Featured Publications
Cost‐effectiveness of second‐line therapies in adults with chronic immune thrombocytopenia
Goshua G, Sinha P, Kunst N, Pischel L, Lee AI, Cuker A. Cost‐effectiveness of second‐line therapies in adults with chronic immune thrombocytopenia. American Journal Of Hematology 2022, 98: 122-130. PMID: 35147241, PMCID: PMC9365880, DOI: 10.1002/ajh.26497.Peer-Reviewed Original ResearchConceptsThrombopoietin receptor agonistsChronic immune thrombocytopeniaSecond-line therapyImmune thrombocytopeniaEarly useCurrent practice trendsProbabilistic sensitivity analysesCost-effective treatmentFirst cost-effectiveness analysisCost-effectiveness analysisU.S. health systemLack of evidenceEarly splenectomyHematology (ASH) guidelinesReceptor agonistCurrent guidelinesSplenectomyClinical practicePractice trendsTherapyHealth systemCost-effective strategyRituximabThrombocytopeniaAmerican Society
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