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
2019
Administration of BPX-501 Cells Following Αβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias (AL)
Galaverna F, Ruggeri A, Merli P, Kapoor N, Agarwal-Hashmi R, Aquino V, Jacobsohn D, Qasim W, Nemecek E, Krishnamurti L, Manwani D, Kuhn M, Locatelli F, Naik S. Administration of BPX-501 Cells Following Αβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias (AL). Transplantation And Cellular Therapy 2019, 25: s15-s16. DOI: 10.1016/j.bbmt.2018.12.082.Peer-Reviewed Original ResearchSteroid-resistant GVHDAcute leukemiaEvaluable ptsEfficacy outcomesΑβ TT cellsB cellsPediatric ptsBetter overall clinical responsePost-transplant GvHD prophylaxisHigh-risk acute leukemiaHLA-haploidentical HSCTOverall clinical responseSubset of ptsPrimary graft failureTumor-specific immunityHost disease symptomsInducible caspase-9 safety switchAllogeneic HSCTEnhanced graftGVHD prophylaxisHaplo-HSCTHaploidentical HSCTLeukemic effectsPlatelet engraftment
2018
Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias
Locatelli F, Ruggeri A, Merli P, Naik S, Agarwal R, Aquino V, Jacobsohn D, Qasim W, Nemecek E, Krishnamurti L, Manwani D, Kuhn M, Kapoor N. Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias. Blood 2018, 132: 307. DOI: 10.1182/blood-2018-99-119481.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationRelapse-free survivalHLA-haploidentical hematopoietic stem cell transplantationEfficacy-evaluable populationHaplo-HSCTComplete remissionOverall survivalAcute leukemiaΑβ TB cellsEvaluable patientsPediatric patientsT cellsLower riskUnrelated donor hematopoietic stem cell transplantationDonor hematopoietic stem cell transplantationHaploidentical hematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationBetter overall clinical responseSteroid-resistant acute GVHDConventional steroid therapyOverall clinical responseSteroid-resistant GVHDTransplant-related mortalityPrimary graft failure
2007
Unrelated cord blood transplantation in children with sickle cell disease: Review of four‐center experience
Adamkiewicz T, Szabolcs P, Haight A, Baker K, Staba S, Kedar A, Chiang K, Krishnamurti L, Boyer M, Kurtzberg J, Wagner J, Wingard J, Yeager A. Unrelated cord blood transplantation in children with sickle cell disease: Review of four‐center experience. Pediatric Transplantation 2007, 11: 641-644. PMID: 17663687, DOI: 10.1111/j.1399-3046.2007.00725.x.Peer-Reviewed Original ResearchConceptsUnrelated cord blood transplantationReduced-intensity regimensPrimary graft failureCord blood transplantationStable mixed chimerismSickle cell diseaseBlood transplantationGraft failureMyeloablative regimensMixed chimerismGrade IIICell diseaseViral infectionGVHDUCBTRegimensPatientsEngraftmentInfectionChildrenTransplantationStrokeChimerismEngraftDisease