2019
A Phase II Trial to Compare Allogeneic Transplant Vs. Standard of Care for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 1503
Eapen M, Neuberg D, Mendizabal A, Stevenson K, Antin J, DiFronzo N, Rassi F, Lulla P, Waller E, Garrison J, Smith S, Sullivan K, Walters M, Krishnamurti L. A Phase II Trial to Compare Allogeneic Transplant Vs. Standard of Care for Severe Sickle Cell Disease: Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Protocol 1503. Blood 2019, 134: 4592. DOI: 10.1182/blood-2019-126793.Peer-Reviewed Original ResearchSickle cell diseaseHematopoietic cell transplantationSevere sickle cell diseaseUnrelated donorsUnrelated donor hematopoietic cell transplantationDonor hematopoietic cell transplantationCurrent phase II trialMyeloablative conditioning regimenPhase II trialConditioning regimenII trialCurative treatmentCell transplantationTreatment optionsCurrent therapiesCell diseasePilot trialRare diseaseSuitable donorStudy designHLAAge 15Young adultsTrialsGold standard
2015
Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease
Walters M, De Castro L, Sullivan K, Krishnamurti L, Kamani N, Bredeson C, Neuberg D, Hassell K, Farnia S, Campbell A, Petersdorf E. Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2015, 22: 207-211. PMID: 26500093, PMCID: PMC5031360, DOI: 10.1016/j.bbmt.2015.10.017.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseCell transplantationCell diseaseSurvival rateEvent-free survival rateTransplant-related complicationsOverall survival rateMarrow Transplant RegistryMarrow Transplant ResearchRisk of mortalityLong-term qualityRate of survivalNontransplant cohortHost diseaseTransplant RegistryOverall survivalProspective trialInternational BloodTransplant ResearchEuropean BloodMortality riskSuitable donorTherapeutic valueStrong recommendations