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
Results of a Multicenter Pilot Investigation of Bone Marrow Transplantation in Adults with Sickle Cell Disease (STRIDE)
Krishnamurti L, Sullivan K, Kamani N, Waller E, Abraham A, Campigotto F, Zhang W, Smith S, Hassell K, Decastro L, Wu C, Neuberg D, Walters M. Results of a Multicenter Pilot Investigation of Bone Marrow Transplantation in Adults with Sickle Cell Disease (STRIDE). Blood 2015, 126: 543. DOI: 10.1182/blood.v126.23.543.543.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseAcute chest syndromeSevere sickle cell diseaseRegurgitant jet velocityAdverse eventsGraft failureUnrelated donorsCell diseaseEligibility criteriaUnrelated donor hematopoietic cell transplantationDonor hematopoietic cell transplantationEvent-free survival probabilityFull donor myeloid chimerismPosterior reversible encephalopathy syndromeDonor myeloid chimerismToxicity conditioning regimenTransplant conditioning regimenUnmodified bone marrowReversible encephalopathy syndromeSerious adverse eventsStandard supportive careTransplant-related toxicityDonor T cellsKaplan-Meier probability