2022
Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial
Schilsky ML, Czlonkowska A, Zuin M, Cassiman D, Twardowschy C, Poujois A, de Assis A Gondim F, Denk G, Cury RG, Ott P, Moore J, Ala A, D'Inca R, Couchonnal-Bedoya E, D'Hollander K, Dubois N, Kamlin COF, Weiss KH, investigators C, To U, Patel A, Hettiarachchi D, Giorgini A, Monico S, Litwin T, Piechal A, Skowronska M, Lachaux A, Belmalih A, Boogers A, Mohr I, Langel A, Freitas C, Barbosa E, Sandahl T, Gerdes L, Obadia A, Rahli D, Cosgrove J. Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial. The Lancet Gastroenterology & Hepatology 2022, 7: 1092-1102. PMID: 36183738, DOI: 10.1016/s2468-1253(22)00270-9.Peer-Reviewed Original ResearchConceptsUrinary copper excretionNon-inferiority marginWilson's diseaseCopper excretionMean differenceStable Wilson's diseaseHealth care centersOral penicillaminePenicillamine groupPenicillamine intoleranceMaintenance therapyPrimary endpointStable patientsDaily doseStudy treatmentTreat approachClinical assessmentAlanine aminotransferaseBaseline valuesSite investigatorsPatientsInherited disorderExtension periodDiseaseGeneral linear model
2020
A window into portal hemodynamics in adult fontan patients?
Simmons MA, Revzin M, To U, Liapakis A, Fahey J, Elder RW. A window into portal hemodynamics in adult fontan patients? International Journal Of Cardiology 2020, 323: 61-64. PMID: 32979426, DOI: 10.1016/j.ijcard.2020.09.047.Peer-Reviewed Original ResearchConceptsAdult Fontan patientsFontan patientsSpleen stiffnessCongenital heart diseaseLiver stiffnessShear wave elastographyControl patientsPortal hemodynamicsLiver diseaseHeart diseaseFontan-associated liver diseaseLimited subgroup analysisPortal venous hemodynamicsAssociated liver diseasePassive hepatic congestionUltrasound shear wave elastographyFontan circulationStudy cohortHealthy patientsHepatic congestionVenous hemodynamicsSubgroup analysisLiver fibrosisHealthy controlsPatients