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-VEGF