2025
Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease
Walters M, Eapen M, Liu Y, El Rassi F, Waller E, Levine J, Strouse J, Antin J, Parikh S, Bakshi N, Dampier C, Jaroscak J, Bergmann S, Wong T, Kota V, Pace B, Lekakis L, Lulla P, Nickel R, Kasow K, Popat U, Smith W, Yu L, DiFronzo N, Geller N, Kamani N, Klings E, Hassell K, Mendizabal A, Sullivan K, Neuberg D, Krishnamurti L. Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease. Blood Advances 2025, 9: 955-965. PMID: 39471440, PMCID: PMC11907447, DOI: 10.1182/bloodadvances.2024013926.Peer-Reviewed Original ResearchHematopoietic cell transplantationVaso-occlusive painStandard of careSickle cell diseaseCell transplantationCell diseaseSCD-related eventsHLA-matched siblingTransplant-related mortalityPeriod of follow-upSurvival 2 yearsIntent-to-treat principleComparison of survivalDisease-modifying therapiesDisease-related mortalityUnrelated donorPrimary endpointCurative potentialTreatment armsDonor searchFollow-upCompare outcomesEligibility confirmationFunctional outcomesSecondary outcomes
2023
Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States
Goshua G, Ito S, Chetlapalli K, Potnis K, Calhoun C, Krishnamurti L, Krumholz H, Pandya A. Allotransplantation and Gene Therapy Equity for Children with Sickle Cell Disease: Distributional Cost-Effectiveness of Allotransplantation Vs Gene Therapy Vs Standard-of-Care in Pediatric Patients with Sickle Cell Disease in the United States. Blood 2023, 142: 490. DOI: 10.1182/blood-2023-191072.Peer-Reviewed Original ResearchSickle cell diseaseIncremental cost-effectiveness ratioDistributional cost-effectiveness analysisPediatric patientsCell diseaseCost-effectiveness analysisDisease severityHealth resource utilization dataPediatric Health Information SystemGene therapyJustifiable treatment optionTransplant-related mortalityVaso-occlusive crisisExpert clinical experienceMarrow Transplant ResearchSubstantial mortality riskVisual analog scaleQuality-adjusted life expectancyConcomitant riskCost-effectiveness ratioResource utilization dataCost-effectiveness frontierHost diseaseMaximum patientsOpioid therapy131I-Apamistamab Effectively Achieved Durable Responses in Patients with R/R AML Irrespective of the Presence of Multiple High-Risk Factors
Seropian S, Foran J, Gyurkocza B, Nath R, Choe H, Litzow M, Koshy N, Stiff P, Tomlinson B, Abhyankar S, Abedin S, Chen G, Al-Kadhimi Z, Kebriaei P, Sabloff M, Orozco J, Jamieson K, Magalhaes-Silverman M, Van Besien K, Schuster M, Law A, Mayer S, Lazarus H, Spross J, Li K, Haeuber E, Vusirikala M, Nahar A, Sandmaier B, Pagel J, Giralt S, Desai A, Abboud C. 131I-Apamistamab Effectively Achieved Durable Responses in Patients with R/R AML Irrespective of the Presence of Multiple High-Risk Factors. Blood 2023, 142: 2159. DOI: 10.1182/blood-2023-187433.Peer-Reviewed Original ResearchDurable complete remissionAdverse-risk cytogeneticsMultiple risk factorsRisk factorsConventional careCC groupComorbidity indexComplete remissionDurable responsesAllogeneic hematopoietic cell transplantHigh transplant-related mortalityMultiple high-risk factorsPrimary induction failureTransplant-related mortalityHematopoietic cell transplantInitiation of therapyMost older patientsPhase 3 studyHigher comorbidity indexTotal body irradiationHigh-risk factorsMechanism of actionAML ptsCRP assessmentEvaluable pts
2021
Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis—A Systematic Review and Meta-Analysis
Bewersdorf JP, Sheth AH, Vetsa S, Grimshaw A, Giri S, Podoltsev NA, Gowda L, Tamari R, Tallman MS, Rampal RK, Zeidan AM, Stahl M. Outcomes of Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis—A Systematic Review and Meta-Analysis. Transplantation And Cellular Therapy 2021, 27: 873.e1-873.e13. PMID: 34052505, PMCID: PMC8478722, DOI: 10.1016/j.jtct.2021.05.016.Peer-Reviewed Original ResearchConceptsConditioning regimen intensityAllo-HCTRegimen intensityChronic graftTransplant characteristicsGraft failureOverall survivalPatient ageSubgroup analysisDynamic International Prognostic Scoring System scoresSystematic reviewAllogeneic hematopoietic cell transplantationInternational Prognostic Scoring System scoreCurative therapeutic modalityNon-relapse mortalityTransplant-related mortalityMedian patient ageOutcomes of patientsSecondary graft failureCo-primary outcomesHematopoietic cell transplantHematopoietic cell transplantationRandomized clinical trialsHeterogeneity of patientsRandom-effects model
2020
AML-123: Targeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Leads to High Rates of Transplantation and Engraftment in Older Patients with Active, Relapsed, or Refractory (rel/ref) AML: Preliminary Midpoint Results from the Prospective, Randomized Phase 3 SIERRA Trial
Gyurkocza B, Nath R, Stiff P, Agura E, Litzow M, Tomlinson B, Choe H, Abhyankar S, Seropian S, Chen G, Hari P, Al-Kadhimi Z, Foran J, Orozco J, Van Besien K, Sabloff M, Kebriaei P, Abboud C, Levy M, Lazarus H, Giralt S, Berger M, Reddy V, Pagel J. AML-123: Targeted Conditioning with Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Leads to High Rates of Transplantation and Engraftment in Older Patients with Active, Relapsed, or Refractory (rel/ref) AML: Preliminary Midpoint Results from the Prospective, Randomized Phase 3 SIERRA Trial. Clinical Lymphoma Myeloma & Leukemia 2020, 20: s182. DOI: 10.1016/s2152-2650(20)30716-3.Peer-Reviewed Original ResearchCC armOlder patientsTransplant ratesLower transplant-related mortalityIncidence of gradeNon-relapse mortalityTransplant-related mortalityMajority of patientsFavorable safety profileFebrile neutropeniaInduction therapyPrior therapyActive diseaseRefractory AMLSalvage therapyDurable responsesGraft failureInfusion reactionsEngraftment dataMedian ageSafety profilePatient populationAdvanced ageDisease progressionGrade 3Incidence, Risk Factors, and Outcomes of Patients Who Develop Mucosal Barrier Injury–Laboratory Confirmed Bloodstream Infections in the First 100 Days After Allogeneic Hematopoietic Stem Cell Transplant
Dandoy CE, Kim S, Chen M, Ahn KW, Ardura MI, Brown V, Chhabra S, Diaz MA, Dvorak C, Farhadfar N, Flagg A, Ganguly S, Hale GA, Hashmi SK, Hematti P, Martino R, Nishihori T, Nusrat R, Olsson RF, Rotz SJ, Sung AD, Perales MA, Lindemans CA, Komanduri KV, Riches ML. Incidence, Risk Factors, and Outcomes of Patients Who Develop Mucosal Barrier Injury–Laboratory Confirmed Bloodstream Infections in the First 100 Days After Allogeneic Hematopoietic Stem Cell Transplant. JAMA Network Open 2020, 3: e1918668. PMID: 31913492, PMCID: PMC6991246, DOI: 10.1001/jamanetworkopen.2019.18668.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantAllogeneic hematopoietic stem cell transplantMucosal barrier injury laboratoryMBI-LCBIOutcomes of patientsStem cell transplantBloodstream infectionsCumulative incidenceCell transplantRisk factorsDay 100First allogeneic hematopoietic stem cell transplantMarrow Transplant Research databaseHost disease (GVHD) prophylaxisLansky performance statusTransplant-related mortalityOne-year mortalityTranslocation of bacteriaCord blood graftsCause of deathChronic graftConsecutive pediatricHost diseaseMyeloablative conditioningAdult patients
2019
Allogeneic CD34-Selected HSCT Following CAR T-Cells Is Associated with Low TRM and Favorable OS in Pediatric/Young Adult Patients with Relapsed/Refractory B-ALL
Fabrizio V, Boulad F, Cancio M, Higman M, Margossian S, Mauguen A, Prockop S, Scaradavou A, Shah N, Spitzer B, Yeager N, Kernan N, O'Reilly R, Boelens J, Curran K. Allogeneic CD34-Selected HSCT Following CAR T-Cells Is Associated with Low TRM and Favorable OS in Pediatric/Young Adult Patients with Relapsed/Refractory B-ALL. Blood 2019, 134: 4582. DOI: 10.1182/blood-2019-121767.Peer-Reviewed Original ResearchTransplant-related mortalityCAR T cellsB-cell acute lymphoblastic leukemiaCAR T-cell therapyAllo-HSCTYoung adult patientsComplete remissionT-cell therapyOverall survivalT cellsCumulative incidenceCalcineurin inhibitorsPlatelet engraftmentAdult patientsLate relapseMedian timeGraft sourceEntire cohortRefractory B-cell acute lymphoblastic leukemiaLess transplant-related mortalityLower transplant-related mortalityMRD-negative complete remissionChimeric antigen receptor T cellsCAR T-cell infusionAntigen receptor T cells
2018
Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias
Locatelli F, Ruggeri A, Merli P, Naik S, Agarwal R, Aquino V, Jacobsohn D, Qasim W, Nemecek E, Krishnamurti L, Manwani D, Kuhn M, Kapoor N. Administration of BPX-501 Cells Following Aβ T and B-Cell-Depleted HLA Haploidentical HSCT (haplo-HSCT) in Children with Acute Leukemias. Blood 2018, 132: 307. DOI: 10.1182/blood-2018-99-119481.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationRelapse-free survivalHLA-haploidentical hematopoietic stem cell transplantationEfficacy-evaluable populationHaplo-HSCTComplete remissionOverall survivalAcute leukemiaΑβ TB cellsEvaluable patientsPediatric patientsT cellsLower riskUnrelated donor hematopoietic stem cell transplantationDonor hematopoietic stem cell transplantationHaploidentical hematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationBetter overall clinical responseSteroid-resistant acute GVHDConventional steroid therapyOverall clinical responseSteroid-resistant GVHDTransplant-related mortalityPrimary graft failure
2016
Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approaches
Arnold S, Bhatia M, Horan J, Krishnamurti L. Haematopoietic stem cell transplantation for sickle cell disease – current practice and new approaches. British Journal Of Haematology 2016, 174: 515-525. PMID: 27255787, DOI: 10.1111/bjh.14167.Peer-Reviewed Original ResearchConceptsHaematopoietic stem cell transplantSickle cell diseaseCell diseaseAllogeneic haematopoietic stem cell transplantOnly available curative therapyHaematopoietic stem cell transplantationAlternative donor transplantsAvailable curative therapyCurrent maintenance therapiesTransplant-related mortalityDisease-free survivalIncidence of rejectionStem cell transplantStem cell transplantationHuman leucocyte antigenPool of donorsComplications persistHaploidentical donorsMaintenance therapyConditioning regimensCurative optionDonor transplantsSibling transplantsSupportive careCurative therapy
2015
A Detailed Evaluation of Transplant-Related Toxicities and Outcome for Patients with CNS Lymphoma (CNSL) Consolidated with High-Dose Therapy and Autologous Stem Cell Transplantation (HDT-ASCT) Using Thiotepa, Busulfan (Bu), Cyclophosphamide (TBC) Conditioning
Scordo M, Bhatt V, Hsu M, Omuro A, Matasar M, DeAngelis L, Dahi P, Moskowitz C, Giralt S, Sauter C. A Detailed Evaluation of Transplant-Related Toxicities and Outcome for Patients with CNS Lymphoma (CNSL) Consolidated with High-Dose Therapy and Autologous Stem Cell Transplantation (HDT-ASCT) Using Thiotepa, Busulfan (Bu), Cyclophosphamide (TBC) Conditioning. Blood 2015, 126: 4354. DOI: 10.1182/blood.v126.23.4354.4354.Peer-Reviewed Original ResearchNon-hematologic toxicitiesProgression-free survivalKaplan-Meier curvesSignificant grade 3HDT-ASCTMore grade 3Whole brain radiotherapyKarnofsky performance statusOverall survivalCNS lymphomaGrade 3Prior regimensHematopoietic cell transplant comorbidity indexAutologous stem cell transplantationHigh-dose therapyTransplant-related mortalityKaplan-Meier methodProspective clinical trialsRetrospective chart reviewStem cell transplantationGroups of ptsInitiation of conditioningYears of ageBU pharmacokineticsCTCAE 4.0Transplant 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
2014
Autologous stem cell transplant in recurrent or refractory primary or secondary central nervous system lymphoma using thiotepa, busulfan and cyclophosphamide
Welch MR, Sauter CS, Matasar MJ, Faivre G, Weaver SA, Moskowitz CH, Omuro AM. Autologous stem cell transplant in recurrent or refractory primary or secondary central nervous system lymphoma using thiotepa, busulfan and cyclophosphamide. Leukemia & Lymphoma 2014, 56: 361-367. PMID: 24745937, DOI: 10.3109/10428194.2014.916800.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAntineoplastic Combined Chemotherapy ProtocolsBusulfanCentral Nervous System NeoplasmsCombined Modality TherapyCyclophosphamideDisease-Free SurvivalDose-Response Relationship, DrugDrug Resistance, NeoplasmFemaleHumansKaplan-Meier EstimateMaleMiddle AgedNeoplasm Recurrence, LocalPrognosisRemission InductionStem Cell TransplantationThiotepaTransplantation, AutologousTreatment OutcomeConceptsAutologous stem cell transplantProgression-free survivalHigh-dose chemotherapyStem cell transplantOverall survivalCell transplantSecondary central nervous system lymphomaRefractory diffuse large B-cell lymphomaMedian progression-free survivalCentral nervous system involvementCentral nervous system lymphomaDiffuse large B-cell lymphomaLarge B-cell lymphomaTransplant-related mortalityNervous system involvementSecondary CNS lymphomaNervous system lymphomaStem cell harvestingB-cell lymphomaPotential treatment alternativeHDC-ASCTInduction chemotherapyRecurrent primaryCNS lymphomaComplete remission
2010
Long-Term Follow-up of Adults with Severe Sickle Cell Disease After Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning
Biernacki M, Okam M, Shenoy S, Krishnamurti L, Horwitz M, Neuberg D, Antin J, Wu C. Long-Term Follow-up of Adults with Severe Sickle Cell Disease After Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning. Blood 2010, 116: 261. DOI: 10.1182/blood.v116.21.261.261.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationSickle cell diseaseSevere sickle cell diseaseLimited chronic GVHDStem cell transplantationLong-term outcomesRIC-HSCTAdult patientsChronic GVHDGraft lossImmunosuppressive medicationsCell transplantationCell diseaseMyeloablative hematopoietic stem cell transplantationMinimal transplant-related toxicityPeripheral blood stem cellsStable donor chimerismSustained donor engraftmentTotal lymphoid irradiationTransplant-related mortalityIntensity conditioning regimensTransplant-related toxicityDisease-free survivalNew adult patientsMajority of patients
2004
Reduced-intensity conditioning regimen with thiotepa and fludarabine followed by allogeneic blood stem cell transplantation in haematological malignancies
Alessandrino EP, Bernasconi P, Colombo AA, Caldera D, Malcovati L, Troletti D, Vanelli L, Varettoni M, Montanari F, Lazzarino M. Reduced-intensity conditioning regimen with thiotepa and fludarabine followed by allogeneic blood stem cell transplantation in haematological malignancies. Bone Marrow Transplantation 2004, 34: 1039-1045. PMID: 15516936, DOI: 10.1038/sj.bmt.1704717.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAntineoplastic Combined Chemotherapy ProtocolsBlood TransfusionFemaleGraft SurvivalHematologic NeoplasmsHumansMaleMiddle AgedPeripheral Blood Stem Cell TransplantationRecurrenceRemission InductionSurvival AnalysisThiotepaTransplantation ChimeraTransplantation ConditioningTransplantation, HomologousVidarabineConceptsAllogeneic blood stem cell transplantationPeripheral stem cell transplantBlood stem cell transplantationReduced-intensity conditioning regimenStandard myeloablative regimenTransplant-related mortalityPoor performance statusStem cell transplantStem cell transplantationOverall survival probabilityMyeloablative regimenNonrelapse causesComplete remissionConditioning regimenMild nauseaPreparative regimenAdverse eventsPerformance statusMedian ageRBC transfusionCell transplantCell transplantationHaematological malignanciesSerum amylasePatients
2003
Reduced-intensity transplantation for patients with myelodysplastic syndrome achieves durable remission with less graft-versus-host disease
Chan GW, Foss FM, Klein AK, Sprague K, Miller KB. Reduced-intensity transplantation for patients with myelodysplastic syndrome achieves durable remission with less graft-versus-host disease. Transplantation And Cellular Therapy 2003, 9: 753-759. PMID: 14677114, DOI: 10.1016/j.bbmt.2003.08.002.Peer-Reviewed Original ResearchConceptsAcute GVHDHost diseasePreparative regimenExcess blastsAllogeneic transplantationDisease relapseGrade IIAllogeneic stem cell infusionReduced-intensity allogeneic transplantationReduced-intensity preparative regimenAllogeneic bone marrow transplantationHigh-risk MDS patientsExtensive chronic GVHDReduced-intensity transplantationTransplant-related mortalityFull donor chimerismTreatment-related mortalityStem cell infusionBone marrow transplantationMyelodysplastic syndrome patientsChronic myelomonocytic leukemiaSuccessful donor engraftmentChronic GVHDGVHD prophylaxisLess graft
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply