2022
Hematopoietic Cell Transplantation for Congenital Dyserythropoietic Anemia: A Report from the Pediatric Transplant and Cellular Therapy Consortium
Rangarajan HG, Stanek JR, Abdel-Azim H, Modi A, Haight A, McKinney CM, McKeone DJ, Buchbinder DK, Katsanis E, Abusin GA, Ahmed I, Law J, Silva JG, Mallhi KK, Burroughs LM, Shah N, Shaw PJ, Greiner R, Shenoy S, Pulsipher MA, Abu-Arja R. Hematopoietic Cell Transplantation for Congenital Dyserythropoietic Anemia: A Report from the Pediatric Transplant and Cellular Therapy Consortium. Transplantation And Cellular Therapy 2022, 28: 329.e1-329.e9. PMID: 35288346, DOI: 10.1016/j.jtct.2022.03.007.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantationEvent-free survivalCongenital dyserythropoietic anemiaCell transplantationAllogeneic hematopoietic cell transplantationSecond hematopoietic cell transplantationDyserythropoietic anemiaSole curative optionVeno-occlusive diseaseRetrospective multicenter studyMajority of patientsOutcome of childrenUmbilical cord bloodCDA type IICDA type IAcute graftAggressive chelationChronic GVHDHost diseaseCumulative incidenceCurative optionGraft failureMedian durationNonmyeloablative regimensOverall survival
2020
Low toxicity and favorable overall survival in relapsed/refractory B-ALL following CAR T cells and CD34-selected T-cell depleted allogeneic hematopoietic cell transplant
Fabrizio VA, Kernan NA, Boulad F, Cancio M, Allen J, Higman M, Margossian SP, Mauguen A, Prockop S, Scaradavou A, Shah N, Spitzer B, Stieglitz E, Yeager N, O’Reilly R, Brentjens RJ, Jan Boelens J, Curran KJ. Low toxicity and favorable overall survival in relapsed/refractory B-ALL following CAR T cells and CD34-selected T-cell depleted allogeneic hematopoietic cell transplant. Bone Marrow Transplantation 2020, 55: 2160-2169. PMID: 32390002, PMCID: PMC7606268, DOI: 10.1038/s41409-020-0926-1.Peer-Reviewed Original ResearchConceptsCAR T cellsAllo-HSCTT cellsCumulative incidenceCell transplantAllogeneic hematopoietic stem cell transplantAllogeneic hematopoietic cell transplantHematopoietic stem cell transplantCAR T-cell therapyConsolidative allo-HSCTHematopoietic cell transplantYoung adult patientsFavorable overall survivalStem cell transplantT-cell therapyFavorable OSOverall survivalAdult patientsSevere toxicitySmall cohortDisease controlPatientsCD34IncidenceRelapse
2019
Allogeneic CD34-Selected HSCT Following CAR T-Cells Is Associated with Low TRM and Favorable OS in Pediatric/Young Adult Patients with Relapsed/Refractory B-ALL
Fabrizio V, Boulad F, Cancio M, Higman M, Margossian S, Mauguen A, Prockop S, Scaradavou A, Shah N, Spitzer B, Yeager N, Kernan N, O'Reilly R, Boelens J, Curran K. Allogeneic CD34-Selected HSCT Following CAR T-Cells Is Associated with Low TRM and Favorable OS in Pediatric/Young Adult Patients with Relapsed/Refractory B-ALL. Blood 2019, 134: 4582. DOI: 10.1182/blood-2019-121767.Peer-Reviewed Original ResearchTransplant-related mortalityCAR T cellsB-cell acute lymphoblastic leukemiaCAR T-cell therapyAllo-HSCTYoung adult patientsComplete remissionT-cell therapyOverall survivalT cellsCumulative incidenceCalcineurin inhibitorsPlatelet engraftmentAdult patientsLate relapseMedian timeGraft sourceEntire cohortRefractory B-cell acute lymphoblastic leukemiaLess transplant-related mortalityLower transplant-related mortalityMRD-negative complete remissionChimeric antigen receptor T cellsCAR T-cell infusionAntigen receptor T cells