2025
Emicizumab for preventing intracranial hemorrhage in infants with severe hemophilia A: a cost-effectiveness analysis
Glaeser-Khan S, Ito S, Sra M, Richmond R, Bona R, Krumholz H, Croteau S, Cuker A, Goshua G. Emicizumab for preventing intracranial hemorrhage in infants with severe hemophilia A: a cost-effectiveness analysis. Blood Advances 2025 PMID: 40795232, DOI: 10.1182/bloodadvances.2025016822.Peer-Reviewed Original ResearchIncremental cost-effectiveness ratioSevere hemophilia AIntracranial hemorrhageEmicizumab prophylaxisHemophilia ASevere HAWTP thresholdStandard careProphylactic factor replacementUS societal perspectiveInfants Aged 0Markov cohort modelLifetime time horizonCost-effectiveness ratioProbabilistic sensitivity analysesFVIII inhibitorsBleeding riskFactor replacementSevere complicationsEmicizumabAccess placementCentral venous access placementPrimary outcomeProphylaxisNeurological disability
2024
Cost-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
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