2024
Incidence and risk factors of pain crisis after hematopoietic cell transplantation for sickle cell disease
Krishnamurti L, Liang J, He Z, Deng Y, Nallagatla V, Hamidi R, Flagg A, Shah N. Incidence and risk factors of pain crisis after hematopoietic cell transplantation for sickle cell disease. Blood Advances 2024, 8: 1908-1919. PMID: 38324722, PMCID: PMC11021890, DOI: 10.1182/bloodadvances.2023010749.Peer-Reviewed Original ResearchHematopoietic cell transplantationSickle cell diseaseVaso-occlusive episodesPainful crisesCell transplantationPost-HCTGraft failureBefore HCTEvent-free survivalOverall survivalPain syndromePatient agePatient-centered outcomesAlternative donorsIncreased riskCell diseaseRisk factorsNatural historyPatientsLogistic regressionPainTransplantationSurvivalAgeAGVHDImpact of CMV Reactivation on Clinical Outcomes Post Allogeneic Stem Cell Transplant in Patients with Sickle Cell Disease
Rahim M, Ngwube A, Shenkar R, Rohaum H, Rao A, Stanek J, Shah N, Rangarajan H. Impact of CMV Reactivation on Clinical Outcomes Post Allogeneic Stem Cell Transplant in Patients with Sickle Cell Disease. Transplantation And Cellular Therapy 2024, 30: s302. DOI: 10.1016/j.jtct.2023.12.413.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationImpact of CMV reactivationReduced intensity conditioningSickle cell disease patientsAllogeneic hematopoietic stem cell transplantationSickle cell diseaseStem cell transplantationCMV reactivationMixed chimerismCell transplantationGraft failurePost-allogeneic hematopoietic stem cell transplantationRecovery of absolute lymphocyte countPost allogeneic stem cell transplantationAbsence of CMV-reactivationAllogeneic stem cell transplantationRabbit anti-thymocyte globulinMulti-institutional retrospective studyWhole bloodRisk of graft failureIncidence of GFPre-engraftment periodAnti-thymocyte globulinAbsolute lymphocyte countCompare clinical outcomes
2023
Abatacept-Prophylaxis Based Haploidentical Transplantation May Allow Sustained Engraftment and Offset Gvhd in Non-Malignant Disorders
Ngwube A, Shah N, Schulz G, Krishnamurti L, Shenoy S. Abatacept-Prophylaxis Based Haploidentical Transplantation May Allow Sustained Engraftment and Offset Gvhd in Non-Malignant Disorders. Blood 2023, 142: 4901. DOI: 10.1182/blood-2023-191145.Peer-Reviewed Original ResearchNon-malignant disordersPost-transplant cyclophosphamideSickle cell diseaseGVHD prophylaxisHematopoietic transplantationEvaluable patientsHaploidentical transplantationImmune suppressionBone marrowFirst year post-HCTGrade 1 acute GVHDChronic GVHD riskChronic skin GVHDGVHD-free survivalPost-transplant lymphoproliferationsRadiation-containing regimensYear post-HCTPhase 2 trialReduced intensity conditioningSerious infectious complicationsSystemic immune suppressionT-cell chimerismAllogeneic hematopoietic transplantationPrimary outcome measureStem cell boostOutcomes 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 antigenCongenital Dyserythropoietic Anemia Type II: An Update from the Congenital Dyseryhtropoietic Anemia Registry of North America (CDAR)
Elgammal Y, Risinger M, Husami A, Walden J, Gupta S, Shah N, Boyer J, Abajas Y, Winstead M, Miller D, Vidal-Anaya V, Vlachos A, Lal A, Ritchey A, Lorsbach R, Zhang W, Kalfa T, Niss O. Congenital Dyserythropoietic Anemia Type II: An Update from the Congenital Dyseryhtropoietic Anemia Registry of North America (CDAR). Blood 2023, 142: 1079. DOI: 10.1182/blood-2023-189389.Peer-Reviewed Original ResearchIron overloadIneffective erythropoiesisCongenital dyserythropoietic anemiaBlood transfusionHepatic hemosiderosisHemolytic anemiaIron levelsFrequency of transfusionGDF-15 levelsWhole-genome sequencingSeverity of anemiaAge of diagnosisCDA IINatural history studiesBody iron levelsDeep intronic variantsCDA II patientsCDA type IIIntronic variantsAutosomal recessive diseaseBone marrow erythroblastsBM examinationFerritin ratioGDF-15Anemia severityMediport use as an acceptable standard for CAR T cell infusion
Eylon M, Prabhu S, John S, King M, Bhatt D, Curran K, Erickson C, Karras N, Phillips C, Satwani P, Hermiston M, Southworth E, Baumeister S, Talano J, MacMillan M, Rossoff J, Bonifant C, Myers G, Rouce R, Toner K, Driscoll T, Katsanis E, Salzberg D, Schiff D, De Oliveira S, Capitini C, Pacenta H, Pfeiffer T, Shah N, Huynh V, Skiles J, Fraint E, McNerney K, Quigg T, Krueger J, Ligon J, Fabrizio V, Baggott C, Laetsch T, Schultz L. Mediport use as an acceptable standard for CAR T cell infusion. Frontiers In Immunology 2023, 14: 1239132. PMID: 37965315, PMCID: PMC10642031, DOI: 10.3389/fimmu.2023.1239132.Peer-Reviewed Original ResearchCAR T-cell infusionT-cell infusionCell infusionT-cell therapyCell infiltrationClinical practiceChimeric antigen receptor T-cell therapyCAR T-cell therapyCAR-T cell infiltrationCell therapyCell therapy administrationRetrospective cohort studyCentral venous cathetersT cell infiltrationCurrent clinical practiceStandard clinical practiceB-cell malignanciesT cell deliveryEvaluable patientsVenous optionsCohort studyVenous cathetersTherapy administrationPractice patternsCare recommendationsMixed donor chimerism following stem cell transplantation for sickle cell disease
Shah N, Rangarajan H, Ngwube A, Shenoy S. Mixed donor chimerism following stem cell transplantation for sickle cell disease. Current Opinion In Hematology 2023, 30: 187-193. PMID: 37694765, DOI: 10.1097/moh.0000000000000786.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationDonor/recipient chimerismStem cell transplantationSickle cell diseasePost-HCTRecipient chimerismDonor chimerismCell transplantationCell diseaseDisease controlDonor-derived erythropoiesisHCT conditioning regimensMixed donor chimerismSerial chimerism analysesRecent clinical trialsDonor erythropoiesisConditioning regimensHigh morbidityCurative interventionsClinical trialsSuccessful engraftmentSCD manifestationsMyeloid cellsHb S traitChimerism analysisCan defibrotide prophylaxis prevent sinusoidal obstruction syndrome following haematopoietic stem-cell transplantation?
Shah N, Shenoy S. Can defibrotide prophylaxis prevent sinusoidal obstruction syndrome following haematopoietic stem-cell transplantation? The Lancet Haematology 2023, 10: e309-e311. PMID: 37001535, DOI: 10.1016/s2352-3026(23)00065-0.Peer-Reviewed Original Research378 Main Reasons for Performing Hematopoietic Cell Transplantation in Patients with Sickle Cell Disease: Evolution over the Last Three Decades
Flagg A, He Z, Deng Y, Hamidi R, Shah N, Krishnamurti L. 378 Main Reasons for Performing Hematopoietic Cell Transplantation in Patients with Sickle Cell Disease: Evolution over the Last Three Decades. Transplantation And Cellular Therapy 2023, 29: s285-s286. DOI: 10.1016/s2666-6367(23)00447-5.Peer-Reviewed Original Research379 The Impact of Current Conditioning Regimen, T Cell Depletion and Graft Versus Host Disease Prophylaxis on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease
Shah N, Flagg A, Hamidi R, Hugo H, Deng Y, Krishnamurti L. 379 The Impact of Current Conditioning Regimen, T Cell Depletion and Graft Versus Host Disease Prophylaxis on the Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2023, 29: s286-s287. DOI: 10.1016/s2666-6367(23)00448-7.Peer-Reviewed Original Research384 Vaso-Occlusive Pain Requiring Hospitalization or Treatment Is Rare Following Successful Hematopoietic Cell Transplantation for Sickle Cell Disease
Krishnamurti L, He Z, Deng Y, Hamidi R, Flagg A, Shah N. 384 Vaso-Occlusive Pain Requiring Hospitalization or Treatment Is Rare Following Successful Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2023, 29: s291. DOI: 10.1016/s2666-6367(23)00453-0.Peer-Reviewed Original Research390 Endothelial Complications of Hematopoietic Cell Transplantation for Sickle Cell Disease
Flagg A, Hamidi R, He Z, Deng Y, Shah N, Krishnamurti L. 390 Endothelial Complications of Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2023, 29: s296. DOI: 10.1016/s2666-6367(23)00459-1.Peer-Reviewed Original Research392 Avascular Necrosis Following Hematopoietic Cell Transplantation for Sickle Cell Disease
Hamidi R, He Z, Deng Y, Flagg A, Shah N, Krishnamurti L. 392 Avascular Necrosis Following Hematopoietic Cell Transplantation for Sickle Cell Disease. Transplantation And Cellular Therapy 2023, 29: s297-s298. DOI: 10.1016/s2666-6367(23)00461-x.Peer-Reviewed Original Research391 Clinical Outcomes and Long-Term Follow-up of Children with Mixed Myeloid Chimerism Following Hematopoietic Cell Transplantation (HCT) for Sickle Cell Disease (SCD)
Guilcher G, Dalal S, Suresh T, Yelamali A, Aljayyousi H, Bhatia M, Haight A, Khandelwal P, Khoury R, Lafay Q, Mandava M, Nickel R, Ngwube A, Rangarajan H, Shah N, Stenger E, Yelamanchili N, Shenoy S. 391 Clinical Outcomes and Long-Term Follow-up of Children with Mixed Myeloid Chimerism Following Hematopoietic Cell Transplantation (HCT) for Sickle Cell Disease (SCD). Transplantation And Cellular Therapy 2023, 29: s296-s297. DOI: 10.1016/s2666-6367(23)00460-8.Peer-Reviewed Original ResearchRole 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
Endothelial Complications of Hematopoietic Cell Transplantation for Sickle Cell Disease
Flagg A, Hamidi R, Deng Y, Shah N, Krishnamurti L, He Z. Endothelial Complications of Hematopoietic Cell Transplantation for Sickle Cell Disease. Blood 2022, 140: 10460-10461. DOI: 10.1182/blood-2022-169107.Peer-Reviewed Original ResearchAntigen Directed Therapy Pre-Transplant Confers Superior Post-Transplant Survival Outcome Compared to Conventional Chemotherapy in Pediatric Patients with Relapsed and Refractory B-ALL
Stiefel J, Flower A, Botwinick M, Ford M, Foti A, Gandhi M, Slovin J, Smilow E, Bowers A, Karpel H, Schwartz L, Fraint E, Gennarini L, Gardner S, Raetz E, Shah N, Chen J, Krajewski J, Hijiya N, Satwani P, Hochberg J, Mauguen A, Curran K, Sulis M. Antigen Directed Therapy Pre-Transplant Confers Superior Post-Transplant Survival Outcome Compared to Conventional Chemotherapy in Pediatric Patients with Relapsed and Refractory B-ALL. Blood 2022, 140: 7763-7765. DOI: 10.1182/blood-2022-160272.Peer-Reviewed Original ResearchThe Use of Extracorporeal Membrane Oxygenation as a Bridge to Bone Marrow Transplantation in a Patient With High-risk Acute Myeloid Leukemia
Nader M, Bonde P, Massaro S, Giuliano J, Shah N. The Use of Extracorporeal Membrane Oxygenation as a Bridge to Bone Marrow Transplantation in a Patient With High-risk Acute Myeloid Leukemia. Journal Of Pediatric Hematology/Oncology 2022, 45: 18-20. PMID: 36219701, DOI: 10.1097/mph.0000000000002567.Peer-Reviewed Original ResearchConceptsHigh-risk acute myeloid leukemiaExtracorporeal membrane oxygenationAcute myeloid leukemiaMyeloid leukemiaMembrane oxygenationAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationVenoarterial extracorporeal membrane oxygenationTransplantation 2 monthsStem cell transplantationBone marrow transplantationMultiorgan dysfunction syndromeExtracorporeal life supportMarrow transplantationCell transplantationPatient selectionSeptic shockDysfunction syndromeRisk stratificationCardiogenic shockBiventricular failureTreatment protocolsCirculatory supportYounger ageMultidisciplinary approachHematopoietic 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 survival159 Relapse Risk for B-ALL Patients By Pre-Hematopoietic Cell Transplantation (HCT) Next-Generation Sequencing (NGS-MRD): An Interim Analysis of Observational Arm Subjects on Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) ONC1701
Quigg T, Skiles J, Keating A, Mahadeo K, Salzberg D, Madden L, Phelan R, Lalefar N, Caywood E, Hanna R, Bhatt S, Stefanski H, Horn B, Oshrine B, Higham C, Duffner U, Chewning J, Law J, Shah N, Huo J, Lehmann L, Ahmed I, Pulsipher M, Abdel-Azim H. 159 Relapse Risk for B-ALL Patients By Pre-Hematopoietic Cell Transplantation (HCT) Next-Generation Sequencing (NGS-MRD): An Interim Analysis of Observational Arm Subjects on Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) ONC1701. Transplantation And Cellular Therapy 2022, 28: s130-s131. DOI: 10.1016/s2666-6367(22)00320-7.Peer-Reviewed Original Research