2025
Initial management of patients with acquired aplastic anemia in the United States: results from a large national claims database
Stempel J, Wang R, Lee A, Zeidan A, Ma X, Podoltsev N. Initial management of patients with acquired aplastic anemia in the United States: results from a large national claims database. Annals Of Hematology 2025, 104: 2507-2515. PMID: 40102305, PMCID: PMC12052795, DOI: 10.1007/s00277-025-06307-z.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationAcquired aplastic anemiaCalcineurin inhibitorsParoxysmal nocturnal hemoglobinuriaTransfusion-dependentTriple therapyAA patientsAplastic anemiaAllogeneic hematopoietic stem cell transplantationTreated with calcineurin inhibitorsTreated with antithymocyte globulinFlow cytometryInitial management of patientsSevere AA patientsStem cell transplantationBone marrow failureImmune-mediated disordersTreatment initiation delayRetrospective cohort studyManagement of patientsDiagnosis to treatmentNational claims databaseAntithymocyte globulinSibling donorMarrow failure
2024
Cost-effectiveness of iptacopan for 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 for paroxysmal nocturnal hemoglobinuria. Blood 2024, 145: 127-140. PMID: 39374533, PMCID: PMC11738035, 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 outcome
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 analysis
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