2025
Prophylactic 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
2023
Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States
Ito S, Potnis K, Allen C, Richmond R, Waldron C, Viswanathan G, Bona R, Goshua G. Equitable Care for Severe Hemophilia_A: Distributional Cost-Effectiveness of Prophylactic Weekly Efanesoctocog Alfa Versus Standard-Care Factor VIII in Patients with Severe Hemophilia_A in the United States. Blood 2023, 142: 492. DOI: 10.1182/blood-2023-190420.Peer-Reviewed Original ResearchIncremental cost-effectiveness ratioSevere hemophilia A.Quality-adjusted life expectancySevere haemophilia AHemophilia AFactor VIIIDistributional cost-effectiveness analysisHemophilia A.Chronic arthropathyOpen-label multicenter studyCoagulation factor replacementLifelong prophylactic treatmentCare of patientsCost-effectiveness ratioUSD/QALYProbabilistic sensitivity analysesCost-effectiveness analysisPackage insert informationDeterministic sensitivity analysesWeekly prophylaxisPrimary outcomeSecondary outcomesWeekly dosingFactor replacementIntracranial hemorrhage
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