2023
Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507
Kassim A, Walters M, Eapen M, Ritzau N, Smith M, Solh M, McKinney C, Nieder M, Ross M, Kent M, Abusin G, Mallhi K, Silva J, Shaughnessy P, Kanter J, Haines H, Farah R, Khaled Y, Abraham A, Bollard C, Cooke K, de La Fuente J, Hanna R, Horowitz M, Jordan L, Krishnamurti L, Leifere E, Mahadeo K, Shenoy S, Ritzau N, DeBaun M, Brodsky R. Reduced Intensity Haploidentical Bone Marrow Transplantation in Adults with Severe Sickle Cell Disease: BMT CTN 1507. Blood 2023, 142: lba-4. DOI: 10.1182/blood-2023-192022.Peer-Reviewed Original ResearchPost-transplant cyclophosphamideEvent-free survivalBone marrow transplantSickle cell diseaseSevere sickle cell diseaseTotal body irradiationGraft failureAcute GVHDPost-BMTCell diseaseDay 100Multi-center phase II trialFirst year post-BMTHaploidentical bone marrow transplantVaso-occlusive pain episodesHaploidentical bone marrow transplantationHematopoietic stem cell transplantationChronic transfusion regimenDurable donor engraftmentHemoglobin SS diseaseAcute chest syndromePrimary graft failureSecondary graft failureEnd-organ toxicityPhase II trial
2005
Mixed Hematopoietic Chimerism for Sickle Cell Disease Prevents Intravascular Hemolysis and Corrects Biomarkers of Endothelial Function.
Wu C, Krishnamurti L, Gladwin M, Biernacki M, Rogers S, Wang X, Zahrieh D, Antin J, Ritz J. Mixed Hematopoietic Chimerism for Sickle Cell Disease Prevents Intravascular Hemolysis and Corrects Biomarkers of Endothelial Function. Blood 2005, 106: 2333. DOI: 10.1182/blood.v106.11.2333.2333.Peer-Reviewed Original ResearchSickle cell diseaseMixed hematopoietic chimerismStem cell transplantationEndothelial functionIntravascular hemolysisMixed chimerismPulmonary hypertensionDonor engraftmentCell transplantationVascular functionHematopoietic chimerismNonmyeloablative allogeneic stem cell transplantationChronic end-organ damageAllogeneic stem cell transplantationEnd-organ injuryExperienced graft rejectionEnd-organ damageEnd-organ toxicityPre-transplant levelsDonor cell engraftmentChronic intravascular hemolysisSuccess of therapyFree Hb concentrationEndothelial dysfunctionGraft rejection