Preventing Long-Term Neurologic Disability in Hemophilia (A): Cost-Effectiveness of Emicizumab Prophylaxis for the Prevention of Intracranial Hemorrhage in Infants with Severe Hemophilia (A)
Glaeser-Khan S, Richmond R, Ito S, Bona R, Krumholz H, Cuker A, Goshua G. Preventing Long-Term Neurologic Disability in Hemophilia (A): Cost-Effectiveness of Emicizumab Prophylaxis for the Prevention of Intracranial Hemorrhage in Infants with Severe Hemophilia (A). Blood 2024, 144: 157-157. DOI: 10.1182/blood-2024-203690.Peer-Reviewed Original ResearchIncremental cost-effectiveness ratioStandard-of-careStandard half-lifeEmicizumab prophylaxisSevere hemophilia AIntracranial hemorrhageIntracranial hemorrhage incidenceSevere HAHemophilia AProbabilistic sensitivity analysesYears of agePrevention of intracranial hemorrhageNeurological disabilityPrevent intracranial hemorrhageIntracranial hemorrhage eventsIntracranial hemorrhage riskPhase III studyBase-casePersistent neurological disabilityAged 1 monthFactor VIII replacementStandard-of-care armUS societal perspectiveInfants aged 0Aged 0Cost-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-VEGF