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
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 patients