A Nationwide Assessment of Anticholestatic Therapy Uptake in Patients With Primary Biliary Cholangitis: Opportunities for Optimisation

BACKGROUND: Optimal care delivery for primary biliary cholangitis (PBC) has become challenging due to new therapies and more individualized biochemical treatment goals. AIM: To evaluate uptake of ursodeoxycholic acid (UDCA) and second-line therapy (SLT) in a nationwide PBC cohort and identify factors associated with suboptimal therapeutical management. METHODS: The Dutch PBC Cohort Study is a retrospective study including every identifiable patient with PBC in the Netherlands from 1990 onwards. Use of UDCA and SLT was assessed among patients in clinical follow-up on 1 January 2019. Biochemical response was defined as alkaline phosphatase (ALP) and aspartate aminotransferase (AST) ≤ 1.5 × ULN and total bilirubin (TB) ≤ ULN. SLT included off-label fibrates, budesonide and/or obeticholic acid (not reimbursed). RESULTS: In total, 2638 patients with PBC were included; median age was 64.0 (IQR 55.0-72.0) years, 2361 (89.5%) were female and 2143 (81.2%) were treated in a non-academic center. Overall, 2532 (96.0%) patients used UDCA. The recommended UDCA dose of ≥ 13 mg/kg/day was used by 1363/2205 (61.8%). Age (aOR 1.01, 95% CI 1.00-1.02, p = 0.02), body weight (aOR 1.04, 95% CI 1.03-1.05, p < 0.01) and biochemical response (aOR 1.35, 95% CI 1.09-1.67, p = 0.01) were associated with UDCA dosage < 13 mg/kg/day. Among patients with an incomplete response, 101/638 (15.8%) were prescribed SLT. Younger age, female sex, treatment in an academic centre and higher levels of ALP and AST were positively associated with use of SLT. CONCLUSIONS: During recent years, UDCA was insufficiently dosed in Dutch patients with PBC. The results indicate that first-line treatment can be optimized, especially in those with a biochemical response and higher body weight.

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Publication Details

Journal
Alimentary Pharmacology & Therapeutics
Published
2026-04-14
DOI
https://doi.org/10.1111/apt.70669
Citations
2
Primary Topic
Liver Diseases and Immunity
Type
article
Field-Weighted Citation Impact
30.11
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article

A Nationwide Assessment of Anticholestatic Therapy Uptake in Patients With Primary Biliary Cholangitis: Opportunities for Optimisation

Frans J.C. Cuperus, Femke Boersma, Jan Maarten Vrolijk, Hendrik J.M. de Jonge et al.
2 citations
Alimentary Pharmacology & Therapeutics
Liver Diseases and Immunity
30.11
article

A Nationwide Assessment of Anticholestatic Therapy Uptake in Patients With Primary Biliary Cholangitis: Opportunities for Optimisation

Frans J.C. Cuperus, Femke Boersma, Jan Maarten Vrolijk, Hendrik J.M. de Jonge, Robert C. Verdonk, L.C. Baak, U. Beuers, Wink de Boer, Nicole S. Erler, Hajo J. Flink, Remco van Dijk, Frank ter Borg, Kirsten Boonstra, Jeroen D. van Bergeijk, Adriaan van der Meer, Rozanne C. de Veer, E de Vries, Frank H.J. Wolfhagen, Paul G. van Putten, E. Werner, Joost P.H. Drenth, Michael Klemt‐Kropp, G.J. de Bruin, Susanne L. Onderwater, Hanneke van Soest, Maria C B van Hooff, Bart J. Veldt, Matthijs Kramer, Suzanne van Meer, Gemma Weijsters, H Janssen, the Dutch PBC Study Group, Bettina E. Hansen, Cyriel Y. Ponsioen, Rob P. R. Adang, Maria N. Aparicio‐Pages, Maria A. van de Vrie
article en
2 citations

Abstract

BACKGROUND: Optimal care delivery for primary biliary cholangitis (PBC) has become challenging due to new therapies and more individualized biochemical treatment goals. AIM: To evaluate uptake of ursodeoxycholic acid (UDCA) and second-line therapy (SLT) in a nationwide PBC cohort and identify factors associated with suboptimal therapeutical management. METHODS: The Dutch PBC Cohort Study is a retrospective study including every identifiable patient with PBC in the Netherlands from 1990 onwards. Use of UDCA and SLT was assessed among patients in clinical follow-up on 1 January 2019. Biochemical response was defined as alkaline phosphatase (ALP) and aspartate aminotransferase (AST) ≤ 1.5 × ULN and total bilirubin (TB) ≤ ULN. SLT included off-label fibrates, budesonide and/or obeticholic acid (not reimbursed). RESULTS: In total, 2638 patients with PBC were included; median age was 64.0 (IQR 55.0-72.0) years, 2361 (89.5%) were female and 2143 (81.2%) were treated in a non-academic center. Overall, 2532 (96.0%) patients used UDCA. The recommended UDCA dose of ≥ 13 mg/kg/day was used by 1363/2205 (61.8%). Age (aOR 1.01, 95% CI 1.00-1.02, p = 0.02), body weight (aOR 1.04, 95% CI 1.03-1.05, p < 0.01) and biochemical response (aOR 1.35, 95% CI 1.09-1.67, p = 0.01) were associated with UDCA dosage < 13 mg/kg/day. Among patients with an incomplete response, 101/638 (15.8%) were prescribed SLT. Younger age, female sex, treatment in an academic centre and higher levels of ALP and AST were positively associated with use of SLT. CONCLUSIONS: During recent years, UDCA was insufficiently dosed in Dutch patients with PBC. The results indicate that first-line treatment can be optimized, especially in those with a biochemical response and higher body weight.

Alimentary Pharmacology & Therapeutics
University Medical Center Groningen (NL), Radboud University Nijmegen (NL), Utrecht University (NL), Leiden University Medical Center (NL), Gelre Hospitals (NL), Catharina Ziekenhuis (NL), Erasmus MC (NL), Medisch Centrum Leeuwarden (NL), Radboud University Medical Center (NL), University Medical Center Utrecht (NL), Maastricht University (NL), Noordwest Ziekenhuisgroep (NL), Albert Schweitzer Ziekenhuis (NL), Medisch Centrum Haaglanden (NL), Reinier de Graaf Hospital (NL), St. Antonius Ziekenhuis (NL), OLVG (NL), Diakonessenhuis hospital (NL), Canisius-Wilhelmina Ziekenhuis (NL), VieCuri Medisch Centrum (NL), Ziekenhuis Gelderse Vallei (NL), Radiotherapiegroep (NL), Udens College (NL), Isala (NL), Tergooi (NL), Amsterdam University Medical Centers (NL), Rijnstate Hospital (NL), Toronto Liver Centre (CA), Deventer Ziekenhuis (NL), Jeroen Bosch Ziekenhuis (NL), University of Amsterdam (NL)
Openalex Percentile: Top 0%
Liver Diseases and Immunity
30.11
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