2025
Development of the natural history component of an early economic model for primary sclerosing cholangitis
Bowlus C, Levy C, Kowdley K, Kachru N, Jeyakumar S, Rodriguez-Guadarrama Y, Smith N, Briggs A, Sculpher M, Ollendorf D. Development of the natural history component of an early economic model for primary sclerosing cholangitis. Orphanet Journal Of Rare Diseases 2025, 20: 133. PMID: 40102907, PMCID: PMC11921552, DOI: 10.1186/s13023-025-03658-8.Peer-Reviewed Original ResearchConceptsQuality-adjusted life yearsTransplant-free survivalProportion of patientsRecurrent PSCLiver transplantationDisease progressionOverall survivalSclerosing cholangitisSecondary cancersCumulative incidenceCumulative incidence of secondary cancersIncidence of secondary cancersChronic cholestatic diseasePrimary sclerosing cholangitisProgression to cirrhosisLifetime quality-adjusted life yearsBackgroundPrimary sclerosing cholangitisMarkov cohort modelInflammatory bowel diseaseEconomic modelGallbladder cancerEstimates of disease progressionCholestatic diseasesFibrosis stageLiver failure
2024
PPAR agonists for the treatment of cholestatic liver diseases: Over a decade of clinical progress
Hayes C, Gallucci G, Boyer J, Assis D, Ghonem N. PPAR agonists for the treatment of cholestatic liver diseases: Over a decade of clinical progress. Hepatology Communications 2024, 9: e0612. PMID: 39699308, PMCID: PMC11661771, DOI: 10.1097/hc9.0000000000000612.Peer-Reviewed Original ResearchConceptsPeroxisome proliferator-activated receptor agonistsPrimary biliary cholangitisPrimary sclerosing cholangitisPeroxisome proliferator-activated receptorUnited States Food and Drug Administration approvalHepatic accumulation of bile acidsRisk of progression to cirrhosisFood and Drug Administration approvalPeroxisome proliferator-activator receptor agonismSecond-line therapyFirst-line therapySymptoms of cholestasisDrug Administration approvalTreatment of cholestatic liver diseasesCholestatic liver diseaseAccumulation of bile acidsProgression to cirrhosisTreatment of cholestasisSignificant clinical interestSmall bile ductsProliferator-activated receptorsBile acid metabolismIncomplete respondersBiliary cholangitisBiliary stricturesSerologic extracellular matrix remodeling markers are related to fibrosis stage and prognosis in a phase 2b trial of simtuzumab in patients with primary sclerosing cholangitis
Thorburn D, Leeming D, Barchuk W, Wang Y, Lu X, Malkov V, Ito K, Bowlus C, Levy C, Goodman Z, Karsdal M, Muir A, Xu J. Serologic extracellular matrix remodeling markers are related to fibrosis stage and prognosis in a phase 2b trial of simtuzumab in patients with primary sclerosing cholangitis. Hepatology Communications 2024, 8: e0467. PMID: 38967589, PMCID: PMC11227354, DOI: 10.1097/hc9.0000000000000467.Peer-Reviewed Original ResearchConceptsPrimary sclerosing cholangitisELF scoreAdvanced fibrosisFibrosis stageFibrosis progressionClinical eventsSclerosing cholangitisExtracellular matrix remodeling markersRemodeling markersPredictors of disease severityPhase 2b trialBaseline to weekPRO-C3Progression to cirrhosisWilcoxon rank sum testMarker of collagen formationType III procollagenModerate diagnostic abilityDiagnosing fibrosis stagesRank sum testSerum markersNoninvasive predictorPrognostic markerModerate prognosisMedian changeAn explainable machine learning model for prediction of high-risk nonalcoholic steatohepatitis
Njei B, Osta E, Njei N, Al-Ajlouni Y, Lim J. An explainable machine learning model for prediction of high-risk nonalcoholic steatohepatitis. Scientific Reports 2024, 14: 8589. PMID: 38615137, PMCID: PMC11016071, DOI: 10.1038/s41598-024-59183-4.Peer-Reviewed Original ResearchConceptsNational Health and Nutrition Examination SurveyRisk of progression to cirrhosisClinical Risk IndexHealth and Nutrition Examination SurveyControlled attenuation parameter valuesNovel therapeutic optionsLiver stiffness measurementProgression to cirrhosisAspartate aminotransferase levelsNutrition Examination SurveyResource-limited settingsFIB-4Fibrosis scorePlatelet countAminotransferase levelsTherapeutic optionsWaist circumferenceClinically applicable modelExamination SurveyNonalcoholic steatohepatitisFAST scoreEarly identificationElastography measurementsStiffness measurementPatients
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