2024
Cost-effectiveness of iptacopan in paroxysmal nocturnal hemoglobinuria
Ito S, Chetlapalli K, Wang D, Potnis K, Richmond R, Krumholz H, Lee A, Cuker A, Goshua G. Cost-effectiveness of iptacopan in paroxysmal nocturnal hemoglobinuria. Blood 2024 PMID: 39374533, DOI: 10.1182/blood.2024025176.Peer-Reviewed Original ResearchStandard-of-careParoxysmal nocturnal hemoglobinuriaIncremental net monetary benefitNocturnal hemoglobinuriaComplement-mediated hemolytic anemiaTreating paroxysmal nocturnal hemoglobinuriaComplement C5 inhibitor eculizumabPhase 3 studyQuality-adjusted life expectancyRare blood disorderComprehensive cost-effectiveness analysisProbabilistic sensitivity analysesCost-saving thresholdsC5 inhibitor eculizumabNet monetary benefitPersistent anemiaIptacopanExtravascular hemolysisIntravenous infusionMonotherapyHemolytic anemiaAnemia resolutionC5 inhibitionFDA approvalPrimary outcomeCancer research is not correlated with driver gene mutation burdens
Mendiratta G, Liarakos D, Tong M, Ito S, Ke E, Goshua G, Stites E. Cancer research is not correlated with driver gene mutation burdens. Med 2024, 5: 832-838.e4. PMID: 38908369, DOI: 10.1016/j.medj.2024.05.013.Peer-Reviewed Original ResearchCancer patient populationCancer researchCancer research effortsResearch allocation decisionsNational InstitutePatient populationResearch fundingBurdenBurden of mutationsFunding decisionsCancerGenetic driversGene mutation burdenFunding amountFundingGenetic drivers of cancerAllocation decisionsCancer-associated genesEpidemiologyDrivers of cancerMutational burdenBaselineEffortsFactorsBalance of prioritiesCost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia
Wang D, Ito S, Waldron C, Butt A, Zhang E, Krumholz H, Al-Samkari H, Goshua G. Cost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia. Blood Advances 2024, 8: 2835-2845. PMID: 38537061, PMCID: PMC11176968, DOI: 10.1182/bloodadvances.2024012589.Peer-Reviewed Original ResearchIncremental net monetary benefitHereditary hemorrhagic telangiectasiaStandard-of-careBevacizumab therapyWillingness-to-payNet monetary benefitHemorrhagic telangiectasiaWillingness-to-pay thresholdsStandard-of-care strategiesCare of patientsCost-effectiveness analysisAnti-VEGF bevacizumabSystemic antiangiogenic therapyCurrent standard-of-careMonetary benefitsTime horizonFormulary placementHealthcare resource utilizationStandard of careLifetime time horizonPatient quality-of-lifeScenario analysisProbabilistic sensitivity analysesIV bevacizumabAnti-VEGFCost-effectiveness of prophylaxis with recombinant vs plasma-derived VWF in severe von Willebrand disease in the United States
Waldron C, Ito S, Wang D, Allen C, Viswanathan G, Bona R, Cuker A, Goshua G. Cost-effectiveness of prophylaxis with recombinant vs plasma-derived VWF in severe von Willebrand disease in the United States. Blood 2024, 143: 2332-2335. PMID: 38635764, DOI: 10.1182/blood.2024024209.Peer-Reviewed Original ResearchCost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura
Allen C, Ito S, Butt A, Purcell A, Richmond R, Tormey C, Krumholz H, Cuker A, Goshua G. Cost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura. Blood Advances 2024, 8: 2279-2289. PMID: 38502197, PMCID: PMC11116991, DOI: 10.1182/bloodadvances.2024012608.Peer-Reviewed Original ResearchImmune thrombotic thrombocytopenic purpuraPLASMIC scoreThrombotic thrombocytopenic purpuraThrombocytopenic purpuraADAMTS13 testingIncremental net monetary benefitPer-patient cost savingsTherapeutic plasma exchangeBase-case analysisMarkov cohort simulationProbabilistic sensitivity analysesAmount of QALYEmpirical therapyADAMTS13 assaysPlasma exchangeEmpirical treatmentCaplacizumabFRET-based assayPrimary outcomePatientsNet monetary benefitCohort simulationCost-effectiveness evaluationPurpuraTesting strategiesCost‐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 anemiaDecreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource
Ito S, Pandya A, Hauser R, Krishnamurti L, Stites E, Tormey C, Krumholz H, Hendrickson J, Goshua G. Decreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource. American Journal Of Hematology 2024, 99: 570-576. PMID: 38279581, DOI: 10.1002/ajh.27211.Peer-Reviewed Original ResearchSickle cell diseaseDelayed hemolytic transfusion reactionQuality-adjusted life expectancyAlloimmunized patientsPatient populationRed blood cell alloimmunizationCell diseaseCost-effective interventionMedical expenditure of patientsHealth system perspectiveExpenditure of patientsIncremental cost-effectiveness ratioHemolytic transfusion reactionsUnited StatesMarkov cohort simulationCost-effectiveAverage patient populationCost-effectiveness ratioBirth cohortAnalytical time horizonAntibody historyCohort simulationTransfusionTransfusion reactionsLife expectancy
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 analysisAllotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States
Goshua G, Ito S, Chetlapalli K, Potnis K, Calhoun C, Krishnamurti L, Krumholz H, Pandya A. Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States. Blood 2023, 142: 490. DOI: 10.1182/blood-2023-191072.Peer-Reviewed Original ResearchSickle cell diseaseIncremental cost-effectiveness ratioDistributional cost-effectiveness analysisPediatric patientsCell diseaseCost-effectiveness analysisDisease severityHealth resource utilization dataPediatric Health Information SystemGene therapyJustifiable treatment optionTransplant-related mortalityVaso-occlusive crisisExpert clinical experienceMarrow Transplant ResearchSubstantial mortality riskVisual analog scaleQuality-adjusted life expectancyConcomitant riskCost-effectiveness ratioResource utilization dataCost-effectiveness frontierHost diseaseMaximum patientsOpioid therapyLongitudinal 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 Rapid Hemosil Versus in-House and Send-out ADAMTS13 Activity Testing in the Care of Patients with Immune Thrombotic Thrombocytopenic Purpura
Allen C, Ito S, Butt A, Purcell A, Richmond R, Cuker A, Goshua G. Cost-Effectiveness of Rapid Hemosil Versus in-House and Send-out ADAMTS13 Activity Testing in the Care of Patients with Immune Thrombotic Thrombocytopenic Purpura. Blood 2023, 142: 368. DOI: 10.1182/blood-2023-184927.Peer-Reviewed Original ResearchImmune thrombotic thrombocytopenic purpuraTherapeutic plasma exchangeIncremental cost-effectiveness ratioThrombotic thrombocytopenic purpuraPLASMIC scoreIncremental net monetary benefitProbabilistic sensitivity analysesPlasma exchangeThrombocytopenic purpuraMedical intensive care unitADAMTS13 activity testingIntensive care unitCare of patientsActivity levelsHigh pretest probabilityClinical trial dataCost-effectiveness ratioCost-effectiveness evaluationDeterministic sensitivity analysesNet monetary benefitEmpiric treatmentAdverse eventsPrimary outcomeIll adultsCare unitCost-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 reductionEquitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States
Ito S, Potnis K, Allen C, Richmond R, Waldron C, Viswanathan G, Bona R, Goshua G. Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States. Blood 2023, 142: 492. DOI: 10.1182/blood-2023-190420.Peer-Reviewed Original ResearchIncremental cost-effectiveness ratioSevere hemophilia A.Quality-adjusted life expectancySevere haemophilia AHemophilia AFactor VIIIDistributional cost-effectiveness analysisHemophilia A.Chronic arthropathyOpen-label multicenter studyCoagulation factor replacementLifelong prophylactic treatmentCare of patientsCost-effectiveness ratioUSD/QALYProbabilistic sensitivity analysesCost-effectiveness analysisPackage insert informationDeterministic sensitivity analysesWeekly prophylaxisPrimary outcomeSecondary outcomesWeekly dosingFactor replacementIntracranial hemorrhageSecond-Line Therapy Sequencing in Primary Warm Autoimmune Hemolytic Anemia: Splenectomy-Rituximab Versus Rituximab-Splenectomy
Waldron C, Ito S, Wang D, Viswanathan G, Butt A, Goshua G. Second-Line Therapy Sequencing in Primary Warm Autoimmune Hemolytic Anemia: Splenectomy-Rituximab Versus Rituximab-Splenectomy. Blood 2023, 142: 2317. DOI: 10.1182/blood-2023-189957.Peer-Reviewed Original ResearchWarm autoimmune hemolytic anemiaSecond-line therapyAutoimmune hemolytic anemiaAdult patientsHemolytic anemiaSecond-line treatmentFirst-line treatmentPrevention of thrombosisDegree of anemiaLaparoscopic surgical techniquesPatient's disease statusQuality-adjusted life expectancyRate of infectionProbabilistic sensitivity analysesNet monetary benefitRituximab cyclesTherapy sequencingComplete remissionPartial remissionPerioperative mortalityPrimary outcomeTherapeutic sequencingLaparoscopic techniquePreferred treatmentRelapse riskGlobal 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 ResearchCost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States
Potnis K, Viswanathan G, Bona R, Ito S, Kempton C, Pandya A, Krumholz H, Goshua G. Cost‐effectiveness of prophylactic emicizumab versus prophylactic recombinant factor VIII in patients with moderate or mild hemophilia A without inhibitors in the United States. American Journal Of Hematology 2023, 98: e247-e250. PMID: 37401660, DOI: 10.1002/ajh.27014.Peer-Reviewed Original ResearchDistributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States.
Goshua G, Calhoun C, Ito S, James L, Luviano A, Krishnamurti L, Pandya A. Distributional Cost-Effectiveness of Equity-Enhancing Gene Therapy in Sickle Cell Disease in the United States. Annals Of Internal Medicine 2023, 176: 779-787. PMID: 37247420, PMCID: PMC10370480, DOI: 10.7326/m22-3272.Peer-Reviewed Original ResearchCross-cultural comparison of continuous deep sedation for advanced cancer patients in East Asian countries: prospective cohort study.
Yang C, Chen P, Mori M, Morita T, Cheng S, Suh S, Kim S, Yokomichi N, Imai K, Ito S, Yamaguchi T, Hiratsuka Y, Tsuneto S, Maeda S. Cross-cultural comparison of continuous deep sedation for advanced cancer patients in East Asian countries: prospective cohort study. Japanese Journal Of Clinical Oncology 2023, 53: 714-721. PMID: 37114920, DOI: 10.1093/jjco/hyad037.Peer-Reviewed Original ResearchConceptsContinuous deep sedationPractice of continuous deep sedationPrevalence of continuous deep sedationPalliative care unitAdvanced cancer patientsProspective cohort studyDeep sedationPhysician discomfortPhysician practicesPsychological symptomsSedation practicesCohort studyAdvanced cancerCompare clinical characteristicsClinical practiceCare unitCancer patientsPhysiciansSedative administrationQuantitative studyTarget symptomsPrevalenceClinical characteristicsNon-sedated groupCross-cultural comparisons
2022
Apixaban at Apex? Aligning Drug Pricing With Value in Cancer-Associated Thrombosis.
Ito S, Goshua G. Apixaban at Apex? Aligning Drug Pricing With Value in Cancer-Associated Thrombosis. Annals Of Internal Medicine 2022, 176: 125-126. PMID: 36571840, DOI: 10.7326/m22-3404.Peer-Reviewed Original Research