2024
Cost-effectiveness of iptacopan in paroxysmal nocturnal hemoglobinuria
Ito S, Chetlapalli K, Wang D, Potnis K, Richmond R, Krumholz H, Lee A, Cuker A, Goshua G. Cost-effectiveness of iptacopan in paroxysmal nocturnal hemoglobinuria. Blood 2024 PMID: 39374533, DOI: 10.1182/blood.2024025176.Peer-Reviewed Original ResearchStandard-of-careParoxysmal nocturnal hemoglobinuriaIncremental net monetary benefitNocturnal hemoglobinuriaComplement-mediated hemolytic anemiaTreating paroxysmal nocturnal hemoglobinuriaComplement C5 inhibitor eculizumabPhase 3 studyQuality-adjusted life expectancyRare blood disorderComprehensive cost-effectiveness analysisProbabilistic sensitivity analysesCost-saving thresholdsC5 inhibitor eculizumabNet monetary benefitPersistent anemiaIptacopanExtravascular hemolysisIntravenous infusionMonotherapyHemolytic anemiaAnemia resolutionC5 inhibitionFDA approvalPrimary outcomeDecreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource
Ito S, Pandya A, Hauser R, Krishnamurti L, Stites E, Tormey C, Krumholz H, Hendrickson J, Goshua G. Decreasing alloimmunization‐specific mortality in sickle cell disease in the United States: Cost‐effectiveness of a shared transfusion resource. American Journal Of Hematology 2024, 99: 570-576. PMID: 38279581, DOI: 10.1002/ajh.27211.Peer-Reviewed Original ResearchSickle cell diseaseDelayed hemolytic transfusion reactionQuality-adjusted life expectancyAlloimmunized patientsPatient populationRed blood cell alloimmunizationCell diseaseCost-effective interventionMedical expenditure of patientsHealth system perspectiveExpenditure of patientsIncremental cost-effectiveness ratioHemolytic transfusion reactionsUnited StatesMarkov cohort simulationCost-effectiveAverage patient populationCost-effectiveness ratioBirth cohortAnalytical time horizonAntibody historyCohort simulationTransfusionTransfusion reactionsLife expectancy
2023
Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States
Goshua G, Ito S, Chetlapalli K, Potnis K, Calhoun C, Krishnamurti L, Krumholz H, Pandya A. Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States. Blood 2023, 142: 490. DOI: 10.1182/blood-2023-191072.Peer-Reviewed Original ResearchSickle cell diseaseIncremental cost-effectiveness ratioDistributional cost-effectiveness analysisPediatric patientsCell diseaseCost-effectiveness analysisDisease severityHealth resource utilization dataPediatric Health Information SystemGene therapyJustifiable treatment optionTransplant-related mortalityVaso-occlusive crisisExpert clinical experienceMarrow Transplant ResearchSubstantial mortality riskVisual analog scaleQuality-adjusted life expectancyConcomitant riskCost-effectiveness ratioResource utilization dataCost-effectiveness frontierHost diseaseMaximum patientsOpioid therapyEquitable 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 hemorrhageSecond-Line Therapy Sequencing in Primary Warm Autoimmune Hemolytic Anemia: Splenectomy-Rituximab Versus Rituximab-Splenectomy
Waldron C, Ito S, Wang D, Viswanathan G, Butt A, Goshua G. Second-Line Therapy Sequencing in Primary Warm Autoimmune Hemolytic Anemia: Splenectomy-Rituximab Versus Rituximab-Splenectomy. Blood 2023, 142: 2317. DOI: 10.1182/blood-2023-189957.Peer-Reviewed Original ResearchWarm autoimmune hemolytic anemiaSecond-line therapyAutoimmune hemolytic anemiaAdult patientsHemolytic anemiaSecond-line treatmentFirst-line treatmentPrevention of thrombosisDegree of anemiaLaparoscopic surgical techniquesPatient's disease statusQuality-adjusted life expectancyRate of infectionProbabilistic sensitivity analysesNet monetary benefitRituximab cyclesTherapy sequencingComplete remissionPartial remissionPerioperative mortalityPrimary outcomeTherapeutic sequencingLaparoscopic techniquePreferred treatmentRelapse riskGlobal Health Resource Utilization and Cost-Effectiveness of Therapeutics and Diagnostics in Immune Thrombotic Thrombocytopenic Purpura (TTP)
Butt A, Allen C, Purcell A, Ito S, Goshua G. Global Health Resource Utilization and Cost-Effectiveness of Therapeutics and Diagnostics in Immune Thrombotic Thrombocytopenic Purpura (TTP). Journal Of Clinical Medicine 2023, 12: 4887. PMID: 37568288, PMCID: PMC10420213, DOI: 10.3390/jcm12154887.Peer-Reviewed Original Research