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-HSCT
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