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 disabilityProphylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A : A Cost-Effectiveness Analysis.
Ito S, Potnis K, Harvey J, Sra M, Bewersdorf J, Bona R, Krumholz H, Cuker A, Pandya A, Goshua G. Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in People Living With Severe Hemophilia A : A Cost-Effectiveness Analysis. Annals Of Internal Medicine 2025, 178: 819-828. PMID: 40258278, PMCID: PMC12170149, DOI: 10.7326/annals-24-02749.Peer-Reviewed Original ResearchConceptsEquity weightsSevere hemophilia AInequality aversion parameterFactor VIIIHemophilia AAversion parameterCost-effectiveness analysisEquity standardsCost-effectiveness of prophylaxisTime horizonOnce-weekly prophylaxisPriceAnnualized bleeding rateIncremental cost-effectiveness ratioFactor VIII prophylaxisScenario analysisCost-effectiveness ratioUnited StatesLifetime QALYsBleeding rateProphylaxisNational Institutes of HealthCost-effectiveInstitutes of HealthAlfa
2024
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 0
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