2023
Outcomes after RIC and Abatacept-Based Acute and Chronic Gvhd Prophylaxis in Allogeneic Transplantation for Sickle Cell Disease - Can Calcineurin Inhibitor Use be Curtailed?
Shah N, Ngwube A, Jacobsohn D, Shenoy S. Outcomes after RIC and Abatacept-Based Acute and Chronic Gvhd Prophylaxis in Allogeneic Transplantation for Sickle Cell Disease - Can Calcineurin Inhibitor Use be Curtailed? Blood 2023, 142: 2185. DOI: 10.1182/blood-2023-190524.Peer-Reviewed Original ResearchDisease-free survivalSickle cell diseaseSevere sickle cell diseaseUnrelated donor transplantsGVHD prophylaxisAllogeneic transplantationCell diseaseChronic GVHDDonor transplantsMarrow transplantationGrade 2Unrelated donor stem cell transplantationAllogeneic T cell proliferationDonor stem cell transplantationUnrelated donor marrow transplantationCalcineurin inhibitor useHost disease (GVHD) prophylaxisRelated donor transplantsSevere chronic GVHDSuccessful allogeneic transplantationGroup 1 patientsPhase II trialImmune suppression therapyStem cell transplantationHuman leucocyte antigenRole of abatacept in the prevention of graft-versus-host disease: current perspectives
Ngwube A, Rangarajan H, Shah N. Role of abatacept in the prevention of graft-versus-host disease: current perspectives. Therapeutic Advances In Hematology 2023, 14: 20406207231152644. PMID: 36845849, PMCID: PMC9943961, DOI: 10.1177/20406207231152644.Peer-Reviewed Original ResearchHematopoietic stem cell transplantAllogeneic hematopoietic stem cell transplantChronic GVHDGVHD prophylaxisDonor hematopoietic stem cell transplantSteroid-refractory chronic GVHDPrevention of graftSevere acute GVHDStem cell transplantAcute GVHDGVHD preventionHLA disparityHost diseaseGraft rejectionHSCT settingUnrelated donorsCalcineurin inhibitorsCell transplantNonmalignant disordersAlternative donorsMouse modelClinical practiceGVHDProphylaxisLimited reports
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
2021
Bone marrow transplant using fludarabine‐based reduced intensity conditioning regimen with in vivo T cell depletion in patients with Fanconi anemia
Gorfinkel L, Demsky C, Pashankar F, Kupfer G, Shah NC. Bone marrow transplant using fludarabine‐based reduced intensity conditioning regimen with in vivo T cell depletion in patients with Fanconi anemia. Pediatric Transplantation 2021, 25: e14009. PMID: 33755277, DOI: 10.1111/petr.14009.Peer-Reviewed Original ResearchConceptsUnrelated donor transplantsDonor transplantsBM graftsVivo T-cell depletionReduced intensity conditioning regimenStem cell transplant programInclusion of alemtuzumabNon-TBI conditioningPost-transplant complicationsSuccess of HSCTDevelopment of GVHDT-cell depletionIntensity conditioning regimenBone marrow transplantFull immune reconstitutionChronic GVHDCyclophosphamide conditioningGVHD prophylaxisMSD transplantsImmune reconstitutionConditioning regimenLate complicationsConditioning regimensSecondary neoplasmsSecondary malignancies
2020
Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease.
Ngwube A, Shah N, Godder K, Jacobsohn D, Hulbert ML, Shenoy S. Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease. Blood Advances 2020, 4: 3894-3899. PMID: 32813873, PMCID: PMC7448594, DOI: 10.1182/bloodadvances.2020002236.Peer-Reviewed Original ResearchConceptsSevere sickle cell diseaseReduced-intensity conditioningSickle cell diseaseUnrelated donorsChronic GVHDCell diseaseBone marrowUnrelated donor stem cell transplantationDonor stem cell transplantationReversible posterior encephalopathy syndromeCord blood recipientsHost disease (GVHD) prophylaxisDisease-free survivalOne-year incidenceBone marrow recipientsStem cell transplantationLonger durationPosterior encephalopathy syndromePhase 1 portionCell transplantation trialsClinical trial settingsT cell costimulationPhase 2 portionStem cell sourceAcute GVHD
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