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
2019
Abatacept Is Effective for Gvhd Prophylaxis after Unrelated Donor Stem Cell Transplantation (URD SCT) for Severe Sickle Cell Disease (SCD)
Ngwube A, Shah N, Jacobsohn D, Dela Ziga E, Shenoy S. Abatacept Is Effective for Gvhd Prophylaxis after Unrelated Donor Stem Cell Transplantation (URD SCT) for Severe Sickle Cell Disease (SCD). Blood 2019, 134: 370. DOI: 10.1182/blood-2019-126405.Peer-Reviewed Original ResearchSevere sickle cell diseaseEvent-free survivalHost disease preventionAntigen-presenting cellsSickle cell diseaseURD-SCTLower incidenceAcute GVHDChronic GVHDGVHD prophylaxisAcute graftOverall survivalCell transplantationCell diseaseSevere SCDT cell co-stimulation blockadeUnrelated donor hematopoietic cell transplantationUnrelated donor stem cell transplantationDisease preventionAcute chest syndrome episodesDonor stem cell transplantationUnrelated donor marrow transplantationVaso-occlusive pain crisesAcute GVHD incidenceAcute GVHD prevention