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 1
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 Network
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
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