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 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