2024
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
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 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
Cost-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 unitSecond-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 risk