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
2022
Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study
Krishnamurti L, Arnold S, Haight A, Abraham A, Guilcher G, John T, Bakshi N, Shenoy S, Syrjala K, Martin P, Chaudhury S, Eames G, Olowoselu O, Hsieh M, De La Fuente J, Kasow K, Stenger E, Mertens A, El-Rassi F, Lane P, Shaw B, Meacham L, Archer D. Sickle Cell Transplantation Evaluation of Long-term and Late Effects Registry (STELLAR) to Compare Long-term Outcomes After Hematopoietic Cell Transplantation to Those in Siblings Without Sickle Cell Disease and in Nontransplanted Individuals With Sickle Cell Disease: Design and Feasibility Study. JMIR Research Protocols 2022, 11: e36780. PMID: 35793124, PMCID: PMC9301564, DOI: 10.2196/36780.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseINTERNATIONAL REGISTERED REPORT IDENTIFIERLong-term outcomesCell diseaseTransplantation evaluationCell transplantationLate effectsPost-HCT patientsYear post-HCTFeasibility of recruitmentHealth-related qualityMarrow Transplant ResearchElectronic pain diaryChronic graftNontransplanted individualsPost-HCTHost diseasePain diaryBlood pressureDaily painInternational BloodHandgrip testHip circumferenceSexual function
2021
Hematopoietic Cell Transplantation Outcomes among Medicaid and Privately Insured Patients with Sickle Cell Disease
Mupfudze T, Meyer C, Preussler J, Mau L, Bolon Y, Steinert P, Arnold S, Saber W, Krishnamurti L. Hematopoietic Cell Transplantation Outcomes among Medicaid and Privately Insured Patients with Sickle Cell Disease. Transplantation And Cellular Therapy 2021, 27: 685.e1-685.e8. PMID: 33895405, PMCID: PMC8680220, DOI: 10.1016/j.jtct.2021.04.009.Peer-Reviewed Original ResearchConceptsEvent-free survivalSickle cell diseaseHematopoietic cell transplantation outcomesSignificant between-group differencesGraft failureOverall survivalChronic GVHDCell transplantation outcomesBetween-group differencesAcute GVHDCumulative incidenceAlloHCT outcomesTransplantation outcomesInsurance statusCell diseaseAllogeneic hematopoietic cell transplantation outcomesLower event-free survivalPrivate insuranceRecipients of MedicaidProportion of patientsRetrospective multicenter studyKaplan-Meier methodPrivate insurance groupMarrow Transplant ResearchHealth insurance status
2019
Excellent Overall Survival and Low Incidence of Late Effects in Patients Undergoing Allogeneic Hematopoietic Cell Transplant for Sickle Cell Disease: A Report from the Center for International Blood and Marrow Transplant Research (CIBMTR)
Stenger E, Phelan R, Shaw B, Battiwalla M, Bo-Subait S, Brazauskas R, Buchbinder D, Hamilton B, Shenoy S, Krishnamurti L. Excellent Overall Survival and Low Incidence of Late Effects in Patients Undergoing Allogeneic Hematopoietic Cell Transplant for Sickle Cell Disease: A Report from the Center for International Blood and Marrow Transplant Research (CIBMTR). Blood 2019, 134: 697. DOI: 10.1182/blood-2019-128797.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseAcute chest syndromeMarrow Transplant ResearchLate effectsCumulative incidenceRisk factorsChest syndromeInternational BloodAvascular necrosisMultivariable analysisUnrelated donorsPatient populationTransplant ResearchCell diseaseAllogeneic hematopoietic cell transplantMyeloablative hematopoietic cell transplantationVaso-occlusive pain crisesAllogeneic hematopoietic cell transplantationDiagnosis of SCDMultivariable Cox regression analysisFirst hematopoietic cell transplantationOlder ageExcellent overall survivalHematopoietic cell transplant
2016
Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation
Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle J, Zecca M, Walters M, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier J, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M. Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation. Blood 2016, 129: 1548-1556. PMID: 27965196, PMCID: PMC5356458, DOI: 10.1182/blood-2016-10-745711.Peer-Reviewed Original ResearchConceptsEvent-free survivalSickle cell diseaseGraft failureCell transplantationHLA-identical sibling transplantationReduced-intensity conditioning regimensHLA-identical sibling transplantsHematopoietic stem cell transplantationMyeloablative conditioning regimenHematopoietic cell transplantationStem cell transplantationCox regression modelMarrow Transplant ResearchBenefit of transplantStem cell sourceSibling transplantationConditioning regimenPrimary endpointConditioning regimensMost patientsOverall survivalSibling transplantsCurative therapyInternational BloodMedian age
2015
Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease
Walters M, De Castro L, Sullivan K, Krishnamurti L, Kamani N, Bredeson C, Neuberg D, Hassell K, Farnia S, Campbell A, Petersdorf E. Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2015, 22: 207-211. PMID: 26500093, PMCID: PMC5031360, DOI: 10.1016/j.bbmt.2015.10.017.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseCell transplantationCell diseaseSurvival rateEvent-free survival rateTransplant-related complicationsOverall survival rateMarrow Transplant RegistryMarrow Transplant ResearchRisk of mortalityLong-term qualityRate of survivalNontransplant cohortHost diseaseTransplant RegistryOverall survivalProspective trialInternational BloodTransplant ResearchEuropean BloodMortality riskSuitable donorTherapeutic valueStrong recommendations