Protocol for the development of a core outcome set for clinical trials in primary sclerosing cholangitis
Hussain N, Ma C, Hirschfield G, Walmsley M, Hanford P, Vesterhus M, Kowdley K, Bergquist A, Ponsioen C, Levy C, Assis D, Schramm C, Bowlus C, Trauner M, Aiyegbusi O, Jairath V, Trivedi P. Protocol for the development of a core outcome set for clinical trials in primary sclerosing cholangitis. BMJ Open 2024, 14: e080143. PMID: 38926149, PMCID: PMC11216047, DOI: 10.1136/bmjopen-2023-080143.Peer-Reviewed Original ResearchMeSH KeywordsCholangitis, SclerosingClinical Trials as TopicDelphi TechniqueEndpoint DeterminationHumansOutcome Assessment, Health CareResearch DesignSystematic Reviews as TopicConceptsPrimary sclerosing cholangitisClinical trialsSclerosing cholangitisCore outcome setImmune-mediated liver diseasesPrimary sclerosing cholangitis treatmentOutcome measuresEvaluate novel therapiesSlow disease progressionPatient-reported outcome measuresMedical therapyNovel therapiesLiver fibrosisDisease progressionHistological assessmentLiver diseaseInternational two-round Delphi surveyImaging-based biomarkersIntervention trialsTherapyConsensus meetingHealthcare payersCholangitisSemistructured qualitative interviewsTwo-round Delphi surveyPatient-focused drug development in primary sclerosing cholangitis: Insights on patient priorities and involvement in clinical trials
Li M, Pai R, Gomel R, Vyas M, Callif S, Hatchett J, Bowlus C, Lai J. Patient-focused drug development in primary sclerosing cholangitis: Insights on patient priorities and involvement in clinical trials. Hepatology Communications 2024, 8: e0433. PMID: 38727680, PMCID: PMC11093571, DOI: 10.1097/hc9.0000000000000433.Peer-Reviewed Original ResearchMeSH KeywordsAdultCholangitis, SclerosingClinical Trials as TopicDisease ProgressionDrug DevelopmentFemaleHumansMaleMiddle AgedPatient ParticipationSurveys and QuestionnairesConceptsPrimary sclerosing cholangitisClinical trialsSclerosing cholangitisDrug trialsTrial participantsMultivariate logistic regressionAssociated with willingnessInflammatory bowel diseaseLong-term riskReferral of patientsDrug developmentTherapy trialsDisease progressionTrial interestBowel diseasePatient-focused drug developmentTreatment trialsPatientsBiopsy requirementsPruritusPatient interestPredictors of patients' willingnessLogistic regressionCholangitisPatients' willingness