwendigkeit einer frühzeitigeren Diagnose und adäquaten UDCA Behandlung hin

Authors

  • Kerstin Stein‡

    1   Practice of Hepatology Magdeburg, Magdeburg, Germany

  • Wolf P. Hofmann‡

    2   Gastroenterology at Bayerischer Platz, Berlin, Germany

  • Annegret Franke

    3   Clinical Trial Centre of Medical Faculty, University of Leipzig, Leipzig, Germany

  • David Petroff

    3   Clinical Trial Centre of Medical Faculty, University of Leipzig, Leipzig, Germany

  • Tobias Müller

    4   Department of Hepatology and Gastroenterology, Campus Virchow-Klinikum (CVK) and Campus Charité Mitte (CCM), Charité – Universitätsmedizin Berlin, Berlin, Germany (Ringgold ID: RIN14903)

  • Heike Bantel

    5   Department of Gastroenterology, Hepatology, Infectious Diseases and Endocrinology, Hannover Medical School, Hannover, Germany (Ringgold ID: RIN9177)

  • Rainer Günther

    6   Division of Hepatology/Transplant Hepatology, Department of Medicine I, Universitätsklinikum Schleswig-Holstein, Kiel, Germany (Ringgold ID: RIN54186)

  • Gerald Denk

    7   Department of Medicine II and Transplantation Center, University Hospital, LMU Munich, Munich, Germany (Ringgold ID: RIN54187)

  • Philipp A. Reuken 

    8   Department of Internal Medicine IV (Gastroenterology, Hepatology, Infectious Diseases), University Hospital Jena, Jena, Germany (Ringgold ID: RIN39065)

  • Jörn M. Schattenberg

    9   Department of Internal Medicine II, Saarland University Medical Center, Homburg, Germany (Ringgold ID: RIN39072)
    10   Department of Medicine I, University Medical Centre of the Johannes Gutenberg University Mainz, Mainz, Germany (Ringgold ID: RIN39068)

  • Uwe Naumann

    11   UBN Private Practice, Berlin, Germany

  • Tobias Böttler

    12   Department of Medicine II, University Hospital Freiburg, Freiburg, Germany (Ringgold ID: RIN570474)

  • Andreas Weber

    13   Department of Internal Medicine 6 Gastroenterology, Hepatology, Endocrinology, Paracelsus Medical University, Hospital Nuremberg, Nuremberg, Germany (Ringgold ID: RIN470426)

  • Stefan Zeuzem

    14   Department of Medicine, Goethe University, University Hospital, Frankfurt, Germany (Ringgold ID: RIN9173)

  • Matthias Hinz

    15   Private Practice for Gastroenterology Herne, Herne, Germany

  • Robin Greinert

    16   Department of Gastroenterology, University Hospital Halle, Halle, Germany

  • Christoph Berg

    17   Department of Internal Medicine I, University of Tübingen, Tübingen, Germany (Ringgold ID: RIN27203)

  • Thaddäus Till Wissniowski

    18   Department of Internal Medicine II, Klinikum Chemnitz gGmbH, Chemnitz, Germany (Ringgold ID: RIN14902)

  • Karl-Georg Simon

    19   Practice for Gastroenterology, Leverkusen, Germany

  • Jonel Trebicka 

    20   Department of Internal Medicine B, University of Münster, Münster, Germany (Ringgold ID: RIN98883)

  • Rüdiger Behrens

    21   Private Practice Halle, Halle, Germany

  • Harald Grümmer

    22   Practice of Internal Medicine/Gastroenterology, Potsdam, Germany

  • Nektarios Dikopoulos

    23   Practice for Gastroenterology & Internal Medicine Ludwig, Dornstadt, Germany

  • Christoph Sarrazin

    24   Department of Internal Medicine II, St. Josef Hospital, Wiesbaden, Germany (Ringgold ID: RIN72204)

  • Elke Roeb

    25   Gastroenterology, University Hospital and Justus Liebig University Giessen, Giessen, Germany (Ringgold ID: RIN9175)

  • Andreas E. Kremer 

    26   Department of Medicine 1, University Hospital Erlangen, Erlangen, Germany
    27   Department of Gastroenterology and Hepatology, University Hospital Zürich, University of Zurich, Zurich, Switzerland (Ringgold ID: RIN27217)

  • Marion Muche

    28   Department of Gastroenterology, Infectious Diseases, and Rheumatology (Campus Benjamin Franklin), Charité – Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt-Universität zu Berlin, Berlin, Germany

  • Marc Ringelhan

    29   TUM School of Medicine and Health - Clinical Department of Internal Medicine II, TUM University Hospital, Munich, Germany

  • Andreas Teufel

    30   Department of Medicine II, Division of Hepatology, Division of Bioinformatics, Medical Faculty Mannheim, Heidelberg University, Mannheim, Germany

  • Uta Merle

    31   University Hospital Heidelberg, Dept. of Internal Medicine IV, University of Heidelberg, Heidelberg, Germany

  • Verena Keitel

    32   University Hospital Magdeburg, Otto-von Guericke University Magdeburg, Magdeburg, Germany

  • Jens U. Marquardt

    33   First Department of Medicine, Campus Lübeck, University Hospital Schleswig-Holstein, Lübeck, Germany

  • Achim Kautz

    34   Kautz gUG, Köln, Germany

  • Frank Tacke

    4   Department of Hepatology and Gastroenterology, Campus Virchow-Klinikum (CVK) and Campus Charité Mitte (CCM), Charité – Universitätsmedizin Berlin, Berlin, Germany (Ringgold ID: RIN14903)

  • Katja Piotrowski

    3   Clinical Trial Centre of Medical Faculty, University of Leipzig, Leipzig, Germany

  • Nicole Köppe-Bauernfeind

    3   Clinical Trial Centre of Medical Faculty, University of Leipzig, Leipzig, Germany

  • Christian Trautwein

    35   Leibniz-Institut für Arbeitsforschung an der TU Dortmund, Dortmund, Germany

  • Thomas Berg

    36   Division of Hepatology, Department of Medicine II, Leipzig University Medical Centre, Leipzig, Germany (Ringgold ID: RIN9180)

  • Johannes Wiegand 

    36   Division of Hepatology, Department of Medicine II, Leipzig University Medical Centre, Leipzig, Germany (Ringgold ID: RIN9180)

Supported by: Advanz Pharma The grant was dedicated to T.B.
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Abstract

Background

Guidelines on primary biliary cholangitis (PBC) recommend therapy with 13–15 mg/kg ursodeoxycholic acid (UDCA) and assessment of treatment response after 12 months. We evaluated to which extent these recommendations are followed in newly diagnosed patients.

Methods

The German PBC Registry recruited three subgroups: Adequate or inadequate UDCA treatment responders (Paris II criteria) and newly diagnosed patients (<6 months prior to recruitment). We focus on newly diagnosed patients with UDCA monotherapy.

Results

82 patients were recruited (43 at 12 tertiary and 39 at 9 secondary centers) thereof 22% with cirrhosis. Individuals with cirrhosis were older (71 ± 9 vs. 55 ± 14 years, p<0.001) and presented more frequently with diabetes mellitus (44% vs. 13%, p=0.0054) and arterial hypertension (78% vs. 42%, p=0.0076) compared to cases without cirrhosis.

12 months follow-up data were available in 62 patients. UDCA underdosing (<13 mg/kg/d) occurred in 47% and 74% of cases (p=0.013) at tertiary and secondary care at treatment initiation and in 29% and 73% (p=0.002) after 12 months, respectively. Paris II criteria were achieved in 74% and a deep UDCA response (alkaline phosphatase < ULN and bilirubin < 0.6 × ULN) in 32% of cases.

Conclusion

Newly diagnosed PBC patients include a substantial proportion of late presenters with cirrhosis. UDCA dosage is suboptimal in many cases. Time point of diagnosis and UDCA dosage should be improved.

Zusammenfassung

Hintergrund

Ursodeoxycholsäure (13–15 mg/kg) ist die Standardtherapie einer primär biliären Cholangitis (PBC). Der Behandlungserfolg soll nach 12 Monaten überprüft werden. Es wurde analysiert, wie diese Empfehlungen umgesetzt werden.

Methoden

Im Deutschen PBC-Register wurden drei Subgruppen rekrutiert: Patienten mit adäquatem und inadäquatem UDCA-Therapieansprechen (Paris-II-Kriterien) und neu diagnostizierte Patienten (PBC-Diagnose <6 Monate). Dieser Artikel beschreibt neu diagnostizierte Patienten mit UDCA-Monotherapie.

Ergebnisse

82 Patienten wurden rekrutiert (43 an 12 tertiären und 39 an 9 sekundären Zentren), darunter 22% mit Zirrhose. Patienten mit Zirrhose waren älter (71 ± 9 vs. 55 ± 14 Jahre, p<0,001) und wiesen häufiger einen Diabetes mellitus (44% vs. 13%, p=0,0054) und eine arterielle Hypertonie (78% vs. 42%, p=0,0076) auf als Patienten ohne Zirrhose.

12-Monats-Follow-up-Daten waren für 62 Fälle verfügbar. Eine UDCA-Unterdosierung (<13 mg/kg/d) lag an tertiären und sekundären Zentren bei 47% und 74% der Patienten (p=0,013) zu Therapiebeginn und bei 29% und 73% (p=0,002) nach 12 Monaten vor. Die Paris-II-Kriterien wurden bei 74% der Patienten und ein „tiefes UDCA-Therapieansprechen“ (Alkalische Phosphatase <ULN und Bilirubin <0,6 × ULN) bei 32% erreicht.

Zusammenfassung

Bei neu diagnostizierten PBC-Patienten findet sich ein relevanter Anteil spät diagnostizierter Fälle mit Zirrhose. UDCA wird häufig zu niedrig dosiert. Der Zeitpunkt einer PBC-Diagnose und die UDCA-Dosis sollten verbessert werden.

Keywords

Liver - Ursodeoxycholic acid - Alkaline Phosphatase - Bilirubin - Paris II criteria

Schlüsselwörter

Leber - Ursodeoxycholsäure - Alkalische Phosphatase - Bilirubin - Paris II Kriterien


Publication History

Received: 17 June 2025

Accepted after revision: 10 October 2025

Article published online:
09 December 2025

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