2024
Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease
Walters M, Eapen M, Liu Y, El Rassi F, Waller E, Levine J, Strouse J, Antin J, Parikh S, Bakshi N, Dampier C, Jaroscak J, Bergmann S, Wong T, Kota V, Pace B, Lekakis L, Lulla P, Nickel R, Kasow K, Popat U, Smith W, Yu L, DiFronzo N, Geller N, Kamani N, Klings E, Hassell K, Mendizabal A, Sullivan K, Neuberg D, Krishnamurti L. Hematopoietic Cell Transplant compared with Standard Care in Adolescents and Young Adults with Sickle Cell Disease. Blood Advances 2024 PMID: 39471440, DOI: 10.1182/bloodadvances.2024013926.Peer-Reviewed Original ResearchHematopoietic cell transplantationVaso-occlusive painStandard of careSickle cell diseaseCell transplantationCell diseaseSCD-related eventsHLA-matched siblingTransplant-related mortalityPeriod of follow-upSurvival 2 yearsIntent-to-treat principleComparison of survivalDisease-modifying therapiesDisease-related mortalityUnrelated donorPrimary endpointCurative potentialTreatment armsDonor searchFollow-upCompare outcomesEligibility confirmationFunctional outcomesSecondary outcomes
2023
Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States
Goshua G, Ito S, Chetlapalli K, Potnis K, Calhoun C, Krishnamurti L, Krumholz H, Pandya A. Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States. Blood 2023, 142: 490. DOI: 10.1182/blood-2023-191072.Peer-Reviewed Original ResearchSickle cell diseaseIncremental cost-effectiveness ratioDistributional cost-effectiveness analysisPediatric patientsCell diseaseCost-effectiveness analysisDisease severityHealth resource utilization dataPediatric Health Information SystemGene therapyJustifiable treatment optionTransplant-related mortalityVaso-occlusive crisisExpert clinical experienceMarrow Transplant ResearchSubstantial mortality riskVisual analog scaleQuality-adjusted life expectancyConcomitant riskCost-effectiveness ratioResource utilization dataCost-effectiveness frontierHost diseaseMaximum patientsOpioid therapy
2018
Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias
Locatelli F, Ruggeri A, Merli P, Naik S, Agarwal R, Aquino V, Jacobsohn D, Qasim W, Nemecek E, Krishnamurti L, Manwani D, Kuhn M, Kapoor N. Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias. Blood 2018, 132: 307. DOI: 10.1182/blood-2018-99-119481.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationRelapse-free survivalHLA-haploidentical hematopoietic stem cell transplantationEfficacy-evaluable populationHaplo-HSCTComplete remissionOverall survivalAcute leukemiaΑβ TB cellsEvaluable patientsPediatric patientsT cellsLower riskUnrelated donor hematopoietic stem cell transplantationDonor hematopoietic stem cell transplantationHaploidentical hematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationBetter overall clinical responseSteroid-resistant acute GVHDConventional steroid therapyOverall clinical responseSteroid-resistant GVHDTransplant-related mortalityPrimary graft failure
2016
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approaches
Arnold S, Bhatia M, Horan J, Krishnamurti L. Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approaches. British Journal Of Haematology 2016, 174: 515-525. PMID: 27255787, DOI: 10.1111/bjh.14167.Peer-Reviewed Original ResearchConceptsHaematopoietic stem cell transplantSickle cell diseaseCell diseaseAllogeneic haematopoietic stem cell transplantOnly available curative therapyHaematopoietic stem cell transplantationAlternative donor transplantsAvailable curative therapyCurrent maintenance therapiesTransplant-related mortalityDisease-free survivalIncidence of rejectionStem cell transplantStem cell transplantationHuman leucocyte antigenPool of donorsComplications persistHaploidentical donorsMaintenance therapyConditioning regimensCurative optionDonor transplantsSibling transplantsSupportive careCurative therapy
2010
Long-Term Follow-up of Adults with Severe Sickle Cell Disease After Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning
Biernacki M, Okam M, Shenoy S, Krishnamurti L, Horwitz M, Neuberg D, Antin J, Wu C. Long-Term Follow-up of Adults with Severe Sickle Cell Disease After Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning. Blood 2010, 116: 261. DOI: 10.1182/blood.v116.21.261.261.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationSickle cell diseaseSevere sickle cell diseaseLimited chronic GVHDStem cell transplantationLong-term outcomesRIC-HSCTAdult patientsChronic GVHDGraft lossImmunosuppressive medicationsCell transplantationCell diseaseMyeloablative hematopoietic stem cell transplantationMinimal transplant-related toxicityPeripheral blood stem cellsStable donor chimerismSustained donor engraftmentTotal lymphoid irradiationTransplant-related mortalityIntensity conditioning regimensTransplant-related toxicityDisease-free survivalNew adult patientsMajority of patients