2023
Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507
Kassim A, Walters M, Eapen M, Ritzau N, Smith M, Solh M, McKinney C, Nieder M, Ross M, Kent M, Abusin G, Mallhi K, Silva J, Shaughnessy P, Kanter J, Haines H, Farah R, Khaled Y, Abraham A, Bollard C, Cooke K, de La Fuente J, Hanna R, Horowitz M, Jordan L, Krishnamurti L, Leifere E, Mahadeo K, Shenoy S, Ritzau N, DeBaun M, Brodsky R. Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507. Blood 2023, 142: lba-4. DOI: 10.1182/blood-2023-192022.Peer-Reviewed Original ResearchPost-transplant cyclophosphamideEvent-free survivalBone marrow transplantSickle cell diseaseSevere sickle cell diseaseTotal body irradiationGraft failureAcute GVHDPost-BMTCell diseaseDay 100Multi-center phase II trialFirst year post-BMTHaploidentical bone marrow transplantVaso-occlusive pain episodesHaploidentical bone marrow transplantationHematopoietic stem cell transplantationChronic transfusion regimenDurable donor engraftmentHemoglobin SS diseaseAcute chest syndromePrimary graft failureSecondary graft failureEnd-organ toxicityPhase II trialParental 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 participantsInfertility379 The Impact of Current Conditioning Regimen, T Cell Depletion and Graft Versus Host Disease Prophylaxis on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease
Shah N, Flagg A, Hamidi R, Hugo H, Deng Y, Krishnamurti L. 379 The Impact of Current Conditioning Regimen, T Cell Depletion and Graft Versus Host Disease Prophylaxis on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2023, 29: s286-s287. DOI: 10.1016/s2666-6367(23)00448-7.Peer-Reviewed Original Research
2021
A Decision Support Tool for Allogeneic Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: Acceptability and Usability Study
Veludhandi A, Ross D, Sinha C, McCracken C, Bakshi N, Krishnamurti L. A Decision Support Tool for Allogeneic Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: Acceptability and Usability Study. JMIR Formative Research 2021, 5: e30093. PMID: 34709190, PMCID: PMC8587189, DOI: 10.2196/30093.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationSickle cell diseaseAllogeneic hematopoietic stem cell transplantationHealth care providersStem cell transplantationCare providersCell transplantationPatient consultsCell diseaseHCT comorbidity indexPhysician-patient collaborationOttawa Decision Support FrameworkChronic blood transfusionsDisease-modifying therapiesTransplant registry dataMost participantsHealth care settingsQuality of lifePatient-specific measuresCurative intentComorbidity indexHCT outcomesBlood transfusionRisk factorsPatient management386 Cryptogenic Organizing Pneumonia Following HLA Identical Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: A Case Series of Three Pediatric Patients
Bauchat A, Williams K, Simon D, Parikh S, Krishnamurti L. 386 Cryptogenic Organizing Pneumonia Following HLA Identical Hematopoietic Stem Cell Transplantation for Sickle Cell Disease: A Case Series of Three Pediatric Patients. Transplantation And Cellular Therapy 2021, 27: s321-s322. DOI: 10.1016/s2666-6367(21)00412-7.Peer-Reviewed Original Research
2020
Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
Walters M, Locatelli F, Thrasher A, Tisdale J, Orchard P, Duncan C, Kühl J, De Oliveira S, Sauer M, Kulozik A, Yannaki E, Hongeng S, Mapara M, Krishnamurti L, Hermine O, Blanche S, Aubourg P, Smith N, Shi W, Colvin R, McNeil E, Ribeil J, Cavazzana M, Williams D. Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy. Transplantation And Cellular Therapy 2020, 26: s38-s39. DOI: 10.1016/j.bbmt.2019.12.104.Peer-Reviewed Original ResearchTransfusion-dependent β-thalassemiaSickle cell diseaseHematopoietic stem cell transplantationCerebral adrenoleukodystrophyStem cell transplantationAdverse eventsAllo-HSCTSafety profileCell transplantationCell diseaseAllogeneic hematopoietic stem cell transplantationAutologous hematopoietic stem cell transplantationGene addition therapyLentiviral vectorsBusulfan/cyclophosphamideCommon adverse eventsSecondary graft failureRisk of GVHDLong-term followRisk of complicationsΒ-thalassemiaImproved safety profileDP infusionFebrile neutropeniaAutologous HSCT
2019
Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia
Smith A, Schiller G, Vercellotti G, Kwiatkowski J, Krishnamurti L, Esrick E, Williams D, Miller W, Woolfson A, Walters M. Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia. Blood 2019, 134: 3544. DOI: 10.1182/blood-2019-125743.Peer-Reviewed Original ResearchTransfusion-dependent beta thalassemiaPeripheral blood mononuclear cellsSerious adverse eventsHematopoietic stem cell transplantationHuman hematopoietic stem cellsHematopoietic reconstitutionBluebird BioPRBC transfusionPatient 1HbF levelsG-CSFJazz PharmaceuticalsPacked red blood cell transfusionPhase I/II studyAllogeneic hematopoietic stem cell transplantationRed blood cell transfusionHematopoietic stem cellsPhase 1/2 clinical studyBeta thalassemiaInvestigational cell therapySpeakers bureauBlood cell transfusionStem cell transplantationBlood mononuclear cellsCells/Curative Therapies for Sickle Cell Disease.
Khemani K, Katoch D, Krishnamurti L. Curative Therapies for Sickle Cell Disease. Ochsner Journal 2019, 19: 131-137. PMID: 31258425, PMCID: PMC6584191, DOI: 10.31486/toj.18.0044.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationSickle cell diseaseHaploidentical donorsSibling donorsCurative therapyUnrelated donorsCell diseaseHLA-identical sibling donorsRelated haploidentical donorsEvent-free survivalIdentical sibling donorsBone marrow transplantationStem cell transplantationAmelioration of symptomsHuman leukocyte antigenComparative clinical trialsGene therapyUse of HLAStable long-term engraftmentLong-term engraftmentMarrow transplantationRelated donorsSevere morbidityCurative treatmentOrgan damage
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 failureAdministration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD)
Elkeky R, Jacobsohn D, Agarwal R, Naik S, Kapoor N, Krishnamurti L, Slatter M, Galaverna F, Merli P, Aldinger M, Locatelli F. Administration of Rimiducid Following Haploidentical BPX-501 Cells in Children with Malignant or Non-Malignant Disorders Who Develop Graft-Versus-Host-Disease (GvHD). Blood 2018, 132: 2207. DOI: 10.1182/blood-2018-99-119792.Peer-Reviewed Original ResearchNon-malignant disordersHematopoietic stem cell transplantationTreatment of GVHDDonor T cellsΑβ T cellsT cellsHaplo-HSCTImmune recoveryAdoptive transferPediatric patientsMedian timeB cellsAllogeneic hematopoietic stem cell transplantationOverall clinical response rateConventional steroid therapyEfficacy-evaluable populationOnset of GVHDSteroid-refractory GVHDClinical response rateHLA-compatible donorsStem cell transplantStem cell transplantationΑβ T cell receptorAdvisory CommitteeEffective treatment approach
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 ageDevelopment, Implementation, and Testing of a Web Based Decision Aid for Facilitating Shared Decision Making for Disease Modifying Therapies for Sickle Cell Disease
Krishnamurti L, Ross D, Bakshi N, Khemani K, Sinha C, Loewenstein G. Development, Implementation, and Testing of a Web Based Decision Aid for Facilitating Shared Decision Making for Disease Modifying Therapies for Sickle Cell Disease. Blood 2016, 128: 5919. DOI: 10.1182/blood.v128.22.5919.5919.Peer-Reviewed Original ResearchSickle cell diseaseHematopoietic stem cell transplantationClinical trialsCell diseaseClinical decisionPatients/family membersFinal decision aidOttawa Decision Support FrameworkDisease-Modifying TherapiesChronic blood transfusionsStem cell transplantationRandomized clinical trialsDecision aidMix of patientsEligible patientsModifying therapiesLate complicationsBlood transfusionFavorable patientsSubstantial morbidityCell transplantationQualitative interviewsPhysicians' perceptionsPremature mortalitySubstantial burden
2012
Oocyte cryopreservation in a patient with sickle cell disease prior to hematopoietic stem cell transplantation: first report
Dovey S, Krishnamurti L, Sanfilippo J, Gunawardena S, Mclendon P, Campbell M, Alway S, Efymow B, Gracia C. Oocyte cryopreservation in a patient with sickle cell disease prior to hematopoietic stem cell transplantation: first report. Journal Of Assisted Reproduction And Genetics 2012, 29: 265-269. PMID: 22219083, PMCID: PMC3288130, DOI: 10.1007/s10815-011-9698-2.Peer-Reviewed Original ResearchConceptsSickle cell diseaseSevere sickle cell diseaseHematopoietic stem cell transplantationStem cell transplantationVaso-occlusive eventsCell diseaseOvarian stimulationOvarian hyperstimulationFertility preservationCell transplantationOocyte cryopreservationHematopoietic stem cell transplantFertility preservation counselingGnRH antagonist protocolUnique patient populationStem cell transplantYear old femaleSuccessful ovarian stimulationAntagonist protocolCell transplantPatient populationOocyte bankingMulti-disciplinary teamElevated riskOld female
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 patientsHematopoietic Stem Cell Transplantation for Hemoglobinopathies: Current Practice and Emerging Trends
Smiers F, Krishnamurti L, Lucarelli G. Hematopoietic Stem Cell Transplantation for Hemoglobinopathies: Current Practice and Emerging Trends. Pediatric Clinics Of North America 2010, 57: 181-205. PMID: 20307718, DOI: 10.1016/j.pcl.2010.01.003.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationStem cell transplantationCell transplantationNew conditioning regimensTreatment-related toxicityOnly curative optionNumber of patientsManagement of thalassemiaSickle cell diseaseConditioning regimensCurative optionTreatment complicationsDonor poolCell diseasePatientsLife expectancyTransplantationHemoglobinopathiesCurrent practiceRegimensComplications
2009
Severe Sickle Cell Disease—Pathophysiology and Therapy
Buchanan G, Vichinsky E, Krishnamurti L, Shenoy S. Severe Sickle Cell Disease—Pathophysiology and Therapy. Transplantation And Cellular Therapy 2009, 16: s64-s67. PMID: 19819341, PMCID: PMC2832723, DOI: 10.1016/j.bbmt.2009.10.001.Peer-Reviewed Original ResearchConceptsSickle cell diseaseHematopoietic stem cell transplantationMarrow Transplant Research databaseSickle cell disease pathophysiologyStem cell transplantationCare of patientsModalities of therapyConservative managementInternational BloodCell transplantationTreatment modalitiesChronic illnessCell diseaseSCD manifestationsResearch DatabaseDisease pathophysiologyDiseaseTherapySignificant riskPatientsTransplantationBabiesPsychosocial costsModalitiesMorbidity
2008
Stable Long-Term Donor Engraftment following Reduced-Intensity Hematopoietic Cell Transplantation for Sickle Cell Disease
Krishnamurti L, Kharbanda S, Biernacki M, Zhang W, Baker K, Wagner J, Wu C. Stable Long-Term Donor Engraftment following Reduced-Intensity Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2008, 14: 1270-1278. PMID: 18940682, DOI: 10.1016/j.bbmt.2008.08.016.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAnemia, Sickle CellChildCyclosporineFemaleGraft SurvivalGraft vs Host DiseaseHematopoietic Stem Cell TransplantationHumansImmunosuppressive AgentsLiving DonorsMaleMycophenolic AcidMyeloablative AgonistsSiblingsTransplantation ChimeraTransplantation ConditioningTransplantation, HomologousConceptsHematopoietic stem cell transplantationSickle cell diseaseRIC regimenCell transplantationCell diseaseEfficacy of HSCTHigh-risk sickle cell diseaseReduced-Intensity Hematopoietic Cell TransplantationAllogeneic hematopoietic stem cell transplantationReduced-intensity conditioning regimensEquine antithymocyte globulinHost disease (GVHD) prophylaxisTotal lymphoid irradiationAcceptable therapeutic optionHematopoietic cell transplantationGroup of patientsStem cell transplantationAntithymocyte globulinLymphoid irradiationConditioning regimenMycophenolate mofetilConditioning regimensDisease prophylaxisDonor engraftmentTherapeutic optionsHematopoietic stem cell transplantation for hemoglobinopathies: Progress and prospects
Krishnamurti L, Gupta D. Hematopoietic stem cell transplantation for hemoglobinopathies: Progress and prospects. Indian Journal Of Medical And Paediatric Oncology 2008, 29: 39-48. DOI: 10.4103/0971-5851.51444.Peer-Reviewed Original ResearchSickle cell diseaseRisk factorsAllogeneic bone marrow transplantationClass IIHematopoietic stem cell transplantationClass ITransplant related morbidityUnrelated donor transplantsHLA-identical donorHLA-identical siblingsStem cell transplantationBone marrow transplantationCourse of diseaseLong-term survivalYears of ageAntithymocyte globulinAllogeneic SCTTransplant outcomesDonor transplantsRelated morbidityMarrow transplantationCell transplantationCell diseaseProbability of survivalTerm survival
2006
Commentary on “Identification of Unrelated Cord Blood Units for Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease”
Krishnamurti L. Commentary on “Identification of Unrelated Cord Blood Units for Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease”. Journal Of Pediatric Hematology/Oncology 2006, 28: 1-3. PMID: 16394884, DOI: 10.1097/01.mph.0000196448.99446.0a.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationUnrelated cord blood unitsStem cell transplantationSickle cell diseaseCord blood unitsCell transplantationCell diseaseBlood units
2005
Stable long term engraftment and amelioration of clinical phenotype following hematopoietic stem cell transplantation from a matched sibling donor for sickle cell disease using a reduced intensity conditioning regimen
Krishnamurti L, Wu C, Baker S, Yeager A, Wagner J. Stable long term engraftment and amelioration of clinical phenotype following hematopoietic stem cell transplantation from a matched sibling donor for sickle cell disease using a reduced intensity conditioning regimen. Transplantation And Cellular Therapy 2005, 11: 28. DOI: 10.1016/j.bbmt.2004.12.080.Peer-Reviewed Original Research