Effectiveness and safety of obeticholic acid in a Southern European multicentre cohort of patients with primary biliary cholangitis and suboptimal response to ursodeoxycholic acid
Open accessAbstract
Summary: Background: Obeticholic acid (OCA) was recently approved as the only on‐label alternative for patients with primary biliary cholangitis (PBC) with intolerance or suboptimal response to ursodeoxycholic acid (UDCA). However, few data are available outside clinical trials. Aim: To assess the effectiveness and safety of OCA in a real‐world cohort of patients with non‐effective UDCA therapy. Methods: Open‐label, prospective, real‐world, multicentre study, enrolling consecutive patients who did not meet Paris II criteria, from 18 institutions in Spain and Portugal. Effectiveness was assessed by the changes in GLOBE and UK‐PBC scores from baseline. POISE and Paris II criteria were evaluated after 12 months of OCA . Liver fibrosis was evaluated by FIB‐4 and AST to platelet ratio index (APRI). Results: One hundred and twenty patients were eligible, median time since PBC diagnosis 9.3 (4.0‐13.8) years, 21.7% had cirrhosis, and 26.7% received had previous or concomitant treatment with fibrates. Seventy‐eight patients completed at least 1 year of OCA. The Globe‐PBC score decreased to 0.17 (95% CI 0.05 to 0.28; P = 0.005) and the UK‐PBC score decreased to 0.81 (95% CI −0.19 to 1.80; P = 0.11). There was a significant decrease in alkaline phosphatase of 81.3 U/L (95% CI 42.5 to 120; P < 0.001), ALT 22.1 U/L (95% CI 10.4 to 33.8; P < 0.001) and bilirubin 0.12 mg/dL (95% CI 0 to 0.24; P = 0.044). FIB‐4 and APRI remained stable. According to the POISE criteria, 29.5% (23 out of 78) achieved response. The adverse events rate was 35%; 11.67% discontinued (8.3% due to pruritus). Conclusions: This study supports data from phase III trials with significant improvement of PBC‐Globe continuous prognostic marker score among OCA‐treated patients with good tolerability.
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In Gomez et al. (2020), significant reductions in ALP, ALT, and bilirubin were observed across the cohort, with a mean decrease in ALP of 81.3 U/L (95% CI: 42.5-120.0; p < 0.001), ALT decrease of 22.1 U/L (95% CI: 10.4-33.8; p < 0.001), and bilirubin decrease of 0.12 mg/dL (95% CI: 0-0.24; p = 0.044).
“Significant reductions in ALP, ALT, and bilirubin were observed across the cohort during the study period, with a mean decrease in ALP of 81.3 U/L (95% CI: 42.5–120.0; p < 0.001), ALT decrease of 22.1 U/L (95% CI: 10.4–33.8; p < 0.001), and bilirubin decrease of 0.12 mg/dL (95% CI: 0–0.24; p = 0.044).”
From Second-Line Therapies in Primary Biliary Cholangitis: A Comparative Review of Obeticholic Acid, Fibrates, Seladelpar, and Elafibranor (2025) · cites this paper as [14]
In Gomez et al. (2020), based on the POISE criteria, 29.5% of patients achieved a treatment response.
“Based on the POISE criteria, 29.5% of patients achieved a treatment response.”
From Second-Line Therapies in Primary Biliary Cholangitis: A Comparative Review of Obeticholic Acid, Fibrates, Seladelpar, and Elafibranor (2025) · cites this paper as [14]
In Gomez et al. (2020), surrogate markers of liver fibrosis, such as FIB-4 and APRI, remained unchanged over the 12-month treatment period.
“However, surrogate markers of liver fibrosis, such as the fibrosis-4 (FIB-4) index and aspartate aminotransferase (AST) to Platelet Ratio Index (APRI), remained unchanged over the 12-month treatment period.”
From Second-Line Therapies in Primary Biliary Cholangitis: A Comparative Review of Obeticholic Acid, Fibrates, Seladelpar, and Elafibranor (2025) · cites this paper as [14]
In Gomez et al. (2020), transient elastography findings indicated that liver fibrosis remained stable during OCA therapy.
“Transient elastography findings indicated that liver fibrosis remained stable during OCA therapy [14].”
From Second-Line Therapies in Primary Biliary Cholangitis: A Comparative Review of Obeticholic Acid, Fibrates, Seladelpar, and Elafibranor (2025) · cites this paper as [14]
In cohort studies in Spain and Portugal (n=120) and Italy (n=191), serum cholestatic enzymes were significantly reduced with obeticholic acid treatment, and 29.5% and 51.9% of patients met the POISE response criteria, respectively.
“Similarly, in other cohort studies in Spain and Portugal (n=120) and Italy (n=191), serum cholestatic enzymes were significantly reduced with obeticholic acid treatment, and 29•5% (Spain and Portugal cohort) and 51•9% (Italy cohort) of patients met the POISE response criteria.90,91”
From Primary biliary cholangitis. (2024) · cites this paper as [91]
Real-world data are gradually indicating an improvement in long-term prognosis with obeticholic acid.
“Regarding the long-term efficacy of obeticholic acid, real-world data are gradually indicating an improvement in long-term prognosis.[89][90][91][92][93]”
From Primary biliary cholangitis. (2024) · cites this paper as [91]
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Obeticholic Acid for Primary Biliary Cholangitis
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- Authors
- Gomez, E., Garcia Buey, L., Molina, E., Casado, M., Conde, I., Berenguer, M., Jorquera, F., Simón, M., Olveira, A., Hernández‐Guerra, M., Mesquita, M., Presa, J., Costa‐Moreira, P., Macedo, G., Arenas, J. I., Manuel Sousa, J., Ampuero, J., Morillas, R. M., Santos, A., De Carvalho, A., Uriz, J., Carrión, J. A., Luisa Gutiérrez, M., Pérez‐Fernández, E., Fernández‐Rodríguez, C. M., & the IBER‐PBC leading Cooperative Group
- Affiliations
- Madrid Spain
- Santiago Spain
- Almeria Spain
- Valencia Spain
- León Spain
- Zaragoza Spain
- Vila Real Portugal
- Porto Portugal
- Santa Cruz de Tenerife Spain
- San Sebastian Spain
- Sevilla Spain
- Barcelona Spain
- Coimbra Spain
- Navarra Spain
- Corresponding author
- Fernández‐Rodríguez, Conrado M.
- ORCID
- Venue
- Alimentary Pharmacology & Therapeutics
- Publisher
- Wiley
- Published
- 2020-12-12
- Volume
- 53
- Issue
- 4
- Pages
- 519-530
- ISSN
- 0269-2813 (ISSN-L)
- 1365-2036
- OpenAlex
- W3111759537
- Cited by
- 41
- References
- 10
- Citations by year
- 2026: 2 · 2025: 12 · 2024: 11 · 2023: 3 · 2022: 10 · 2021: 3
- FWCI
- 2.35
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Alimentary Pharmacology & Therapeutics (publishedVersion)
- PubMed (publishedVersion)
- DIGITAL.CSIC (Spanish National Research Council (CSIC)) (submittedVersion)
- Repository of Digital Objects for Teaching Research and Culture (University of Valencia) (submittedVersion, cc-by-nc)
- Zaguan (Universidad de Zaragoza) (submittedVersion)
- Topics
- Health Sciences › Medicine › Hepatology › Liver Diseases and Immunity
- Health Sciences › Medicine › Surgery › Pediatric Hepatobiliary Diseases and Treatments
- Health Sciences › Medicine › Oncology › Drug Transport and Resistance Mechanisms
- Keywords
- obeticholic acid, primary biliary cirrhosis, real-world effectiveness, drug safety, liver fibrosis, GLOBE score, pruritus, adverse events, liver cirrhosis, fibrates, alkaline phosphatase, bilirubin
- MeSH terms
- Chenodeoxycholic Acid, Cholagogues and Choleretics, Humans, Liver Cirrhosis, Biliary, Prospective Studies, Spain, Ursodeoxycholic Acid
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