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 therapyParental perspective on the risk of infertility and fertility preservation options for children and adolescents with sickle cell disease considering hematopoietic stem cell transplantation
Sinha C, Meacham L, Bakshi N, Ross D, Krishnamurti L. Parental perspective on the risk of infertility and fertility preservation options for children and adolescents with sickle cell disease considering hematopoietic stem cell transplantation. Pediatric Blood & Cancer 2023, 70: e30276. PMID: 37051746, PMCID: PMC10544372, DOI: 10.1002/pbc.30276.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationRisk of infertilitySickle cell diseaseStem cell transplantationFertility preservationCell transplantationCell diseaseMajor long-term complicationPrimary caregiversIdentical related donorsDisease-free survivalFertility preservation optionsFertility preservation proceduresLong-term complicationsHuman leukocyte antigenConditioning regimenHost diseaseRelated donorsLeukocyte antigenPreservation optionsHCT physiciansSurvival rateAvailable HLAEleven participantsInfertility
2022
Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance
Stenger E, Xiang Y, Wetzel M, Gillespie S, Chellapandian D, Shah R, Arnold S, Bhatia M, Chaudhury S, Eckrich M, Kanter J, Kasow K, Krajewski J, Nickel R, Ngwube A, Olson T, Rangarajan H, Wobma H, Guilcher G, Horan J, Krishnamurti L, Shenoy S, Abraham A. Long-Term Organ Function After HCT for SCD: A Report From the Sickle Cell Transplant Advocacy and Research Alliance. Transplantation And Cellular Therapy 2022, 29: 47.e1-47.e10. PMID: 36273784, DOI: 10.1016/j.jtct.2022.10.012.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseOrgan functionMultivariable analysisBrain magnetic resonance imagingCentral nervous system indicationsPost-HCT patientsRelated bone marrowSevere acute GVHDPredictors of dysfunctionLong-term survivalMagnetic resonance imagingSevere clinical phenotypeAcute GVHDChronic graftIntense conditioningHost diseaseMyeloablative conditioningNeurologic eventsOvert strokeRetrospective cohortMedian ageOrgan dysfunctionCardiac dysfunctionCell transplantationDecision-making about gene therapy in transfusion dependent thalassemia
Quarmyne M, Ross D, Sinha C, Bakshi N, Boudreaux J, Krishnamurti L. Decision-making about gene therapy in transfusion dependent thalassemia. BMC Pediatrics 2022, 22: 536. PMID: 36085025, PMCID: PMC9461218, DOI: 10.1186/s12887-022-03598-3.Peer-Reviewed Original ResearchConceptsTransfusion-dependent thalassemiaPatient/family knowledgeDependent thalassemiaTransfusion independenceTreatment optionsStudy participantsGene therapyFamily knowledgeElimination of transfusionsFrequency of transfusionMorbidity/mortalityStem cell transplantationLong-term outcomesPreferred treatment modalityPromising treatment optionBackgroundHematopoietic stem cell transplantationPatients/familiesMethodsParents of childrenCurative intentTransfusion reductionHost diseaseDonor HSCTParents of childrenCell transplantationMean ageSickle 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
Mobility and Muscle Strength in Recipients of Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from Sickle Transplant Evaluation of Longterm and Late Effects Registry (STELLaR)
Veludhandi A, John T, Bakshi N, Arnold S, Bhatia M, Lane P, Meacham L, Khemani K, Chaudhury S, Olowoselu F, Kasow K, Hsieh M, Abraham A, Guilcher G, Krishnamurti L. Mobility and Muscle Strength in Recipients of Hematopoietic Cell Transplantation for Sickle Cell Disease: A Preliminary Report from Sickle Transplant Evaluation of Longterm and Late Effects Registry (STELLaR). Blood 2021, 138: 3030. DOI: 10.1182/blood-2021-152383.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseMuscle strengthCGVHD symptomsHCT recipientsCell transplantationPhysical fitnessYoung adultsPost-HCTChronic graftHost diseaseMarrow transplantationGrip strengthTUG scoresRisk factorsCell diseasePathophysiology of SCDJournal of ParenteralCardiometabolic risk factorsLong-term survivorsHand-grip dynamometryHand grip strengthCross-sectional studyHandgrip strength testYears of age
2019
Application of Parafilm As a Physical Barrier on CVC Connections Is Feasible and May Reduce Clabsi Among Pediatric HCT Patients
Stenger E, Newton J, Leong T, Kendrick L, McManus L, Rooke C, Krishnamurti L. Application of Parafilm As a Physical Barrier on CVC Connections Is Feasible and May Reduce Clabsi Among Pediatric HCT Patients. Transplantation And Cellular Therapy 2019, 25: s131. DOI: 10.1016/j.bbmt.2018.12.416.Peer-Reviewed Original ResearchPediatric HCT patientsCentral venous cathetersHCT patientsCLABSI ratesLong-term central venous cathetersExternal central venous cathetersPediatric patients ages 0Pilot quality improvement initiativeHematopoietic cell transplantation patientsYounger aged patientsHistorical control patientsPatients ages 0Cell transplantation patientsHistorical control groupNon-malignant diseasesQuality improvement initiativesCLABSI occurrenceCVC hubsHost diseaseProlonged immunosuppressionTransplantation patientsAutologous HCTCohort studyControl patientsHCT outcomes
2017
Bone Marrow–Derived Mesenchymal Stromal Cells from Patients with Sickle Cell Disease Display Intact Functionality
Stenger E, Chinnadurai R, Yuan S, Garcia M, Arafat D, Gibson G, Krishnamurti L, Galipeau J. Bone Marrow–Derived Mesenchymal Stromal Cells from Patients with Sickle Cell Disease Display Intact Functionality. Transplantation And Cellular Therapy 2017, 23: 736-745. PMID: 28132869, PMCID: PMC5390328, DOI: 10.1016/j.bbmt.2017.01.081.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAnemia, Sickle CellCell CommunicationCell Culture TechniquesCell ProliferationChildChild, PreschoolFemaleHealthy VolunteersHematopoietic Stem Cell TransplantationHumansImmunophenotypingIndoleamine-Pyrrole 2,3,-DioxygenaseMaleMesenchymal Stem Cell TransplantationMesenchymal Stem CellsTransplantation, AutologousYoung AdultConceptsSickle cell diseaseHematopoietic cell transplantationMesenchymal stromal cellsAutologous mesenchymal stromal cellsBone marrowTime of HCTAutologous T cell proliferationStromal cellsThird-party mesenchymal stromal cellsBM-derived mesenchymal stromal cellsMajor histocompatibility complex compatibilityExperimental murine modelT cell proliferationSurface marker phenotypeDose-dependent mannerIFN-γ stimulationAmeliorate graftHost diseaseSCD patientsCell transplantationImmunomodulatory pathwaysSCD subjectsCell diseaseHealthy volunteersMurine model
2015
Mesenchymal stromal cells to modulate immune reconstitution early post-hematopoietic cell transplantation
Stenger E, Krishnamurti L, Galipeau J. Mesenchymal stromal cells to modulate immune reconstitution early post-hematopoietic cell transplantation. BMC Immunology 2015, 16: 74. PMID: 26674007, PMCID: PMC4681052, DOI: 10.1186/s12865-015-0135-7.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationMesenchymal stromal cellsCell transplantationPost-hematopoietic cell transplantationStromal cellsHematopoietic stem cell engraftmentPre-clinical studiesStem cell engraftmentHost diseaseImmune reconstitutionClinical trialsAnimal modelsCell engraftmentMultipotent progenitor cellsImmune systemHSC engraftmentCellular therapyProgenitor cellsTransplantationEngraftmentTrialsDual effectCellsEarly evidenceGVHDIndications 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
2005
Matched Sibling Donor Hematopoietic Cell Transplantation for Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen Can Lead To Stable Long Term Engraftment.
Krishnamurti L, Wu C, Baker K, Wagner J. Matched Sibling Donor Hematopoietic Cell Transplantation for Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen Can Lead To Stable Long Term Engraftment. Blood 2005, 106: 3172. DOI: 10.1182/blood.v106.11.3172.3172.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseDaily x 5 daysIntensity conditioning regimenCell diseaseConditioning regimenDay 100Stable long-term engraftmentLong-term engraftmentRIC regimenClinical characteristicsCell transplantationDay 180Term engraftmentHigh-risk sickle cell diseaseDonor hematopoietic cell transplantationLineage-specific chimerism analysisPeripheral blood genomic DNARegimen-Related ToxicitiesAnti-thymocyte globulinGroup of patientsChronic graftDonor erythropoiesisGVHD prophylaxisHost disease