Featured Publications
Cost‐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 anemiaCost-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 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 second‐line therapies in adults with chronic immune thrombocytopenia
Goshua G, Sinha P, Kunst N, Pischel L, Lee AI, Cuker A. Cost‐effectiveness of second‐line therapies in adults with chronic immune thrombocytopenia. American Journal Of Hematology 2022, 98: 122-130. PMID: 35147241, PMCID: PMC9365880, DOI: 10.1002/ajh.26497.Peer-Reviewed Original ResearchConceptsThrombopoietin receptor agonistsChronic immune thrombocytopeniaSecond-line therapyImmune thrombocytopeniaEarly useCurrent practice trendsProbabilistic sensitivity analysesCost-effective treatmentFirst cost-effectiveness analysisCost-effectiveness analysisU.S. health systemLack of evidenceEarly splenectomyHematology (ASH) guidelinesReceptor agonistCurrent guidelinesSplenectomyClinical practicePractice trendsTherapyHealth systemCost-effective strategyRituximabThrombocytopeniaAmerican SocietyCost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
Goshua G, Sinha P, Hendrickson J, Tormey C, Bendapudi PK, Lee AI. Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura. Blood 2021, 137: 969-976. PMID: 33280030, PMCID: PMC7918179, DOI: 10.1182/blood.2020006052.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedClinical Trials, Phase II as TopicClinical Trials, Phase III as TopicCombined Modality TherapyCost-Benefit AnalysisDecision TreesDrug CostsDrug Therapy, CombinationFemaleFibrinolytic AgentsHemorrhageHumansImmunosuppressive AgentsLength of StayMaleMarkov ChainsMiddle AgedModels, EconomicMulticenter Studies as TopicPlasma ExchangePurpura, Thrombotic ThrombocytopenicRecurrenceRituximabSingle-Domain AntibodiesStandard of CareUnited StatesYoung AdultConceptsIncremental cost-effectiveness ratioThrombotic thrombocytopenic purpuraTherapeutic plasma exchangeVon Willebrand factorRelapse rateThrombocytopenic purpuraClinical trialsMajor randomized clinical trialsThrombotic microangiopathy leadingEnd-organ damageWillebrand factorPlatelet count recoveryRandomized clinical trialsHealth system costsOne-way sensitivity analysesCost-effectiveness ratioLife-threatening diseaseProbabilistic sensitivity analysesCost-effectiveness analysisHospital lengthCount recoveryPlasma exchangeTPE treatmentTTP patientsImmunomodulatory agents
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 outcomeCost-effectiveness of adding quizartinib to induction chemotherapy for patients with FLT3-mutant acute myeloid leukemia
Bewersdorf J, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Stahl M, Stein E, Huntington S, Goshua G, Zeidan A. Cost-effectiveness of adding quizartinib to induction chemotherapy for patients with FLT3-mutant acute myeloid leukemia. Leukemia & Lymphoma 2024, 65: 1136-1144. PMID: 38648559, PMCID: PMC11265977, DOI: 10.1080/10428194.2024.2344052.Peer-Reviewed Original ResearchQuality-adjusted life yearsCompletion of consolidation therapyFLT3-mutant acute myeloid leukemiaAllogeneic hematopoietic cell transplantationIncremental cost-effectiveness ratioProbabilistic sensitivity analysesImproved overall survivalHematopoietic cell transplantationPartitioned survival analysis modelAcute myeloid leukemiaCost-effectiveness ratioFLT3 inhibitor quizartinibHealth economic implicationsConsolidation therapyInduction chemotherapyAverage wholesale priceOverall survivalCell transplantationContinuous therapyMyeloid leukemiaITD mutationQuizartinibIncremental costCost-effective optionLife years
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 analysisCost-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 risk