2024
OP-1 LONG-TERM EFFICACY AND SAFETY OF OPEN-LABEL SELADELPAR TREATMENT IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: INTERIM 2-YEAR RESULTS FROM THE ASSURE STUDY
Trivedi P, Levy C, Kowdley K, Gordon S, Bowlus C, Hurtado M, Hirschfield G, Gulamhusien A, Lawitz E, Villamil A, de Guevara Cetina A, Mayo M, Younes Z, Shibolet O, Yimam K, Pratt D, Heo J, Morgera U, Andreone P, Kremer A, Corpechot C, Goel A, Peyton A, Elbeshbeshy H, Crittenden D, Heusner C, Proehl S, Zhou S, McWherter C. OP-1 LONG-TERM EFFICACY AND SAFETY OF OPEN-LABEL SELADELPAR TREATMENT IN PATIENTS WITH PRIMARY BILIARY CHOLANGITIS: INTERIM 2-YEAR RESULTS FROM THE ASSURE STUDY. Annals Of Hepatology 2024, 29: 101599. DOI: 10.1016/j.aohep.2024.101599.Peer-Reviewed Original ResearchPrimary biliary cholangitisCrossover patientsBiliary cholangitisBiochemical markers of cholestasisBiochemical markersMarkers of cholestasisPhase 3 trialLong-term efficacyNumerical rating scaleLong-term usePlacebo patientsOpen-labelMethods PatientsAdverse eventsComposite endpointSafety resultsSeladelparUrsodeoxycholic acidPlaceboPhase 3 responsePruritusPatientsEndpointAssurance studyCholangitisA Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis
Hirschfield G, Bowlus C, Mayo M, Kremer A, Vierling J, Kowdley K, Levy C, Villamil A, Ladrón de Guevara Cetina A, Janczewska E, Zigmond E, Jeong S, Yilmaz Y, Kallis Y, Corpechot C, Buggisch P, Invernizzi P, Londoño Hurtado M, Bergheanu S, Yang K, Choi Y, Crittenden D, McWherter C. A Phase 3 Trial of Seladelpar in Primary Biliary Cholangitis. New England Journal Of Medicine 2024, 390: 783-794. PMID: 38381664, DOI: 10.1056/nejmoa2312100.Peer-Reviewed Original ResearchConceptsPrimary biliary cholangitisAlkaline phosphatase levelsPruritus Numerical Rating ScalePercentage of patientsNumerical rating scaleBiliary cholangitisAdverse eventsPhosphatase levelsPlacebo groupUrsodeoxycholic acidPeroxisome proliferator-activated receptor delta agonistBaseline to month 6Severity of adverse eventsAlkaline phosphatase normalizationModerate-to-severe pruritusPlacebo-controlled trialPhase 3 trialUnacceptable side effectsRating ScaleBiochemical responsesBackground therapyDouble-blindDelta agonistsBilirubin levelsMonth 6Per-oral cholangioscopy in patients with primary sclerosing cholangitis: a 12-month follow-up study
Mohamed R, Tejaswi S, Aabakken L, Ponsioen C, Bowlus C, Adler D, Forbes N, Paulsen V, Voermans R, Urayama S, Peetermans J, Rousseau M, Eksteen B. Per-oral cholangioscopy in patients with primary sclerosing cholangitis: a 12-month follow-up study. Endoscopy International Open 2024, 12: e237-e244. PMID: 38362361, PMCID: PMC10869209, DOI: 10.1055/a-2236-7557.Peer-Reviewed Original ResearchEndoscopic retrograde cholangiographySerious adverse eventsPrimary sclerosing cholangitisLiver transplantationIndex procedurePSC patientsSclerosing cholangitisOral cholangioscopyRepeat endoscopic retrograde cholangiographyPrior liver transplantationCases of cholangiocarcinomaCCA incidencePrimary endpointSecondary endpointsAdverse eventsBiopsy findingsConsecutive patientsRetrograde cholangiographyAdditional patientsCytology findingsRepeat proceduresMean ageTherapeutic maneuversBenign cytologyCCA diagnosis
2023
Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis
Kowdley K, Bowlus C, Levy C, Akarca U, Alvares-da-Silva M, Andreone P, Arrese M, Corpechot C, Francque S, Heneghan M, Invernizzi P, Jones D, Kruger F, Lawitz E, Mayo M, Shiffman M, Swain M, Valera J, Vargas V, Vierling J, Villamil A, Addy C, Dietrich J, Germain J, Mazain S, Rafailovic D, Taddé B, Miller B, Shu J, Zein C, Schattenberg J. Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis. New England Journal Of Medicine 2023, 390: 795-805. PMID: 37962077, DOI: 10.1056/nejmoa2306185.Peer-Reviewed Original ResearchMeSH KeywordsAdministration, OralAlkaline PhosphataseBilirubinChalconesCholagogues and CholereticsCholestasisDouble-Blind MethodGastrointestinal AgentsHumansLiver Cirrhosis, BiliaryPeroxisome Proliferator-Activated ReceptorsPPAR alphaPPAR deltaPropionatesPruritusTreatment OutcomeUrsodeoxycholic AcidConceptsPrimary biliary cholangitisBiliary cholangitisAlkaline phosphatase levelsPeroxisome proliferator-activated receptorPhosphatase levelsDual peroxisome proliferator-activated receptorLeast-squares mean changeChronic cholestatic liver diseaseItch Numeric Rating ScaleDestruction of interlobular bile ductsModerate-to-severe pruritusPlacebo-controlled trialCholestatic liver diseaseNumerical rating scaleInterlobular bile ductsWorst Itch Numeric Rating ScaleIndicator of cholestasisProliferator-activated receptorsAbdominal painDouble-blindPlacebo groupRelevant biochemical indicatorsAdverse eventsPruritus intensityUrsodeoxycholic acidOpen‐label, clinical trial extension: Two‐year safety and efficacy results of seladelpar in patients with primary biliary cholangitis
Mayo M, Vierling J, Bowlus C, Levy C, Hirschfield G, Neff G, Galambos M, Gordon S, Borg B, Harrison S, Thuluvath P, Goel A, Shiffman M, Swain M, Jones D, Trivedi P, Kremer A, Aspinall R, Sheridan D, Dörffel Y, Yang K, Choi Y, McWherter C. Open‐label, clinical trial extension: Two‐year safety and efficacy results of seladelpar in patients with primary biliary cholangitis. Alimentary Pharmacology & Therapeutics 2023, 59: 186-200. PMID: 37904314, DOI: 10.1111/apt.17755.Peer-Reviewed Original ResearchConceptsPrimary biliary cholangitisBiliary cholangitisLiver injurySerious treatment-related adverse eventsBiochemical markersLong-term extension studyTreatment-related adverse eventsAnti-pruritic effectsClinical trial extensionsSecondary efficacy analysesTwo-Year SafetyUnexpected histological findingsNon-alcoholic steatohepatitisLong-term safetyAdverse eventsElevated bilirubinComposite endpointDose adjustmentEfficacy analysisHistological findingsTrial extensionEfficacy dataEfficacy resultsSelective peroxisomePatients