Cited by 180Liver Diseases and ImmunityPediatric Hepatobiliary Diseases and TreatmentsDrug Transport and Resistance Mechanisms

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Long-term efficacy and safety of obeticholic acid for patients with primary biliary cholangitis: 3-year results of an international open-label extension study

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Trauner, Michael, Nevens, Frederik, Shiffman, Mitchell L, Drenth, Joost P H, Bowlus, Christopher L, Vargas, Victor, Andreone, Pietro, Hirschfield, Gideon M, Pencek, Richard, Malecha, Elizabeth Smoot, MacConell, Leigh, Shapiro, David·2019·The Lancet Gastroenterology & Hepatology

DOI 10.1016/s2468-1253(19)30094-9

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Abstract

BACKGROUND The aim of this study was to evaluate the long-term efficacy and safety of obeticholic acid for patients with primary biliary cholangitis using 3-year interim data from the 5-year open-label extension of the pivotal phase 3 POISE trial. METHODS In the double-blind phase of POISE, 217 patients with primary biliary cholangitis with inadequate response to or intolerance to ursodeoxycholic acid were randomised to receive placebo, obeticholic acid 5 to 10 mg, or obeticholic acid 10 mg once daily for 12 months. During the open-label extension phase, patients received variable, adjusted doses of obeticholic acid. Markers of cholestasis and liver injury, alkaline phosphatase (ALP), and total and direct bilirubin were evaluated, and safety was assessed for up to 48 months of treatment with obeticholic acid. All analyses in the open-label extension were done in the safety population, defined as any patient randomised in the double-blind phase who received at least one dose of obeticholic acid during the open-label extension. This trial is registered at ClinicalTrials.gov (NCT01473524) and with EudraCT (2011-004728-36). FINDINGS 193 patients were treated during the open-label extension. In this 3-year interim analysis, ALP concentrations were significantly reduced compared with baseline at 12 months (mean change -105·2 U/L [SD 87·6]), 24 months (-101·0 U/L [98·5]), 36 months (-108·6 U/L [95·7]), and 48 months (-95·6 U/L [121·1]; p<0·0001 for all yearly time points). Total bilirubin concentrations were stabilised, with significant reductions versus baseline at 12 months (mean change -0·9 μmol/L [SD 4·1]; p=0·0042) and 48 months (-0·8 μmol/L [3·8]; p=0·016). Stabilisation was also noted for direct bilirubin, with a significant change from baseline at 12 months (mean change -0·5 μmol/L [SD 3·0]; p=0·021). However, changes in total and direct bilirubin were not significant at other time points. Obeticholic acid was generally well tolerated, with pruritus (149 [77%] patients) and fatigue (63 [33%]) being the most common adverse events. No serious adverse events were considered related to obeticholic acid. INTERPRETATION Interim analyses suggest long-term efficacy and safety of obeticholic acid in patients with primary biliary cholangitis who are intolerant to or inadequately responsive to ursodeoxycholic acid. FUNDING Intercept Pharmaceuticals.

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Authors: Trauner, M., Nevens, F., Shiffman, M. L., Drenth, J. P. H., Bowlus, C. L., Vargas, V., Andreone, P., Hirschfield, G. M., Pencek, R., Malecha, E. S., MacConell, L., & Shapiro, D.
Affiliations: Medical University of Vienna (AT)
KU Leuven (BE)
Bon Secours Liver Institute of Hampton Roads (US)
Radboud University Medical Center (NL)
University of California, Davis (US)
National Institute for Health and Care Research (GB)
Intercept Pharmaceuticals (United States) (US)
Corresponding author: Trauner, Michael
ORCID: Trauner, Michael
Nevens, Frederik
Shiffman, Mitchell L
Drenth, Joost P H
Bowlus, Christopher L
Hirschfield, Gideon M
Venue: The Lancet Gastroenterology & Hepatology
Publisher: Elsevier BV
Published: 2019-06
Volume: 4
Issue: 6
Pages: 445-453
ISSN: 2468-1156 (ISSN-L)
2468-1253
DOI: 10.1016/s2468-1253(19)30094-9
OpenAlex: W2922727372
Semantic Scholar: 0b165ee31de96a2d016dc69d19703615a5965f51
Cited by: 180
References: 24
Citations by year: 2026: 10 · 2025: 29 · 2024: 23 · 2023: 21 · 2022: 37 · 2021: 27 · 2020: 23 · 2019: 10
FWCI: 15.46
Type: journal article
Language: en
Open access: green
hdl.handle.net
Licence: cc-by
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
www.elsevier.com/legal/tdmrep-license (tdm)
Locations: The Lancet. Gastroenterology & hepatology (publishedVersion)
PubMed (publishedVersion)
Archivio istituzionale della ricerca (Alma Mater Studiorum Università di Bologna) (submittedVersion)
IRIS UNIMORE (University of Modena and Reggio Emilia) (submittedVersion, cc-by)
Lirias (KU Leuven) (submittedVersion)
Radboud Repository (Radboud University) (submittedVersion)
Funders: Intercept Pharmaceuticals
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, long-term efficacy, drug safety, alkaline phosphatase, total bilirubin, direct bilirubin, cholestasis, liver injury, open-label extension, POISE trial, fumarate esters
MeSH terms: Alkaline Phosphatase, Bilirubin, Chenodeoxycholic Acid, Cholagogues and Choleretics, Cholangitis, Dose-Response Relationship, Drug, Double-Blind Method, Fatigue, Female, Gastrointestinal Agents, Humans, Male, Middle Aged, Pruritus, Ursodeoxycholic Acid

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Long-term efficacy and safety of obeticholic acid for patients with primary biliary cholangitis: 3-year results of an international open-label extension study

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