2024
Characterizing an Underdiagnosed Condition: Hemophilia in Females
Restrepo V, Prozora S, Louizos E, Van Doren L, Bona R. Characterizing an Underdiagnosed Condition: Hemophilia in Females. Blood 2024, 144: 2584-2584. DOI: 10.1182/blood-2024-210735.Peer-Reviewed Original ResearchPost-partum hemorrhageHeavy menstrual bleedingIron deficiency anemiaLow factor levelsClotting factor levelsHemophilia treatment centersHaemophilia carriersBlood transfusionHemophilia RegistryFactor deficiencyFactor levelsRates of heavy menstrual bleedingFrequency of blood transfusionLife-threatening bleedingFactor replacement therapyManagement of HCAnti-fibrinolytic agentDiagnosis of HCAge of diagnosisHemophilia B carriersYale-New Haven HospitalHealthcare disparitiesNormal factor VIIIHemophilia A carriersFactor activity
2023
Cost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease
Waldron C, Ito S, Wang D, Allen C, Viswanathan G, Bona R, Cuker A, Goshua G. Cost-Effectiveness of Long-Term Prophylaxis with Plasma-Derived Vs. Recombinant Von Willebrand Factor in Severe Von Willebrand Disease. Blood 2023, 142: 3698. DOI: 10.1182/blood-2023-188018.Peer-Reviewed Original ResearchSevere von Willebrand diseaseLong-term prophylaxisIncremental net monetary benefitVon Willebrand diseasePlasma-derived von Willebrand factorIncremental cost-effectiveness ratioNet monetary benefitVon Willebrand factorProbabilistic sensitivity analysesDeterministic sensitivity analysesSevere haemophiliaWillebrand factorRecombinant von Willebrand factorWillebrand diseasePhase 3 clinical trialsHealth resource utilizationFactor replacement therapyFrequency of infusionsSubset of patientsCost of prophylaxisType 3 von Willebrand diseaseFactor replacement productsSimilar significant improvementsSevere phenotypeNon-significant reduction