2024
Identifying Patients with Erythrocytosis Not Requiring JAK2 Testing: External Validation and Decision Analysis of the Jakpot Prediction Rule
Lam B, Ito S, Ryu J, O'Sullivan K, Potnis K, Lu R, Bidikian A, Freed J, La J, Goshua G. Identifying Patients with Erythrocytosis Not Requiring JAK2 Testing: External Validation and Decision Analysis of the Jakpot Prediction Rule. Blood 2024, 144: 3669-3669. DOI: 10.1182/blood-2024-198033.Peer-Reviewed Original ResearchNegative predictive valueBeth Israel Deaconess Medical CenterClinical prediction rulePolycythemia veraIncremental net monetary benefitHealth resource utilizationPV patientsMyeloproliferative neoplasmsVA cohortTest characteristicsUS health system perspectivePrediction ruleAssociated with increased riskNon-fatal cardiovascular eventsExternal validation cohortAssociated with increased risk of strokeRandomized trial dataMarkov cohort modelLifetime time horizonProbabilistic sensitivity analysesRisk of strokePersistent erythrocytosisMedian ageHematologic malignanciesDiagnostic workupCost‐effectiveness of sutimlimab in cold agglutinin disease
Ito S, Wang D, Purcell A, Chetlapalli K, Lee A, Cuker A, Goshua G. Cost‐effectiveness of sutimlimab in cold agglutinin disease. American Journal Of Hematology 2024, 99: 1475-1484. PMID: 38733355, DOI: 10.1002/ajh.27358.Peer-Reviewed Original ResearchPrimary cold agglutinin diseaseCold agglutinin diseaseStandard-of-careIncremental cost-effectiveness ratioUS willingness-to-pay thresholdRare autoimmune hemolytic anemiaPhase 3 studyWillingness-to-pay thresholdsAutoimmune hemolytic anemiaTime-limited treatmentHuman monoclonal antibodyCold-reactive antibodiesBinding to red blood cellsFollow-up dataCost-effectiveness ratioComplement-mediated hemolysisDeterministic sensitivity analysisHealth resource utilizationProbabilistic sensitivity analysesTransfusion-DependentClinical remissionTransfusion supportImprove quality of lifeRed blood cellsHemolytic anemia
2023
Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States
Ito S, Chetlapalli K, Potnis K, Richmond R, Wang D, Lee A, Goshua G. Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States. Blood 2023, 142: 5042. DOI: 10.1182/blood-2023-188063.Peer-Reviewed Original ResearchTreatment-experienced patientsParoxysmal nocturnal hemoglobinuriaIncremental net monetary benefitIncremental cost-effectiveness ratioNursing timeExtravascular hemolysisNocturnal hemoglobinuriaPhase III study resultsTreatment-experienced adult patientsTransfusion-associated adverse eventsCost-effective therapeutic optionAnti-C5 therapyComplement inhibition therapyComplement inhibitor therapyHealth resource utilizationCare of patientsIndirect medical costsComplement component 5Clinical trial dataLife-threatening hematological disorderCost-effectiveness ratioBreakthrough therapy designationProbabilistic sensitivity analysesFirst cost-effectiveness analysisCost-effectiveness analysisCost-Effectiveness of Sutimlimab Versus Standard-of-Care in Transfusion Dependent Patients with Primary Cold Agglutinin Disease in the United States
Ito S, Wang D, Purcell A, Chetlapalli K, Lee A, Cuker A, Goshua G. Cost-Effectiveness of Sutimlimab Versus Standard-of-Care in Transfusion Dependent Patients with Primary Cold Agglutinin Disease in the United States. Blood 2023, 142: 2316. DOI: 10.1182/blood-2023-187270.Peer-Reviewed Original ResearchPrimary cold agglutinin diseaseCold agglutinin diseaseIncremental cost-effectiveness ratioTransfusion-dependent patientsIncremental net monetary benefitProbabilistic sensitivity analysesDistributional cost-effectiveness analysisAnemia severityBody weightPhase 3 clinical studiesHealth resource utilizationRed blood cell destructionDisease-specific mortalityHistory of transfusionAutoimmune hemolytic anemiaMarkov state transition modelCare of patientsHumanized monoclonal antibodyLower body weightBlood cell destructionCost-effectiveness ratioFDA package insertsCold-reactive antibodiesBase-case analysisCost-effectiveness analysisLongitudinal Systemic Bevacizumab Therapy Produces Cost Savings and Improves Quality-Adjusted Life Expectancy in the Care of Patients with Hereditary Hemorrhagic Telangiectasia with Moderate-to-Severe Bleeding
Wang D, Ito S, Waldron C, Butt A, Zhang E, Krumholz H, Al-Samkari H, Goshua G. Longitudinal Systemic Bevacizumab Therapy Produces Cost Savings and Improves Quality-Adjusted Life Expectancy in the Care of Patients with Hereditary Hemorrhagic Telangiectasia with Moderate-to-Severe Bleeding. Blood 2023, 142: 2312. DOI: 10.1182/blood-2023-185340.Peer-Reviewed Original ResearchHereditary hemorrhagic telangiectasiaIncremental net monetary benefitIncremental cost-effectiveness ratioEmergency department visitsCare of patientsHemostatic proceduresIron infusionPatient timeBevacizumab therapyDepartment visitsRBC transfusionHemorrhagic telangiectasiaVascular endothelial growth factor inhibitionRed blood cell transfusionEQ-5D index valuesCommon hereditary bleeding disorderLocal hemostatic proceduresRBC transfusion needSystemic bevacizumab therapyHealth resource utilizationBlood cell transfusionWorld Health Organization VigiBaseLong-term followGrowth factor inhibitionCost-saving interventionCost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease
Waldron C, Ito S, Wang D, Allen C, Viswanathan G, Bona R, Cuker A, Goshua G. Cost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease. Blood 2023, 142: 3698. DOI: 10.1182/blood-2023-188018.Peer-Reviewed Original ResearchSevere von Willebrand diseaseLong-term prophylaxisIncremental net monetary benefitVon Willebrand diseasePlasma-derived von Willebrand factorIncremental cost-effectiveness ratioNet monetary benefitVon Willebrand factorProbabilistic sensitivity analysesDeterministic sensitivity analysesSevere haemophiliaWillebrand factorRecombinant von Willebrand factorWillebrand diseasePhase 3 clinical trialsHealth resource utilizationFactor replacement therapyFrequency of infusionsSubset of patientsCost of prophylaxisType 3 von Willebrand diseaseFactor replacement productsSimilar significant improvementsSevere phenotypeNon-significant reductionGlobal Health Resource Utilization and Cost-Effectiveness of Therapeutics and Diagnostics in Immune Thrombotic Thrombocytopenic Purpura (TTP)
Butt A, Allen C, Purcell A, Ito S, Goshua G. Global Health Resource Utilization and Cost-Effectiveness of Therapeutics and Diagnostics in Immune Thrombotic Thrombocytopenic Purpura (TTP). Journal Of Clinical Medicine 2023, 12: 4887. PMID: 37568288, PMCID: PMC10420213, DOI: 10.3390/jcm12154887.Peer-Reviewed Original Research
2022
Resource utilization and treatment costs of patients with severe hemophilia A: Real‐world data from the ATHNdataset
Recht M, He C, Chen E, Cheng D, Solari P, Hinds D. Resource utilization and treatment costs of patients with severe hemophilia A: Real‐world data from the ATHNdataset. EJHaem 2022, 3: 341-352. PMID: 35846041, PMCID: PMC9176109, DOI: 10.1002/jha2.412.Peer-Reviewed Original ResearchHemophilia treatment centersSevere hemophilia AComprehensive care visitsHistory of inhibitorsTransfusion-transmitted infectionsClinical characteristicsHemophilia APlasma-derived factor productsReal-world dosingTreatment costsTreatment cost of patientsPrevalence of HBVPatient clinical characteristicsSubgroup of patientsPrevalence of HCVRetrospective cohort studySubpopulation of patientsHealthcare resource utilizationDrivers of resource utilizationAnnual cost of treatmentCare visitsHealth resource utilizationTreatment frequencyCosts of patientsAnnual treatment costs
2021
Evaluation of diagnostic yield of EUS among patients with asymptomatic common bile duct dilation: systematic review and meta-analysis
Chhoda A, Dawod S, Grimshaw A, Gunderson C, Mahadev S. Evaluation of diagnostic yield of EUS among patients with asymptomatic common bile duct dilation: systematic review and meta-analysis. Gastrointestinal Endoscopy 2021, 94: 890-901.e8. PMID: 34245751, DOI: 10.1016/j.gie.2021.07.002.Peer-Reviewed Original ResearchConceptsCommon bile duct dilationBile duct dilationAsymptomatic patientsBiliary dilationDiagnostic yieldCBD dilationDuct dilationEUS evaluationSystematic reviewHealth resource utilizationInverse variance methodRandom-effects modelCost-effectiveness analysisRadiologic evidenceBenign etiologyMalignant etiologyFrequent indicationOvid EmbaseAsymptomatic individualsCochrane LibraryMalignant pathologyOvid MEDLINEFurther interventionPatientsGoogle ScholarHealth Resource Utilization of Labor Induction Versus Expectant Management
Grobman W, Sandoval G, Reddy U, Tita A, Silver R, Mallett G, Hill K, Rice M, El-Sayed Y, Wapner R, Rouse D, Saade G, Thorp J, Chauhan S, Iams J, Chien E, Casey B, Gibbs R, Srinivas S, Swamy G, Simhan H, Macones G. Health Resource Utilization of Labor Induction Versus Expectant Management. Obstetric Anesthesia Digest 2021, 41: 34-34. DOI: 10.1097/01.aoa.0000732480.32100.cf.Peer-Reviewed Original ResearchExpectant managementElective inductionNulliparous womenLow-risk nulliparous womenHealth care resource utilizationHealth resource utilizationNeonatal respiratory morbidityElective labor inductionARRIVE trialDelivery admissionHypertensive disordersCesarean deliveryLabor inductionRespiratory morbidityWeeks postpartumTrialsManagement trialsWeeksWomenInductionCurrent studyResource utilizationAntepartumMorbidityPregnancyAdult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model
Li N, Sawyer E, Maruszczyk K, Guzauskas G, Slomka M, Burke T, Martin A, O’Hara J, Stevenson M, Recht M. Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model. Journal Of Medical Economics 2021, 24: 363-372. PMID: 33591884, DOI: 10.1080/13696998.2021.1891088.Peer-Reviewed Original ResearchConceptsStandard half-lifeExtended half-lifeSevere HBFactor IXHemophilia BAssociated health resource utilizationUS third-party payerFactor IX prophylaxisBleeding-related complicationsLifetime costsFixed treatmentStandard of careChronic joint damageTreatment costsSocietal perspectiveHealth resource utilizationHalf-lifeDecision-analytic modelClotting factor IXBleeding eventsTreatment paradigmTreatment optionsCongenital disorderProphylaxisLifetime horizon
2018
Incidence, Predictors, and In-Hospital Outcomes of Transcatheter Aortic Valve Implantation After Nonelective Admission in Comparison With Elective Admission: From the Nationwide Inpatient Sample Database
Ando T, Adegbala O, Villablanca P, Akintoye E, Ashraf S, Shokr M, Briasoulis A, Takagi H, Grines C, Afonso L, Schreiber T. Incidence, Predictors, and In-Hospital Outcomes of Transcatheter Aortic Valve Implantation After Nonelective Admission in Comparison With Elective Admission: From the Nationwide Inpatient Sample Database. The American Journal Of Cardiology 2018, 123: 100-107. PMID: 30360892, DOI: 10.1016/j.amjcard.2018.09.023.Peer-Reviewed Original ResearchConceptsTranscatheter aortic valve implantationNationwide Inpatient Sample databaseNonelective admissionsPropensity-matched cohortAortic valve implantationHospital mortalityValve implantationElective admissionsOutcomes of TAVITransarterial transcatheter aortic valve implantationSample databaseHigher adverse outcomesPulmonary circulation disordersAcute kidney injuryHealth resource utilizationIn-Hospital OutcomesChronic kidney diseaseIndependent risk factorCongestive heart failureHigh-risk cohortAcute myocardial infarctionCardiogenic shockHospital lengthKidney injuryRespiratory failureRemote monitoring of implantable cardiac devices
Ganeshan R, Enriquez AD, Freeman JV. Remote monitoring of implantable cardiac devices. Current Opinion In Cardiology 2018, 33: 20-30. PMID: 29059076, DOI: 10.1097/hco.0000000000000471.Peer-Reviewed Original ResearchConceptsCardiac implantable electronic devicesPatient outcomesHealth resource utilizationRemote monitoring transmissionsRate of hospitalizationNew practice settingsImplantable cardiac devicesImplantable electronic devicesTreatment guidelinesAtrial fibrillationClinical eventsEmergency departmentPhysician carePatient experienceComprehensive followDose relationshipSuperior outcomesPractice settingsCardiac devicesEarly detectionRecent evidenceCareAppropriate useOutcomesPatients
2001
Disease status and use of ventilatory support by ALS patients
Cedarbaum J, Stambler N. Disease status and use of ventilatory support by ALS patients. Amyotrophic Lateral Sclerosis And Frontotemporal Degeneration 2001, 2: 19-22. PMID: 11465928, DOI: 10.1080/146608201300079373.Peer-Reviewed Original ResearchConceptsMechanical ventilationPlacebo patientsUse of MVRespiratory adverse eventsUse of BiPAPHealth resource utilizationPhase III trialsBiPAP useR-metHuBDNFProspective databaseSymptom durationVentilatory supportAdverse eventsIII trialsALS patientsMean baselinePrescribing practicesVital capacityBehavior of physiciansBiPAPALSFRS scorePatientsMortality rateDisease statusTrials
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