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 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 strategies
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 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 reduction