2023
Phase II Trial of Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation Conditioning Results in Favorable Outcomes with Haploidentical Donor Peripheral Blood Stem Cell Transplant
Elmariah H, Kim J, Mishra A, Faramand R, Perez L, Lazaryan A, Khimani F, Liu H, Ochoa-Bayona J, Nieder M, Figura N, Robinson T, Nishihori T, Anasetti C, Pidala J, Bejanyan N. Phase II Trial of Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation Conditioning Results in Favorable Outcomes with Haploidentical Donor Peripheral Blood Stem Cell Transplant. Blood 2023, 142: 4900. DOI: 10.1182/blood-2023-187473.Peer-Reviewed Original ResearchDisease-free survivalPost-transplant cyclophosphamideHematopoietic cell transplantNon-relapse mortalityPhase II trialTotal body irradiationAllogeneic hematopoietic cell transplantReduced-intensity conditioningBone marrow graftsPrimary endpointII trialCumulative incidenceGVHD prophylaxisCell transplantHematologic malignanciesCyclophosphamide/total body irradiationGVHD-free relapse-free survivalHematopoietic cell transplant comorbidity indexPeripheral blood stem cell graftsSingle-center phase II trialPeripheral blood stem cell transplantBlood stem cell graftsHigh-risk hematologic malignanciesMonths disease free survivalDay 6Impact of Type of Hypomethylating Agent (HMA) Used on Outcomes of Patients (Pts) with Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) - a Large, Multicenter, Retrospective Analysis
Bewersdorf J, Kewan T, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Uy G, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, McMahon C, Zeidner J, Savona M, Stempel J, Chandhok N, Ramaswamy R, Roboz G, Rolles B, Wang E, Harris A, Amaya M, Hawkins H, Grenet J, Gurnari C, Shallis R, Xie Z, Maciejewski J, Sallman D, Della Porta M, Komrokji R, Zeidan A. Impact of Type of Hypomethylating Agent (HMA) Used on Outcomes of Patients (Pts) with Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) - a Large, Multicenter, Retrospective Analysis. Blood 2023, 142: 4613. DOI: 10.1182/blood-2023-178728.Peer-Reviewed Original ResearchCox multivariable regression modelOverall survivalHMA initiationHypomethylating agentMultivariable regression modelsTP53 mutationsAllo-HCTComplete remissionComplex karyotypePartner drugsBone marrowSurvival analysisAllogeneic hematopoietic cell transplantMultivariable Cox regression modelsTreatment typeOverall responseAdverse genetic featuresMedian overall survivalOutcomes of patientsHematopoietic cell transplantAdverse overall survivalKaplan-Meier methodCox regression modelLog-rank testPredictors of response131I-Apamistamab-Led Allogeneic Hematopoietic Cell Transplant Significantly Improves Overall Survival in Patients with TP53 Mutated R/R AML
Choe H, Tomlinson B, Gyurkocza B, Nath R, Seropian S, Litzow M, Abboud C, Stiff P, Abhyankar S, Foran J, 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, Koshy N. 131I-Apamistamab-Led Allogeneic Hematopoietic Cell Transplant Significantly Improves Overall Survival in Patients with TP53 Mutated R/R AML. Blood 2023, 142: 469. DOI: 10.1182/blood-2023-182177.Peer-Reviewed Original ResearchMedian overall survivalAllogeneic hematopoietic cell transplantDurable complete remissionHematopoietic cell transplantOverall survivalR AMLConventional careTP53 mutationsPositive ptsCC groupComplete remissionRelapse rateCell transplantHigh post-transplant relapse ratesInitiation of therapyPhase 3 studyTotal body irradiationHigh relapse rateCRP assessmentEvaluable ptsOlder ptsActive diseaseBaseline characteristicsRefractory AMLBody irradiationHigh-Dose Targeted Radiation with 131I-Apamistamab Prior to HCT Demonstrated a Dose-Response for Durable Complete Remission in Patients with R/R AML
Litzow M, Chen G, Gyurkocza B, Nath R, Seropian S, Choe H, Abboud C, Koshy N, Tomlinson B, Abhyankar S, Foran J, Abedin S, 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, Leung E, Chen M, Natwa M, Spross J, Li K, Nagl N, Haeuber E, Vusirikala M, Nahar A, Sandmaier B, Pagel J, Giralt S, Desai A, Wahl R, Pandit-Taskar N, Brodin P, Stiff P. High-Dose Targeted Radiation with 131I-Apamistamab Prior to HCT Demonstrated a Dose-Response for Durable Complete Remission in Patients with R/R AML. Blood 2023, 142: 3529. DOI: 10.1182/blood-2023-189040.Peer-Reviewed Original ResearchDurable complete remissionConventional careLiver dosePrimary endpointBone marrowR AMLComplete remissionLiver ratioHigh doseLeukemic cellsRadiation doseAllogeneic hematopoietic cell transplantRadiation dose-response relationshipHematopoietic cell transplantMost older patientsPhase 3 studyTotal body irradiationFavorable biodistributionRadiation dose toleranceYears of ageDose toleranceDose-response relationshipDiseased bone marrowMedian doseRefractory AML131I-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 ptsBleeding in Critically Ill Children With Malignancy or Hematopoietic Cell Transplant: A Single-Center Prospective Cohort Study
Levasseur J, Fikse L, Mauguen A, Killinger J, Karam O, Nellis M. Bleeding in Critically Ill Children With Malignancy or Hematopoietic Cell Transplant: A Single-Center Prospective Cohort Study. Pediatric Critical Care Medicine 2023, 24: e602-e610. PMID: 37678406, PMCID: PMC10843653, DOI: 10.1097/pcc.0000000000003374.Peer-Reviewed Original ResearchConceptsProspective cohort studyBleeding episodesSevere bleedingIll childrenCohort studyCell transplantIncidence rateLaboratory valuesMinimal bleedingRadiation therapySingle-center prospective cohort studyAssociated Patient CharacteristicsHigher PICU mortalityNew bleeding episodesOnly laboratory valueHematopoietic cell transplantCritically Ill ChildrenLow platelet countSevere bleeding episodesStem cell transplantPatient demographic dataSpecialized cancer hospitalModerate bleeding episodesYears of ageLonger PICU488 Feasibility of Using the American Time Use Survey to Study the Impact of Sickle Cell Disease and Hematopoietic Cell Transplant on the Daily Lives of Patients and Their Caregivers: A Stellar Study
Khemani K, Stenger E, Arnold S, Bakshi N, Ross D, Smith C, Basu A, Krishnamurti L. 488 Feasibility of Using the American Time Use Survey to Study the Impact of Sickle Cell Disease and Hematopoietic Cell Transplant on the Daily Lives of Patients and Their Caregivers: A Stellar Study. Transplantation And Cellular Therapy 2023, 29: s366. DOI: 10.1016/s2666-6367(23)00556-0.Peer-Reviewed Original ResearchHematopoietic cell transplantSickle cell diseaseCell transplantCell diseasePatientsTransplantDiseaseCaregiversSecondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease
Eapen M, Brazauskas R, Williams D, Walters M, St Martin A, Jacobs B, Antin J, Bona K, Chaudhury S, Coleman-Cowger V, DiFronzo N, Esrick E, Field J, Fitzhugh C, Kanter J, Kapoor N, Kohn D, Krishnamurti L, London W, Pulsipher M, Talib S, Thompson A, Waller E, Wun T, Horowitz M. Secondary Neoplasms After Hematopoietic Cell Transplant for Sickle Cell Disease. Journal Of Clinical Oncology 2023, 41: 2227-2237. PMID: 36623245, PMCID: PMC10448940, DOI: 10.1200/jco.22.01203.Peer-Reviewed Original ResearchConceptsLow-intensity regimensSickle cell diseaseSecondary neoplasmsRisk factorsCell diseaseLeukemia/myelodysplastic syndromeMixed donor chimerismReduced-intensity regimensFull donor chimerismHematopoietic cell transplantTotal body irradiationLow-dose radiationGray regression modelsMyeloid mutationsPrior inflammationCell transplantMyelodysplastic syndromeTolerance inductionIntense regimensPlausible etiologyHigh riskRegimensSolid tumorsNeoplasmsMyeloid malignancies
2022
Gonadal Hormone Production after Hematopoietic Cell Transplant (HCT) in Patients with Sickle Cell Disease (SCD): A Stellar Study
George S, Veludhandi A, Xiang Y, Stenger E, Arnold S, Mehta A, Schirmer D, Spencer J, Guilcher G, Bhatia M, Abraham A, Gomez-Lobo V, Krishnamurti L, Meacham L. Gonadal Hormone Production after Hematopoietic Cell Transplant (HCT) in Patients with Sickle Cell Disease (SCD): A Stellar Study. Blood 2022, 140: 1391-1392. DOI: 10.1182/blood-2022-168060.Peer-Reviewed Original ResearchHematopoietic cell transplantSickle cell diseaseGonadal hormone productionCell transplantCell diseaseHormone productionPatientsTransplantDiseaseMultimodal phenotyping and correlates of pain following hematopoietic cell transplant in children with sickle cell disease
Bakshi N, Astles R, Chou E, Hurreh A, Sil S, Sinha C, Sanders K, Peddineni M, Gillespie S, Keesari R, Krishnamurti L. Multimodal phenotyping and correlates of pain following hematopoietic cell transplant in children with sickle cell disease. Pediatric Blood & Cancer 2022, 70: e30046-e30046. PMID: 36322607, PMCID: PMC9820671, DOI: 10.1002/pbc.30046.Peer-Reviewed Original ResearchConceptsHematopoietic cell transplantPatient-reported outcomesSickle cell diseaseYear post-HCTPost-HCTExperimental pain sensitivitySickle cell anemiaCell transplantPain thresholdPain sensitivityCell diseaseCorrelates of painPressure pain thresholdHealth-related qualityCold pain thresholdAssessment of painPsychological factorsUnderstanding of painEffect sizePain assessmentObservational studySevere genotypePainCell anemiaOptional substudies58 Prevalence of and Risk Factors for Cardiac, Pulmonary, and Neurologic Dysfunction Following Hematopoietic Cell Transplant for Sickle Cell Disease: A STAR Study
Stenger E, Chellapandian D, Shah R, Gillepsie S, Xiang Y, Bhatia M, Chaudhury S, Eckrich M, Guilcher G, Jaroscak J, Kasow K, Krajewski J, Ngwube A, Olson T, Rangarajan H, Horan J, Krishnamurti L, Shenoy S, Abraham A. 58 Prevalence of and Risk Factors for Cardiac, Pulmonary, and Neurologic Dysfunction Following Hematopoietic Cell Transplant for Sickle Cell Disease: A STAR Study. Transplantation And Cellular Therapy 2022, 28: s49-s51. DOI: 10.1016/s2666-6367(22)00219-6.Peer-Reviewed Original ResearchAzacitidine maintenance in AML post induction and posttransplant
Bewersdorf JP, Prebet T, Gowda L. Azacitidine maintenance in AML post induction and posttransplant. Current Opinion In Hematology 2022, 29: 84-91. PMID: 35013047, DOI: 10.1097/moh.0000000000000700.Peer-Reviewed Original ResearchConceptsDisease-free survivalPostremission therapyAcute myeloid leukemiaAllo-HCTCC-486Allogeneic hematopoietic cell transplantNonrelapse mortality riskOverall survival benefitHematopoietic cell transplantDuration of remissionSubset of patientsRobust study designsAML trialsAzacitidine maintenanceInduction therapySurvival benefitAML patientsCell transplantDisease relapseRelapse reductionRelapse riskMyeloid leukemiaCommon causeSelect populationMortality risk
2021
Clinical Experience in the Randomized Phase 3 Sierra Trial: Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Conditioning Enables Hematopoietic Cell Transplantation with Successful Engraftment and Acceptable Safety in Patients with Active, Relapsed/Refractory AML Not Responding to Targeted Therapies
Gyurkocza B, Nath R, Seropian S, Choe H, Litzow M, Koshy N, Stiff P, Abboud C, Tomlinson B, Abhyankar S, Hari P, Al-Kadhimi Z, Chen G, Sabloff M, Orozco J, Foran J, Kebriaei P, Jamieson K, Magalhaes-Silverman M, van Besien K, Schuster M, Law A, Levy M, Lazarus H, Giralt S, Berger M, Spross J, Desai A, Reddy V, Pagel J. Clinical Experience in the Randomized Phase 3 Sierra Trial: Anti-CD45 Iodine (131I) Apamistamab [Iomab-B] Conditioning Enables Hematopoietic Cell Transplantation with Successful Engraftment and Acceptable Safety in Patients with Active, Relapsed/Refractory AML Not Responding to Targeted Therapies. Blood 2021, 138: 1791. DOI: 10.1182/blood-2021-148497.Peer-Reviewed Original ResearchHematopoietic cell transplantAcute myeloid leukemiaR AMLAllogeneic hematopoietic cell transplantComplete remissionMedian radiation doseBristol-Myers SquibbCC patientsTargeted therapyFebrile neutropeniaFLT-3 inhibitorsCurrent equity holderSpeakers bureauConventional careMedian timeBcl-2 inhibitorsIDH inhibitorsRelapsed/Refractory Acute Myeloid LeukemiaIncidence of FNRefractory acute myeloid leukemiaAdvisory CommitteePrincipal investigatorSeattle GeneticsIncidence of gradeIncidence of sepsisAssessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies
Katoch D, Krishnamurti L. Assessing Patient Preferences for Treatment Options for Pediatric Sickle Cell Disease: A Critical Review of Quantitative and Qualitative Studies. Patient Preference And Adherence 2021, 15: 2221-2229. PMID: 34629865, PMCID: PMC8493010, DOI: 10.2147/ppa.s264918.Peer-Reviewed Original ResearchSickle cell diseasePatient preferencesTreatment preferencesCell diseasePediatric sickle cell diseaseHematopoietic cell transplantPatients' treatment preferencesBurden of careQuality of lifeQualitative studyStandard gamble studyCurative intentRed blood cellsSupportive careSubstantial morbidityCell transplantImpaired qualityTreatment optionsIntravascular hemolysisPatient utilitiesPremature mortalityCaregiver perspectivesTreatment alternativesBlood cellsUtility studiesHypomethylating Agents and FLT3 Inhibitors As Maintenance Treatment for Acute Myeloid Leukemia and Myelodysplastic Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation–A Systematic Review and Meta-Analysis
Bewersdorf JP, Allen C, Mirza AS, Grimshaw AA, Giri S, Podoltsev NA, Gowda L, Cho C, Tallman MS, Zeidan AM, Stahl M. Hypomethylating Agents and FLT3 Inhibitors As Maintenance Treatment for Acute Myeloid Leukemia and Myelodysplastic Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation–A Systematic Review and Meta-Analysis. Transplantation And Cellular Therapy 2021, 27: 997.e1-997.e11. PMID: 34551341, PMCID: PMC9533376, DOI: 10.1016/j.jtct.2021.09.005.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaRelapse-free survivalMaintenance therapyMyelodysplastic syndromeFLT3 inhibitorsAllo-HCTHazard ratioMaintenance treatmentClinical trialsMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationControl groupSystematic reviewAllogeneic hematopoietic cell transplantMeasurable residual disease statusHematopoietic stem cell transplantationMeasurable residual disease testingNon-relapse mortalityHematopoietic cell transplantPre-emptive treatmentResidual disease statusStem cell transplantationFavorable safety profileSame patient cohortRandom-effects modelVenetoclax-based combinations in AML and high-risk MDS prior to and following allogeneic hematopoietic cell transplant
Bewersdorf JP, Derkach A, Gowda L, Menghrajani K, DeWolf S, Ruiz JD, Ponce DM, Shaffer BC, Tamari R, Young JW, Jakubowski AA, Gyurkocza B, Chan A, Xiao W, Glass J, King AC, Cai SF, Daniyan A, Famulare C, Cuello BM, Podoltsev NA, Roshal M, Giralt S, Perales MA, Seropian S, Cho C, Zeidan AM, Prebet T, Stein EM, Tallman MS, Goldberg AD, Stahl M. Venetoclax-based combinations in AML and high-risk MDS prior to and following allogeneic hematopoietic cell transplant. Leukemia & Lymphoma 2021, 62: 3394-3401. PMID: 34477024, PMCID: PMC9012492, DOI: 10.1080/10428194.2021.1966788.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaAllogeneic hematopoietic cell transplantAllo-HCTHematopoietic cell transplantSalvage therapyMyelodysplastic syndromeCell transplantMemorial Sloan-Kettering Cancer CenterHigh-risk myelodysplastic syndromeSecond allo-HCTSalvage treatment optionOverall response rateMedian followMedian OSVenetoclax therapyRetrospective studyCancer CenterTreatment optionsOS estimatesMyeloid leukemiaPatientsResponse rateTherapyTransplantMonthsOutcomes 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 modelThe development and clinical use of oral hypomethylating agents in acute myeloid leukemia and myelodysplastic syndromes: dawn of the total oral therapy era
Schiffer M, Zhao J, Johnson A, Lee J, Bewersdorf JP, Zeidan AM. The development and clinical use of oral hypomethylating agents in acute myeloid leukemia and myelodysplastic syndromes: dawn of the total oral therapy era. Expert Review Of Anticancer Therapy 2021, 21: 989-1002. PMID: 33853476, DOI: 10.1080/14737140.2021.1918002.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsChronic myelomonocytic leukemiaAcute myeloid leukemiaMyelodysplastic syndromeMyeloid leukemiaMyelomonocytic leukemiaHypomethylating agentAllogeneic hematopoietic cell transplantPost-transplant maintenance therapyAcute myeloid leukemia (AML) treatmentHematopoietic cell transplantHigh-risk MDSMyeloid leukemia treatmentPotential combination therapyCC-486Induction chemotherapyOral azacitidineTherapy eraMaintenance therapyOral agentsAdult patientsCell transplantClinical benefitMaintenance treatmentCombination therapyPredictive biomarkersRisk-Adapted, Individualized Treatment Strategies of Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML)
Bewersdorf JP, Zeidan AM. Risk-Adapted, Individualized Treatment Strategies of Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML). Cancers 2021, 13: 1610. PMID: 33807279, PMCID: PMC8036734, DOI: 10.3390/cancers13071610.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsChronic myelomonocytic leukemiaMyelodysplastic syndromeMyelomonocytic leukemiaAllogeneic hematopoietic cell transplantRed blood cell transfusionClinical symptom burdenLR-MDS patientsOverall survival benefitBlood cell transfusionHematopoietic cell transplantHigh-risk patientsMainstay of therapyErythropoiesis-stimulating agentsRisk of progressionRisk stratification toolIndividualized treatment strategiesAcute myeloid leukemiaRoutine clinical useAgent azacitidineAllo-HCTCell transfusionMost patientsSupportive careSurvival benefitSymptom burden
2020
Safety and Efficacy of Maintenance Treatment Following Allogeneic Hematopoietic Cell Transplant in Acute Myeloid Leukemia and Myelodysplastic Syndrome - a Systematic Review and Meta-Analysis
Bewersdorf J, Tallman M, Cho C, Zeidan A, Stahl M. Safety and Efficacy of Maintenance Treatment Following Allogeneic Hematopoietic Cell Transplant in Acute Myeloid Leukemia and Myelodysplastic Syndrome - a Systematic Review and Meta-Analysis. Blood 2020, 136: 34-35. DOI: 10.1182/blood-2020-136671.Peer-Reviewed Original ResearchAcute myeloid leukemiaRelapse-free survivalAllogeneic hematopoietic cell transplantHematopoietic cell transplantFLT3 inhibitorsMyelodysplastic syndromeAllo-HCTOverall survivalRFS ratesChronic GVHDMaintenance therapyCell transplantPatient selectionClinical trialsMyeloid leukemiaADC therapeuticsSystematic reviewMinimal residual disease testingAdvisory CommitteeDaiichi SankyoHigh-risk geneticsRate of GVHDPre-emptive treatmentCareful patient selectionPrognosis of patients
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