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
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
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 intervention