2023
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
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 management
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/
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
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 options
2000
Treatment of sickle cell disease (SCD) by an allogeneic hematopoietic stem cell transplantation (HSCT) using a non-myeloablative preparative regimen
Krishnamurti L, Blazar B, Grossi M, Abel S, Vichinsky E, Lubin B, Wagner J. Treatment of sickle cell disease (SCD) by an allogeneic hematopoietic stem cell transplantation (HSCT) using a non-myeloablative preparative regimen. Journal Of Pediatric Hematology/Oncology 2000, 22: 387. DOI: 10.1097/00043426-200007000-00093.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationSickle cell diseaseAllogeneic hematopoietic stem cell transplantationNon-myeloablative preparative regimenStem cell transplantationPreparative regimenCell transplantationCell disease