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 survivalPost-transplant CD34+ selected stem cell boost as an intervention for declining mixed chimerism following reduced intensity conditioning allogeneic stem cell transplant in children and young adults with sickle cell disease: A case series
Ngwube A, Franay C, Shah N. Post-transplant CD34+ selected stem cell boost as an intervention for declining mixed chimerism following reduced intensity conditioning allogeneic stem cell transplant in children and young adults with sickle cell disease: A case series. Pediatric Hematology And Oncology 2022, 39: 475-480. PMID: 35147476, DOI: 10.1080/08880018.2021.2013369.Peer-Reviewed Original Research
2021
Evidence-Based Minireview: In young children with severe sickle cell disease, do the benefits of HLA-identical sibling donor HCT outweigh the risks?
Shah N, Krishnamurti L. Evidence-Based Minireview: In young children with severe sickle cell disease, do the benefits of HLA-identical sibling donor HCT outweigh the risks? Hematology 2021, 2021: 190-195. PMID: 34889371, PMCID: PMC8791135, DOI: 10.1182/hematology.2021000322.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantVaso-occlusive pain episodesSickle cell diseaseCell diseaseDonor hematopoietic stem cell transplantAllogeneic hematopoietic stem cell transplantHuman leukocyte antigen identicalSevere sickle cell diseaseBenefits of HLAAcute chest syndromeHLA-identical siblingsStem cell transplantYears of ageAge-appropriate activitiesChest syndromePain crisisPain episodesFebrile episodesCell transplantMedical conditionsMale childrenFemale childrenPatientsCase 2Case 1Bone 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
Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR)
Niss O, Lorsbach R, Berger M, Chonat S, McLemore M, Buchbinder D, McCavit T, Shaffer L, Simpson J, Schwartz J, Meznarich J, Emberesh M, Seu K, Zhang W, Kalfa T, Husami A, Kalfa T, Lorsbach R, Lutzko C, Nelson A, Niss O, Quinn C, Seu K, Zhang W, Chonat S, Buchbinder D, Johnson C, McCavit T, Shaffer L, Rothman J, Gupta S, Toscano M, Forouhar M, Gidvani-Diaz V, Ball J, Roach G, Wagner K, Milanovich S, Boyer J, Chawla J, Smith C, Lee A, Simpson J, Schwartz J, Radulescu V, Abajas Y, Ritchey A, Meznarich J, Underhill H, Ravindranath Y, McLemore M, Shah N. Congenital dyserythropoietic anemia type I: First report from the Congenital Dyserythropoietic Anemia Registry of North America (CDAR). Blood Cells Molecules And Diseases 2020, 87: 102534. PMID: 33401150, PMCID: PMC8809105, DOI: 10.1016/j.bcmd.2020.102534.Peer-Reviewed Original ResearchConceptsCongenital dyserythropoietic anemiaNatural historyTransfusion-dependent patientsSevere hemolytic anemiaPulmonary hypertensionMedian ageHemolytic anemiaIron overloadMedical attentionIneffective erythropoiesisHeterogeneous diseasePatientsAccurate diagnosisAnemiaSignificant phenotypic heterogeneityGenetic testingPerinatal anemiaDyserythropoietic anemiaBiologic studiesDiagnosisAge 5TransfusionRegistryPhenotypic heterogeneityResearch NetworkAbatacept 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 GVHDLow 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
GRFS and CRFS in alternative donor hematopoietic cell transplantation for pediatric patients with acute leukemia
Mehta RS, Holtan SG, Wang T, Hemmer MT, Spellman SR, Arora M, Couriel DR, Alousi AM, Pidala J, Abdel-Azim H, Ahmed I, Aljurf M, Askar M, Auletta JJ, Bhatt V, Bredeson C, Chhabra S, Gadalla S, Gajewski J, Gale RP, Gergis U, Hematti P, Hildebrandt GC, Inamoto Y, Kitko C, Khandelwal P, MacMillan ML, Majhail N, Marks DI, Mehta P, Nishihori T, Olsson RF, Pawarode A, Diaz MA, Prestidge T, Qayed M, Rangarajan H, Ringden O, Saad A, Savani BN, Seo S, Shah A, Shah N, Schultz KR, Solh M, Spitzer T, Szer J, Teshima T, Verdonck LF, Williams KM, Wirk B, Wagner J, Yared JA, Weisdorf DJ. GRFS and CRFS in alternative donor hematopoietic cell transplantation for pediatric patients with acute leukemia. Blood Advances 2019, 3: 1441-1449. PMID: 31053571, PMCID: PMC6517657, DOI: 10.1182/bloodadvances.2018030171.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAlemtuzumabBone Marrow CellsChildChild, PreschoolDisease-Free SurvivalFemaleFetal BloodGraft vs Host DiseaseHematopoietic Stem Cell TransplantationHumansLeukemia, Myeloid, AcuteMalePrecursor Cell Lymphoblastic Leukemia-LymphomaProportional Hazards ModelsRecurrenceSurvival RateThyroglobulinTransplantation ConditioningWhole-Body IrradiationConceptsRelapse-free survivalPediatric patientsGrade IIIBM groupAcute leukemiaHigh riskAlternative donor hematopoietic cell transplantationTotal body irradiation-based conditioningMultivariate analysisDonor hematopoietic cell transplantationFree relapse-free survivalCox proportional hazards modelAnti-thymocyte globulinHematopoietic cell transplantationIrradiation-based conditioningAcute lymphoblastic leukemiaProportional hazards modelUmbilical cord bloodBM recipientsGraft variablesGVHD prophylaxisUCB groupHost diseaseMycophenolate mofetilClinical characteristics
2018
CAR‐T cell manufacture: snatching victory when defeat is looming
Gowda L, Shah NC. CAR‐T cell manufacture: snatching victory when defeat is looming. Transfusion 2018, 58: 1335-1337. PMID: 29949188, DOI: 10.1111/trf.14760.Peer-Reviewed Original Research
2015
Transplant Outcomes for Children with T Cell Acute Lymphoblastic Leukemia in Second Remission: A Report from the Center for International Blood and Marrow Transplant Research
Burke M, Verneris M, Le Rademacher J, He W, Abdel-Azim H, Abraham A, Auletta J, Ayas M, Brown V, Cairo M, Chan K, Diaz Perez M, Dvorak C, Egeler R, Eldjerou L, Frangoul H, Guilcher G, Hayashi R, Ibrahim A, Kasow K, Leung W, Olsson R, Pulsipher M, Shah N, Shah N, Thiel E, Talano J, Kitko C. Transplant Outcomes for Children with T Cell Acute Lymphoblastic Leukemia in Second Remission: A Report from the Center for International Blood and Marrow Transplant Research. Transplantation And Cellular Therapy 2015, 21: 2154-2159. PMID: 26327632, PMCID: PMC4654112, DOI: 10.1016/j.bbmt.2015.08.023.Peer-Reviewed Original ResearchMeSH KeywordsAcademic Medical CentersAcute DiseaseAdolescentBone Marrow TransplantationChildChild, PreschoolChronic DiseaseFemaleGraft vs Host DiseaseHumansImmunosuppressive AgentsInternational CooperationMaleMyeloablative AgonistsPrecursor T-Cell Lymphoblastic Leukemia-LymphomaProspective StudiesRecurrenceRemission InductionSeverity of Illness IndexSurvival AnalysisTransplantation ConditioningTransplantation, HomologousTreatment OutcomeConceptsHematopoietic cell transplantationT-cell acute lymphoblastic leukemiaCell acute lymphoblastic leukemiaMarrow Transplant ResearchAcute lymphoblastic leukemiaInternational BloodLymphoblastic leukemiaTransplant ResearchRelapsed T-cell acute lymphoblastic leukemiaThree-year overall survivalAllogeneic hematopoietic cell transplantationDisease-free survival ratesBone marrow/peripheral bloodTransplant-related mortalitySecond complete remissionBone marrow relapseUmbilical cord bloodPediatric T-ALLChronic graftExtramedullary relapseSecond remissionComplete remissionExtramedullary diseaseHost diseaseMarrow relapse