2020
Should young children with sickle cell disease and an available human leukocyte antigen identical sibling donor be offered hematopoietic cell transplantation?
Krishnamurti L. Should young children with sickle cell disease and an available human leukocyte antigen identical sibling donor be offered hematopoietic cell transplantation? Hematology/Oncology And Stem Cell Therapy 2020, 13: 53-57. PMID: 32202246, DOI: 10.1016/j.hemonc.2019.12.008.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationSickle cell diseaseQuality of lifeOverall survivalCell transplantationCell diseaseYoung childrenHLA-identical siblingsHuman leukocyte antigenSevere clinical presentationAvailable human leukocyte antigenFree survivalOrgan dysfunctionClinical presentationLeukocyte antigenIdentical siblingsPremature mortalityAvailable HLAYounger ageTransplantationHLAAgeDiseaseChildrenSurvival
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