2025
Portal and Hepatic Vein Embolization vs Portal Venous Embolization Alone in Cirrhotic and Non-cirrhotic Swine: A Pilot Study
Tefera J, Kuhn T, Matuschewski N, Meister E, Nguyenová J, Kao T, Mutonga M, Bitar R, Kahl V, Zhang X, Shewarega A, Chapiro J, Madoff D. Portal and Hepatic Vein Embolization vs Portal Venous Embolization Alone in Cirrhotic and Non-cirrhotic Swine: A Pilot Study. Journal Of Vascular And Interventional Radiology 2025 PMID: 40043833, DOI: 10.1016/j.jvir.2025.02.028.Peer-Reviewed Original ResearchPost-embolizationCirrhotic groupPortal venous embolizationHepatic vein embolizationPortal venous phaseNon-cirrhotic groupStudent's t-testFLR hypertrophyFLR increaseVein embolizationVenous phaseEffects of PVELiver volumeCT scanVenous embolizationEmbolizationCirrhotic liverFour-week intervalsWeeksT-testFLRLiverPilot studyYorkshire pigsPVEAcid sphingomyelinase deficiency and Gaucher disease: Underdiagnosed and often treatable causes of hepatomegaly, splenomegaly, and low HDL cholesterol in lean individuals
Mistry P, Cassiman D, Jones S, Lachmann R, Lukina E, Prada C, Wasserstein M, Thurberg B, Foster M, Patel R, Underhill L, Peterschmitt M. Acid sphingomyelinase deficiency and Gaucher disease: Underdiagnosed and often treatable causes of hepatomegaly, splenomegaly, and low HDL cholesterol in lean individuals. Hepatology Communications 2025, 9: e0621. PMID: 39774103, PMCID: PMC11717527, DOI: 10.1097/hc9.0000000000000621.Peer-Reviewed Original ResearchConceptsGaucher disease type 1Acid sphingomyelinase deficiencyHDL cholesterolSphingomyelinase deficiencyDifferential diagnosis of patientsBiomarkers of disease activityLipoprotein phenotypeLower body mass indexMean HDL cholesterolLow HDL cholesterolLiver function testsDiagnosis of patientsBody mass indexModerate hepatosplenomegalySpleen volumeLipid abnormalitiesLow HDLDiagnostic delayIrreversible complicationsLiver volumeLyso-sphingomyelinMultisystemic manifestationsDisease activityLDL cholesterolDifferential diagnosis
2024
Artificial Intelligence–Driven Patient Selection for Preoperative Portal Vein Embolization for Patients with Colorectal Cancer Liver Metastases
Kuhn T, Engelhardt W, Kahl V, Alkukhun A, Gross M, Iseke S, Onofrey J, Covey A, Camacho J, Kawaguchi Y, Hasegawa K, Odisio B, Vauthey J, Antoch G, Chapiro J, Madoff D. Artificial Intelligence–Driven Patient Selection for Preoperative Portal Vein Embolization for Patients with Colorectal Cancer Liver Metastases. Journal Of Vascular And Interventional Radiology 2024, 36: 477-488. PMID: 39638087, DOI: 10.1016/j.jvir.2024.11.025.Peer-Reviewed Original ResearchTotal liver volumeMetastatic colorectal cancer patientsPreoperative portal vein embolizationColorectal cancer liver metastasesPortal vein embolizationCancer liver metastasesMulticenter retrospective studyColorectal cancer patientsStudent's t-testBoard-certified radiologistsVein embolizationConsecutive patientsLiver metastasesLiver volumePatient selectionRetrospective studyCancer patientsRadiomic featuresInclusion criteriaPatientsSemi-automatic segmentationLab valuesT-testSDAUCLong‐term effectiveness of eliglustat treatment: A real‐world analysis from the International Collaborative Gaucher Group Gaucher Registry
Mistry P, Balwani M, Charrow J, Lorber J, Niederau C, Carwile J, Oliveira‐dos‐Santos A, Perichon M, Cil S, Kishnani P. Long‐term effectiveness of eliglustat treatment: A real‐world analysis from the International Collaborative Gaucher Group Gaucher Registry. American Journal Of Hematology 2024, 99: 1500-1510. PMID: 38686876, DOI: 10.1002/ajh.27347.Peer-Reviewed Original ResearchGaucher disease type 1International Collaborative Gaucher Group Gaucher RegistryTreatment naive patientsBone mineral densityGaucher RegistrySwitching patientsPlatelet countSpleen volumeClinical parametersFollow-upLumbar spine BMD Z-scoreLumbar spine bone mineral densityClinical trialsSpine bone mineral densitySpine BMD Z-scoreZ-scoreYears of follow-upBMD Z-scoresGaucher disease type 1 patientsRegistration clinical trialsEnzyme replacement therapyEliglustat treatmentMineral densityReplacement therapyLiver volume
2022
Dragon 1 Protocol Manuscript: Training, Accreditation, Implementation and Safety Evaluation of Portal and Hepatic Vein Embolization (PVE/HVE) to Accelerate Future Liver Remnant (FLR) Hypertrophy
Korenblik R, Olij B, Aldrighetti LA, Hilal MA, Ahle M, Arslan B, van Baardewijk LJ, Baclija I, Bent C, Bertrand CL, Björnsson B, de Boer MT, de Boer SW, Bokkers RPH, Rinkes IHMB, Breitenstein S, Bruijnen RCG, Bruners P, Büchler MW, Camacho JC, Cappelli A, Carling U, Chan BKY, Chang DH, choi J, Font JC, Crawford M, Croagh D, Cugat E, Davis R, De Boo DW, De Cobelli F, De Wispelaere JF, van Delden OM, Delle M, Detry O, Díaz-Nieto R, Dili A, Erdmann JI, Fisher O, Fondevila C, Fretland Å, Borobia FG, Gelabert A, Gérard L, Giuliante F, Gobardhan PD, Gómez F, Grünberger T, Grünhagen DJ, Guitart J, Hagendoorn J, Heil J, Heise D, Herrero E, Hess GF, Hoffmann MH, Iezzi R, Imani F, Nguyen J, Jovine E, Kalff JC, Kazemier G, Kingham TP, Kleeff J, Kollmar O, Leclercq WKG, Ben SL, Lucidi V, MacDonald A, Madoff DC, Manekeller S, Martel G, Mehrabi A, Mehrzad H, Meijerink MR, Menon K, Metrakos P, Meyer C, Moelker A, Modi S, Montanari N, Navines J, Neumann UP, Peddu P, Primrose JN, Qu X, Raptis D, Ratti F, Ridouani F, Rogan C, Ronellenfitsch U, Ryan S, Sallemi C, Moragues JS, Sandström P, Sarriá L, Schnitzbauer A, Serenari M, Serrablo A, Smits MLJ, Sparrelid E, Spüntrup E, Stavrou GA, Sutcliffe RP, Tancredi I, Tasse JC, Udupa V, Valenti D, Fundora Y, Vogl TJ, Wang X, White SA, Wohlgemuth WA, Yu D, Zijlstra IAJ, Binkert CA, Bemelmans MHA, van der Leij C, Schadde E, van Dam RM. Dragon 1 Protocol Manuscript: Training, Accreditation, Implementation and Safety Evaluation of Portal and Hepatic Vein Embolization (PVE/HVE) to Accelerate Future Liver Remnant (FLR) Hypertrophy. CardioVascular And Interventional Radiology 2022, 45: 1391-1398. PMID: 35790566, PMCID: PMC9458562, DOI: 10.1007/s00270-022-03176-1.Peer-Reviewed Original ResearchConceptsFuture liver remnant hypertrophyHepatic vein embolizationMulticenter prospective single-arm trialResectable colorectal cancer liver metastasesColorectal cancer liver metastasesProspective single-arm trialFeasibility of resectionCancer liver metastasesDisease-free survivalSingle-arm trialSmall FLRPrimary endpointSecondary endpointsFLR hypertrophyLiver metastasesOverall survivalProspective trialVein embolizationLiver functionSafety profileArm trialLiver volumeWeek 1Week 3Study centers
2021
Benefit of Deep Inspiratory Breath Hold for Right Breast Cancer When Regional Lymph Nodes Are Irradiated
Peters GW, Gao SJ, Knowlton C, Zhang A, Evans SB, Higgins S, Wilson LD, Saltmarsh N, Picone M, Moran MS. Benefit of Deep Inspiratory Breath Hold for Right Breast Cancer When Regional Lymph Nodes Are Irradiated. Practical Radiation Oncology 2021, 12: e7-e12. PMID: 34508890, DOI: 10.1016/j.prro.2021.08.010.Peer-Reviewed Original ResearchConceptsRight-sided breast cancerRegional nodal irradiationLiver VBreast cancerLung VDIBH plansLiver parametersLung doseComprehensive regional nodal irradiationLeft-sided breast cancerRight breast cancerWilcoxon signed-rank testingRegional lymph nodesTotal liver volumeIpsilateral lung VLiver DHeart VNodal irradiationRBC patientsHeart doseLymph nodesInspiratory breathLiver volumeLarge cohortTreatment planClinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results
Mistry PK, Lukina E, Turkia H, Shankar SP, Feldman H, Ghosn M, Mehta A, Packman S, Lau H, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Foster MC, Gaemers SJM, Peterschmitt MJ. Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results. American Journal Of Hematology 2021, 96: 1156-1165. PMID: 34161616, PMCID: PMC8457136, DOI: 10.1002/ajh.26276.Peer-Reviewed Original ResearchConceptsDisease type 1Gaucher disease type 1Type 1Disease manifestationsENGAGE trialOpen-label extension periodOral substrate reduction therapySpine T-scoreYears of treatmentSubstrate reduction therapyMagnitude of improvementEliglustat therapyMean hemoglobinUntreated patientsAdverse eventsTrial cohortClinical outcomesEligible adultsPlatelet countUntreated adultsSpleen volumeLiver volumeHematologic parametersReduction therapyPatientsIdentifying enhancement-based staging markers on baseline MRI in patients with colorectal cancer liver metastases undergoing intra-arterial tumor therapy
Ghani MA, Fereydooni A, Chen E, Letzen B, Laage-Gaupp F, Nezami N, Deng Y, Gan G, Thakur V, Lin M, Papademetris X, Schernthaner RE, Huber S, Chapiro J, Hong K, Georgiades C. Identifying enhancement-based staging markers on baseline MRI in patients with colorectal cancer liver metastases undergoing intra-arterial tumor therapy. European Radiology 2021, 31: 8858-8867. PMID: 34061209, PMCID: PMC8848338, DOI: 10.1007/s00330-021-08058-7.Peer-Reviewed Original ResearchConceptsColorectal cancer liver metastasesCancer liver metastasesTotal tumor volumeIntra-arterial therapyTotal liver volumeLiver metastasesTumor volumeTumor burdenTumor diameterPatient survivalBaseline MRILiver volumeMultivariable Cox proportional hazards modelsKaplan-Meier survival curvesWhole liverCox proportional hazards modelKaplan-Meier methodPrognostic staging systemSurvival of patientsColorectal cancer metastasisMethodsThis retrospective studyPre-treatment MRIProportional hazards modelAppropriate cutoff valueHR 1.7Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment
Weinreb NJ, Camelo JS, Charrow J, McClain MR, Mistry P, Belmatoug N, investigators F. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment. Molecular Genetics And Metabolism 2021, 132: 100-111. PMID: 33485799, DOI: 10.1016/j.ymgme.2020.12.295.Peer-Reviewed Original ResearchConceptsBone painNon-splenectomized patientsType 1 patientsBone crisesPlatelet countLiver volumeSubset analysisBody mass index (BMI) outcomesGaucher diseaseEarly treatment yearsInitial clinical improvementDifferent patient subsetsPre-treatment baselineLong-term treatmentEnzyme replacement therapyICGG Gaucher RegistryGD type 1 patientsImiglucerase treatmentAdult patientsClinical improvementSplenectomy statusGaucher RegistryPatient subsetsTreatment initiationNormal weight
2020
Real‐world effectiveness of eliglustat in treatment‐naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry
Mistry PK, Balwani M, Charrow J, Kishnani P, Niederau C, Underhill LH, McClain MR. Real‐world effectiveness of eliglustat in treatment‐naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry. American Journal Of Hematology 2020, 95: 1038-1046. PMID: 32438452, PMCID: PMC7497238, DOI: 10.1002/ajh.25875.Peer-Reviewed Original ResearchConceptsSpine Z-scoreInternational Collaborative Gaucher Group Gaucher RegistryGaucher disease type 1Treatment-naïve patientsSwitch patientsMean hemoglobinPlatelet countLiver volumeZ-scoreGaucher RegistrySpleen volumeLumbar spine Z-scoreFirst-line oral therapyCYP2D6 metabolizer phenotypeMean platelet countReal-world effectivenessClinical trial resultsDisease type 1Real-world outcomesEliglustat therapyGD1 patientsOral therapyTreatment-naïveMost patientsERT patientsAccuracy of chitotriosidase activity and CCL18 concentration in assessing type I Gaucher disease severity. A systematic review with meta-analysis of individual participant data
Raskovalova T, Deegan PB, Mistry PK, Pavlova E, Yang R, Zimran A, Berger J, Bourgne C, Pereira B, Labarere J, Berger MG. Accuracy of chitotriosidase activity and CCL18 concentration in assessing type I Gaucher disease severity. A systematic review with meta-analysis of individual participant data. Haematologica 2020, 106: 437-445. PMID: 32001533, PMCID: PMC7849573, DOI: 10.3324/haematol.2019.236083.Peer-Reviewed Original ResearchConceptsIndividual participant dataCCL18 concentrationsChitotriosidase activityPrimary outcomeSystematic reviewParticipant dataProspective cohort studyEligible primary studiesPrimary studiesDisease activityBone eventsCohort studyPlatelet countGD patientsSpleen volumeLiver volumeClinical assessmentVisceral parametersGD severityPatientsHemoglobin concentrationRoutine practiceDisease severityLimited sample sizeGD activity
2019
Two years of efficacy of oral eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher registry
Mistry P, Balwani M, Charrow J, Kishnani P, Niederau C, McClain M. Two years of efficacy of oral eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher registry. Molecular Genetics And Metabolism 2019, 126: s100-s101. DOI: 10.1016/j.ymgme.2018.12.252.Peer-Reviewed Original ResearchX109/LTreatment-naïve patientsInternational Collaborative Gaucher Group Gaucher RegistryGaucher disease type 1Enzyme replacement therapySwitch patientsMean hemoglobinPlatelet countLiver volumeGaucher RegistrySpleen volumeClinical trialsOral substrate reduction therapyLong-term clinical improvementCYP2D6 metabolizer phenotypeMajor disease manifestationsDisease type 1Substrate reduction therapyKey disease parametersOral eliglustatTreatment-naïveClinical improvementChitotriosidase levelsDisease manifestationsReduction therapy
2017
Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial
Mistry PK, Lukina E, Turkia H, Shankar SP, Baris H, Ghosn M, Mehta A, Packman S, Pastores G, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Gaemers SJM, Tayag R, Peterschmitt MJ. Outcomes after 18 months of eliglustat therapy in treatment‐naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial. American Journal Of Hematology 2017, 92: 1170-1176. PMID: 28762527, PMCID: PMC5656936, DOI: 10.1002/ajh.24877.Peer-Reviewed Original ResearchConceptsGaucher disease type 1Double-blind periodBone mineral densityDisease type 1Liver volumeEliglustat treatmentPlatelet countMineral densityENGAGE trialHemoglobin concentrationOral substrate reduction therapyType 1Bone marrow burdenExtension periodExtensive CYP2D6 metabolizersOpen-label periodTreatment-naïve patientsFirst-line treatmentTreatment-naïve adultsBone marrow burden scoreNew safety concernsSubstrate reduction therapyEliglustat therapyTrial patientsCYP2D6 metabolizers
2016
3D Quantitative tumour burden analysis in patients with hepatocellular carcinoma before TACE: comparing single-lesion vs. multi-lesion imaging biomarkers as predictors of patient survival
Fleckenstein FN, Schernthaner RE, Duran R, Sohn JH, Sahu S, Zhao Y, Hamm B, Gebauer B, Lin M, Geschwind JF, Chapiro J. 3D Quantitative tumour burden analysis in patients with hepatocellular carcinoma before TACE: comparing single-lesion vs. multi-lesion imaging biomarkers as predictors of patient survival. European Radiology 2016, 26: 3243-3252. PMID: 26762942, PMCID: PMC4942412, DOI: 10.1007/s00330-015-4168-3.Peer-Reviewed Original ResearchConceptsHazard ratioOverall survivalTransarterial chemoembolizationBaseline MRIHepatocellular carcinomaTumor burdenDominant lesionTumor assessmentTumor volumeMultivariate cox proportional hazard ratiosCox proportional hazard ratiosCurrent radiological methodMethodsThis retrospective analysisProportional hazard ratiosBCLC staging systemKaplan-Meier plotsTumor diameterPatient survivalStaging systemHCC patientsMultiple lesionsLiver volumeRetrospective analysisLesion assessmentPatients
2015
Long-Term Hematologic Response to Eliglustat in Patients with Gaucher Disease Type 1: Results from a Phase 2 and Two Phase 3 Trials
Weinreb N, Cox T, Lukina E, Mistry P, Angell J, Gaemers S, Peterschmitt M. Long-Term Hematologic Response to Eliglustat in Patients with Gaucher Disease Type 1: Results from a Phase 2 and Two Phase 3 Trials. Blood 2015, 126: 884. DOI: 10.1182/blood.v126.23.884.884.Peer-Reviewed Original ResearchGaucher disease type 1Enzyme replacement therapyPhase 3 trialMean platelet countHemoglobin levelsPlatelet countSpleen volumeDisease type 1Substrate reduction therapySpeakers bureauLiver volumeOpen-label phase 2 trialYears of ERTMononuclear phagocytesPlacebo-controlled phase 3 trialBaseline meanOral substrate reduction therapyIntravenous enzyme replacement therapyType 1Acid β-glucosidaseTravel reimbursementCYP2D6 metabolizer phenotypeMain efficacy parametersX109/LTreatment-naïve patientsFeasibility of a Modified Cone-Beam CT Rotation Trajectory to Improve Liver Periphery Visualization during Transarterial Chemoembolization.
Schernthaner RE, Chapiro J, Sahu S, Withagen P, Duran R, Sohn JH, Radaelli A, van der Bom IM, Geschwind JF, Lin M. Feasibility of a Modified Cone-Beam CT Rotation Trajectory to Improve Liver Periphery Visualization during Transarterial Chemoembolization. Radiology 2015, 277: 833-41. PMID: 26000642, PMCID: PMC4654716, DOI: 10.1148/radiol.2015142821.Peer-Reviewed Original ResearchConceptsCone-beam CTTransarterial chemoembolizationLiver volumeRadiation exposureInstitutional review board-approved studyPeripheral hepatic tumorsSecondary liver cancerCT radiation exposureCone beam CT examinationsBoard-approved studyTumor detectabilityRadiation exposure reductionHepatic tumorsCT examinationsLiver cancerCT protocolPatientsCone beamMagnetic resonance imagesCT indicesC-arm rotatesCTTumorsExposure reductionRank testEffect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1: The ENGAGE Randomized Clinical Trial
Mistry PK, Lukina E, Turkia H, Amato D, Baris H, Dasouki M, Ghosn M, Mehta A, Packman S, Pastores G, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Shankar S, Solano MH, Ross L, Angell J, Peterschmitt MJ. Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1: The ENGAGE Randomized Clinical Trial. JAMA 2015, 313: 695-706. PMID: 25688781, PMCID: PMC4962880, DOI: 10.1001/jama.2015.459.Peer-Reviewed Original ResearchConceptsGaucher disease type 1Disease type 1Efficacy end pointSpleen volumeHemoglobin levelsPlatelet countLiver volumeType 1End pointUntreated adultsOral substrate reduction therapyPrimary efficacy end pointSecondary efficacy end pointsOpen-label extension studyPercentage changeAbsolute treatment differenceEffective oral therapyMean spleen volumePlacebo-controlled trialSecondary end pointsSerious adverse eventsEnzyme replacement therapySubstrate reduction therapyBaseline splenomegalyEligible patients
2013
Engage: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Study To Investigate The Efficacy and Safety Of Eliglustat In Adults With Gaucher Disease Type 1: 9 Month Results
Shankar S, Lukina E, Amato D, Dasouki M, Packman S, Pastores G, Assouline S, Balwani M, Mistry P, Ross L, Marulkar S, Peterschmitt M. Engage: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multi-center Study To Investigate The Efficacy and Safety Of Eliglustat In Adults With Gaucher Disease Type 1: 9 Month Results. Blood 2013, 122: 2275. DOI: 10.1182/blood.v122.21.2275.2275.Peer-Reviewed Original ResearchGaucher disease type 1Disease type 1Spleen volumeSubstrate reduction therapyType 1Efficacy endpointUntreated adultsLiver volumeReduction therapyBone marrowDisease severity scoring systemOral substrate reduction therapyBone mineral density changesAdvisory CommitteePrimary efficacy endpointSecondary efficacy endpointsSymptomatic bone diseaseMainstay of therapyOpen-label trialPhase 3 trialBone marrow burden scoreMineral density changesBurden of diseaseCommon lysosomal storage disorderSeverity scoring system
2011
Indications and Contraindications for Portal Vein Embolization
Madoff D, Vauthey J. Indications and Contraindications for Portal Vein Embolization. 2011, 123-128. DOI: 10.1007/978-1-84882-122-4_13.Peer-Reviewed Original ResearchPortal vein embolizationTotal liver volumeVein embolizationUse of PVEFuture liver remnant volumeLiver remnant volumePatient body sizeFLR volumePerioperative outcomesPatient ageSurgical candidatesLiver diseaseRemnant volumeSurgical interventionLiver volumeCT volumetryHealthy liverParticular patientPatientsContraindicationsEmbolizationAgeMultiple factorsCirrhosisComorbiditiesEvaluation of high density lipoprotein as a circulating biomarker of Gaucher disease activity
Stein P, Yang R, Liu J, Pastores GM, Mistry PK. Evaluation of high density lipoprotein as a circulating biomarker of Gaucher disease activity. Journal Of Inherited Metabolic Disease 2011, 34: 429-437. PMID: 21290183, PMCID: PMC3186206, DOI: 10.1007/s10545-010-9271-7.Peer-Reviewed Original ResearchConceptsEnzyme replacement therapyHigh-density lipoproteinHDL cholesterolDisease severityDisease activitySpleen volumeIndividual patientsLow high-density lipoproteinLow HDL cholesterolSeverity Score IndexMonitoring of patientsGD1 patientsSerum levelsReplacement therapyLiver volumeScore indexDensity lipoproteinPatientsCholesterolSeverityChitotriosidaseBiomarkersStriking increaseImportant surrogateLipoprotein
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