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 regimen
2008
Hematopoietic 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