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
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