Treatment response to ursodeoxycholic acid in primary biliary cholangitis: A systematic review and meta-analysis.
Barcelona
GLOBE Score
Meta-analysis
Paris-1
Paris-2
Primary biliary cholangitis
Rotterdam
Systematic review
Toronto
UDCA response
UK-PBC Risk score
Ursodeoxycholic acid
Journal
Digestive and liver disease : official journal of the Italian Society of Gastroenterology and the Italian Association for the Study of the Liver
ISSN: 1878-3562
Titre abrégé: Dig Liver Dis
Pays: Netherlands
ID NLM: 100958385
Informations de publication
Date de publication:
10 2023
10 2023
Historique:
received:
26
08
2022
revised:
09
11
2022
accepted:
19
12
2022
medline:
23
10
2023
pubmed:
3
1
2023
entrez:
2
1
2023
Statut:
ppublish
Résumé
Several ursodeoxycholic acid (UDCA) treatment response definitions have been introduced in primary biliary cholangitis (PBC). However, the lack of a gold standard results in heterogeneity in second-line treatment research and clinical practice. This study aimed to explore which UDCA treatment response endpoint serves as the most accurate predictive model of long-term outcome. A systematic review and meta-analysis of UDCA treatment response endpoints (and corresponding validations) were performed. Sixteen individual UDCA treatment response endpoints and 96 external validations were found. Barcelona, Paris-1, Paris-2, Rotterdam, Toronto and GLOBE and UK-PBC Risk Scores are currently most robustly validated in external populations. The results show that the continuous models (GLOBE and UK-PBC Risk Scores) serve as the most accurate predictive models. Besides standard UDCA treatment response endpoints, the alkaline phosphatase and total bilirubin normalization has been suggested as a new therapeutic target. The GLOBE and UK-PBC Risk Scores are the most suitable for the real-world allocation of second-line therapies (obeticholic acid and fibrates). However, in the wake of the recent findings, alkaline phosphatase and total bilirubin normalization should be the primary outcome in trial research in PBC.
Sections du résumé
BACKGROUND
Several ursodeoxycholic acid (UDCA) treatment response definitions have been introduced in primary biliary cholangitis (PBC). However, the lack of a gold standard results in heterogeneity in second-line treatment research and clinical practice.
AIMS
This study aimed to explore which UDCA treatment response endpoint serves as the most accurate predictive model of long-term outcome.
METHODS
A systematic review and meta-analysis of UDCA treatment response endpoints (and corresponding validations) were performed.
RESULTS
Sixteen individual UDCA treatment response endpoints and 96 external validations were found. Barcelona, Paris-1, Paris-2, Rotterdam, Toronto and GLOBE and UK-PBC Risk Scores are currently most robustly validated in external populations. The results show that the continuous models (GLOBE and UK-PBC Risk Scores) serve as the most accurate predictive models. Besides standard UDCA treatment response endpoints, the alkaline phosphatase and total bilirubin normalization has been suggested as a new therapeutic target.
CONCLUSIONS
The GLOBE and UK-PBC Risk Scores are the most suitable for the real-world allocation of second-line therapies (obeticholic acid and fibrates). However, in the wake of the recent findings, alkaline phosphatase and total bilirubin normalization should be the primary outcome in trial research in PBC.
Identifiants
pubmed: 36593158
pii: S1590-8658(22)00834-9
doi: 10.1016/j.dld.2022.12.010
pii:
doi:
Substances chimiques
Ursodeoxycholic Acid
724L30Y2QR
Cholagogues and Choleretics
0
Alkaline Phosphatase
EC 3.1.3.1
Bilirubin
RFM9X3LJ49
Types de publication
Meta-Analysis
Systematic Review
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
1318-1327Informations de copyright
Copyright © 2022 Editrice Gastroenterologica Italiana S.r.l. Published by Elsevier Ltd. All rights reserved.
Déclaration de conflit d'intérêts
Conflict of interest Jakub Gazda, Matej Gazda, Tomas Koky, and Marian Macej declare that they have no conflicts of interest. Sylvia Drazilova, Martin Janicko, and Peter Jarcuska received lecture fees from PRO.MED.CS Praha a. s. Marco Carbone is on the advisory board for Intercept, Perspectum, Mayoly, Calliditas and received lecture fees from Intercept and Mayoly.