2022
Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials
Krishnamurti L, Neuberg D, Sullivan K, Smith S, Eapen M, Walters M. Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials. Transplantation And Cellular Therapy 2022, 29: 217-221. PMID: 36270432, PMCID: PMC10539686, DOI: 10.1016/j.jtct.2022.10.008.Peer-Reviewed Original ResearchConceptsBone marrow transplantationSickle cell diseaseMarrow transplantationNonmalignant diseasesCell diseaseAdult sickle cell diseaseMyeloablative bone marrow transplantationSingle-arm feasibility studyYoung adultsCellular therapy trialsMulticenter National InstituteRegimen-related toxicitySingle-arm studyFrequency of HLACoronavirus disease 2019 (COVID-19) pandemicAcademic medical centerDisease 2019 pandemicShort-term endpointsBMT centersBMT regimenCare armPain episodesPrimary endpointStandard careCurative therapy
2019
Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study
Krishnamurti L, Neuberg D, Sullivan K, Kamani N, Abraham A, Campigotto F, Zhang W, Dahdoul T, De Castro L, Parikh S, Bakshi N, Haight A, Hassell K, Loving R, Rosenthal J, Smith S, Smith W, Spearman M, Stevenson K, Wu C, Wiedl C, Waller E, Walters M. Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study. American Journal Of Hematology 2019, 94: 446-454. PMID: 30637784, PMCID: PMC6542639, DOI: 10.1002/ajh.25401.Peer-Reviewed Original ResearchConceptsSevere sickle cell diseaseBone marrow transplantationEvent-free survivalSickle cell diseaseMarrow transplantationCell diseaseElevated tricuspid regurgitant jet velocityRegular red blood cell transfusionsSevere SCDDonor bone marrow transplantationOne-year overall survivalSecond bone marrow transplantationTricuspid regurgitant jet velocityRed blood cell transfusionProspective multicenter pilot studyDeveloped chronic GVHDHost disease (GVHD) prophylaxisStable donor chimerismAcute chest syndromeSecondary graft failureBlood cell transfusionHealth-related qualityPhysical function domainProspective clinical trialsRegurgitant jet velocity
2016
A trial of unrelated donor marrow transplantation for children with severe sickle cell disease
Shenoy S, Eapen M, Panepinto J, Logan B, Wu J, Abraham A, Brochstein J, Chaudhury S, Godder K, Haight A, Kasow K, Leung K, Andreansky M, Bhatia M, Dalal J, Haines H, Jaroscak J, Lazarus H, Levine J, Krishnamurti L, Margolis D, Megason G, Yu L, Pulsipher M, Gersten I, DiFronzo N, Horowitz M, Walters M, Kamani N. A trial of unrelated donor marrow transplantation for children with severe sickle cell disease. Blood 2016, 128: 2561-2567. PMID: 27625358, PMCID: PMC5123194, DOI: 10.1182/blood-2016-05-715870.Peer-Reviewed Original ResearchConceptsEvent-free survivalIncidence rateAcute chest syndrome episodesPosterior reversible encephalopathy syndromeSevere sickle cell diseaseUnrelated donor marrow transplantationVaso-occlusive pain crisesAllogeneic bone marrow transplantEffective GVHD prophylaxisGVHD-related deathsHost disease (GVHD) prophylaxisShort-course methotrexateReversible encephalopathy syndromeUnrelated donor transplantsPhase 2 trialBone marrow transplantTranscranial Doppler velocitiesSickle cell diseaseAcute GVHDChronic GVHDEFS ratesEncephalopathy syndromeGVHD prophylaxisTransplant indicationConditioning regimenHaematopoietic 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 ResearchMeSH KeywordsAnemia, Sickle CellDisease-Free SurvivalGenetic TherapyHematopoietic Stem Cell TransplantationHLA AntigensHumansUnrelated DonorsConceptsHaematopoietic 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