2024
Identifying Patients with Erythrocytosis Not Requiring JAK2 Testing: External Validation and Decision Analysis of the Jakpot Prediction Rule
Lam B, Ito S, Ryu J, O'Sullivan K, Potnis K, Lu R, Bidikian A, Freed J, La J, Goshua G. Identifying Patients with Erythrocytosis Not Requiring JAK2 Testing: External Validation and Decision Analysis of the Jakpot Prediction Rule. Blood 2024, 144: 3669-3669. DOI: 10.1182/blood-2024-198033.Peer-Reviewed Original ResearchNegative predictive valueBeth Israel Deaconess Medical CenterClinical prediction rulePolycythemia veraIncremental net monetary benefitHealth resource utilizationPV patientsMyeloproliferative neoplasmsVA cohortTest characteristicsUS health system perspectivePrediction ruleAssociated with increased riskNon-fatal cardiovascular eventsExternal validation cohortAssociated with increased risk of strokeRandomized trial dataMarkov cohort modelLifetime time horizonProbabilistic sensitivity analysesRisk of strokePersistent erythrocytosisMedian ageHematologic malignanciesDiagnostic workupCost-Effectiveness of Universal Duffy Testing at 9-12 Months: An Effort Towards Equity in Interpreting Absolute Neutrophil Counts (ANC)
Merz L, Ito S, Walkovich K, Achebe M, Van Havre N, Frustace P, Goshua G. Cost-Effectiveness of Universal Duffy Testing at 9-12 Months: An Effort Towards Equity in Interpreting Absolute Neutrophil Counts (ANC). Blood 2024, 144: 162. DOI: 10.1182/blood-2024-202605.Peer-Reviewed Original ResearchIncremental net monetary benefitAbsolute neutrophil countWork-upBirth cohortBase-caseCenters for Medicare & Medicaid ServicesMitigate patient harmCost-effective strategyUniversal testingUS health system perspectiveThreshold sensitivity analysisDiscounted quality-adjusted life yearsInfectious work-upHealth system perspectiveNo testBone marrow biopsyIncreased risk of infectionCross-sectional studyAnti-neutrophil antibodiesDiagnostic work-upQuality-adjusted life yearsUniversal testing strategiesUnited StatesCost-effectiveMarkov cohort modelDeciding between Multiple Curative Options in Sickle Cell Disease: Cost-Effectiveness of Non-Myeloablative/Reduced Intensity Conditioning Haploidentical Allo-HSCT Vs Gene Therapy Vs Standard of Care in Adult Patients with Sickle Cell Disease
Chetlapalli K, Butt A, Ito S, Wang D, Calhoun C, Krishnamurti L, Pandya A, Goshua G. Deciding between Multiple Curative Options in Sickle Cell Disease: Cost-Effectiveness of Non-Myeloablative/Reduced Intensity Conditioning Haploidentical Allo-HSCT Vs Gene Therapy Vs Standard of Care in Adult Patients with Sickle Cell Disease. Blood 2024, 144: 601-601. DOI: 10.1182/blood-2024-203234.Peer-Reviewed Original ResearchChronic graft-versus-host diseaseSickle cell diseaseQuality-adjusted life yearsPost-transplant cyclophosphamideReduced intensity conditioningAllo-HSCTGene therapyCurative optionIncremental net monetary benefitProbabilistic sensitivity analysesMyeloablative conditioningHaploidentical donorsCell diseaseAdult patientsExpansion of donor poolUS health system perspectiveCenter for International Blood and Marrow Transplant ResearchGraft-versus-host diseaseEuropean Society for BloodSickle cell disease severityUS commercially insured patientsCost-effective therapeutic optionCost-effectiveness analysisAllo-HSCT outcomesDonor allo-HSCTCost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States
Potnis K, Ito S, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Zeidan A, Bewersdorf J, Goshua G. Cost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States. Blood 2024, 144: 3644. DOI: 10.1182/blood-2024-204312.Peer-Reviewed Original ResearchSevere aplastic anemiaTreatment of severe aplastic anemiaQuality-adjusted life yearsAplastic anemiaIncremental net monetary benefitDeterministic sensitivity analysisProbabilistic sensitivity analysesImmunosuppressive therapyNewly diagnosed severe aplastic anemiaCost-effectiveness of eltrombopagOral thrombopoietin receptor agonistPatients treated with eltrombopagUntreated severe aplastic anemiaHematopoietic stem cell transplantationCost-effective therapeutic strategyDevelopment of adverse eventsLonger-term follow-up dataRACE trialsLater-line therapyThrombopoietin receptor agonistsPhase I/II trialSecond-line therapyStem cell transplantationU.S. payer perspectiveFirst-line treatmentCost-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 outcomeCost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia
Wang D, Ito S, Waldron C, Butt A, Zhang E, Krumholz H, Al-Samkari H, Goshua G. Cost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia. Blood Advances 2024, 8: 2835-2845. PMID: 38537061, PMCID: PMC11176968, DOI: 10.1182/bloodadvances.2024012589.Peer-Reviewed Original ResearchIncremental net monetary benefitHereditary hemorrhagic telangiectasiaStandard-of-careBevacizumab therapyWillingness-to-payNet monetary benefitHemorrhagic telangiectasiaWillingness-to-pay thresholdsStandard-of-care strategiesCare of patientsCost-effectiveness analysisAnti-VEGF bevacizumabSystemic antiangiogenic therapyCurrent standard-of-careMonetary benefitsTime horizonFormulary placementHealthcare resource utilizationStandard of careLifetime time horizonPatient quality-of-lifeScenario analysisProbabilistic sensitivity analysesIV bevacizumabAnti-VEGFCost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura
Allen C, Ito S, Butt A, Purcell A, Richmond R, Tormey C, Krumholz H, Cuker A, Goshua G. Cost-effectiveness of rapid vs in-house vs send-out ADAMTS13 testing for immune thrombotic thrombocytopenic purpura. Blood Advances 2024, 8: 2279-2289. PMID: 38502197, PMCID: PMC11116991, DOI: 10.1182/bloodadvances.2024012608.Peer-Reviewed Original ResearchImmune thrombotic thrombocytopenic purpuraPLASMIC scoreThrombotic thrombocytopenic purpuraThrombocytopenic purpuraADAMTS13 testingIncremental net monetary benefitPer-patient cost savingsTherapeutic plasma exchangeBase-case analysisMarkov cohort simulationProbabilistic sensitivity analysesAmount of QALYEmpirical therapyADAMTS13 assaysPlasma exchangeEmpirical treatmentCaplacizumabFRET-based assayPrimary outcomePatientsNet monetary benefitCohort simulationCost-effectiveness evaluationPurpuraTesting strategies
2023
Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States
Ito S, Chetlapalli K, Potnis K, Richmond R, Wang D, Lee A, Goshua G. Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States. Blood 2023, 142: 5042. DOI: 10.1182/blood-2023-188063.Peer-Reviewed Original ResearchTreatment-experienced patientsParoxysmal nocturnal hemoglobinuriaIncremental net monetary benefitIncremental cost-effectiveness ratioNursing timeExtravascular hemolysisNocturnal hemoglobinuriaPhase III study resultsTreatment-experienced adult patientsTransfusion-associated adverse eventsCost-effective therapeutic optionAnti-C5 therapyComplement inhibition therapyComplement inhibitor therapyHealth resource utilizationCare of patientsIndirect medical costsComplement component 5Clinical trial dataLife-threatening hematological disorderCost-effectiveness ratioBreakthrough therapy designationProbabilistic sensitivity analysesFirst cost-effectiveness analysisCost-effectiveness analysisCost-Effectiveness of Sutimlimab Versus Standard-of-Care in Transfusion Dependent Patients with Primary Cold Agglutinin Disease in the United States
Ito S, Wang D, Purcell A, Chetlapalli K, Lee A, Cuker A, Goshua G. Cost-Effectiveness of Sutimlimab Versus Standard-of-Care in Transfusion Dependent Patients with Primary Cold Agglutinin Disease in the United States. Blood 2023, 142: 2316. DOI: 10.1182/blood-2023-187270.Peer-Reviewed Original ResearchPrimary cold agglutinin diseaseCold agglutinin diseaseIncremental cost-effectiveness ratioTransfusion-dependent patientsIncremental net monetary benefitProbabilistic sensitivity analysesDistributional cost-effectiveness analysisAnemia severityBody weightPhase 3 clinical studiesHealth resource utilizationRed blood cell destructionDisease-specific mortalityHistory of transfusionAutoimmune hemolytic anemiaMarkov state transition modelCare of patientsHumanized monoclonal antibodyLower body weightBlood cell destructionCost-effectiveness ratioFDA package insertsCold-reactive antibodiesBase-case analysisCost-effectiveness analysisLongitudinal Systemic Bevacizumab Therapy Produces Cost Savings and Improves Quality-Adjusted Life Expectancy in the Care of Patients with Hereditary Hemorrhagic Telangiectasia with Moderate-to-Severe Bleeding
Wang D, Ito S, Waldron C, Butt A, Zhang E, Krumholz H, Al-Samkari H, Goshua G. Longitudinal Systemic Bevacizumab Therapy Produces Cost Savings and Improves Quality-Adjusted Life Expectancy in the Care of Patients with Hereditary Hemorrhagic Telangiectasia with Moderate-to-Severe Bleeding. Blood 2023, 142: 2312. DOI: 10.1182/blood-2023-185340.Peer-Reviewed Original ResearchHereditary hemorrhagic telangiectasiaIncremental net monetary benefitIncremental cost-effectiveness ratioEmergency department visitsCare of patientsHemostatic proceduresIron infusionPatient timeBevacizumab therapyDepartment visitsRBC transfusionHemorrhagic telangiectasiaVascular endothelial growth factor inhibitionRed blood cell transfusionEQ-5D index valuesCommon hereditary bleeding disorderLocal hemostatic proceduresRBC transfusion needSystemic bevacizumab therapyHealth resource utilizationBlood cell transfusionWorld Health Organization VigiBaseLong-term followGrowth factor inhibitionCost-saving interventionCost-Effectiveness of Rapid Hemosil Versus in-House and Send-out ADAMTS13 Activity Testing in the Care of Patients with Immune Thrombotic Thrombocytopenic Purpura
Allen C, Ito S, Butt A, Purcell A, Richmond R, Cuker A, Goshua G. Cost-Effectiveness of Rapid Hemosil Versus in-House and Send-out ADAMTS13 Activity Testing in the Care of Patients with Immune Thrombotic Thrombocytopenic Purpura. Blood 2023, 142: 368. DOI: 10.1182/blood-2023-184927.Peer-Reviewed Original ResearchImmune thrombotic thrombocytopenic purpuraTherapeutic plasma exchangeIncremental cost-effectiveness ratioThrombotic thrombocytopenic purpuraPLASMIC scoreIncremental net monetary benefitProbabilistic sensitivity analysesPlasma exchangeThrombocytopenic purpuraMedical intensive care unitADAMTS13 activity testingIntensive care unitCare of patientsActivity levelsHigh pretest probabilityClinical trial dataCost-effectiveness ratioCost-effectiveness evaluationDeterministic sensitivity analysesNet monetary benefitEmpiric treatmentAdverse eventsPrimary outcomeIll adultsCare unitCost-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