2025
Contemporary understanding of myeloid-derived suppressor cells in the acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) tumor microenvironment
Alhajahjeh A, Stahl M, Kim T, Kewan T, Stempel J, Zeidan A, Bewersdorf J. Contemporary understanding of myeloid-derived suppressor cells in the acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) tumor microenvironment. Expert Review Of Anticancer Therapy 2025, ahead-of-print: 1-22. PMID: 40122075, DOI: 10.1080/14737140.2025.2483855.Peer-Reviewed Original ResearchMyeloid-derived suppressor cellsAcute myeloid leukemiaMyelodysplastic syndromeSuppressor cellsTumor microenvironmentMyeloid leukemiaEffects of myeloid-derived suppressor cellsTargets myeloid-derived suppressor cellsLeukemic stem cell survivalRisk of leukemia relapseMDSC-targeted therapiesMDSC-mediated immunosuppressionBone marrow nicheStem cell survivalCytokine-mediated pathwaysLeukemia relapseMyeloid diseasesImprove patient outcomesMarrow nichePost-transplantationPreclinical modelsImmunosuppressive propertiesImmunosuppressive componentsFunctional reprogrammingImmune evasionVenetoclax–Azacitidine Versus Azacitidine for the Treatment of Primary Refractory or First Relapsed Acute Myeloid Leukemia. An IPC‐DATAML‐MSKCC Retrospective Study
Petit C, Higue J, Acheaibi Z, Gilhodes J, Hospital M, Devillier R, Bewersdorf J, Goldberg A, Pigneux A, Vey N, Récher C, Stahl M, Bertoli S, Dumas P, Garciaz S. Venetoclax–Azacitidine Versus Azacitidine for the Treatment of Primary Refractory or First Relapsed Acute Myeloid Leukemia. An IPC‐DATAML‐MSKCC Retrospective Study. American Journal Of Hematology 2025, 100: 906-908. PMID: 40088036, DOI: 10.1002/ajh.27626.Peer-Reviewed Original ResearchAcute Myeloid Leukemia: 2025 Update on Diagnosis, Risk‐Stratification, and Management
Shimony S, Stahl M, Stone R. Acute Myeloid Leukemia: 2025 Update on Diagnosis, Risk‐Stratification, and Management. American Journal Of Hematology 2025, 100: 860-891. PMID: 39936576, PMCID: PMC11966364, DOI: 10.1002/ajh.27625.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaTherapeutic management of acute myeloid leukemiaManagement of acute myeloid leukemiaEuropean Leukemia NetworkStem cell cancerTherapeutic decision-makingImmature leukemia cellsExtra-medullary tissuesMRD findingsPrognostic factorsCell cancerTherapeutic algorithmRisk classification algorithmApproved therapiesMyeloid leukemiaResponse assessmentRisk stratificationBone marrowTherapeutic managementMonitoring of disease statusPathophysiological understandingDisease characteristicsMolecular findingsTherapeutic approachesDisease status
2024
A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS
Norman M, Yamartino K, Gerstein R, Shallis R, Mendez L, Podoltsev N, Stahl M, Eighmy W, Zeidan A. A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS. Expert Review Of Hematology 2024, 17: 755-767. PMID: 39474840, DOI: 10.1080/17474086.2024.2422554.Peer-Reviewed Original ResearchDiagnosed AMLSurvival benefitManagement of acute myeloid leukemiaDevelopment of oral therapiesIsocitrate dehydrogenase inhibitorsNewly diagnosed AMLManagement of adult patientsPost-transplant maintenanceAcute myeloid leukemiaSingle-arm studyExcellent response ratesIDH inhibitorsRelapsed AMLHypomethylating agentsInhibitor therapyMyelodysplastic syndromeOral therapyCombination therapyPost-transplantMyeloid leukemiaImproved survivalSingle-armAdult patientsAzacitidineRandomized studyImmune Landscape and Outcomes of Patients with RNA Splicing Factor-Mutant Acute Myeloid Leukemia and Myelodysplastic Syndromes Treated with Azacitidine +/- the Anti-PD-L1 Antibody Durvalumab
Bewersdorf J, Hasle V, Shallis R, Thompson E, De Menezes D, Rose S, Boss I, Mendez L, Podoltsev N, Stahl M, Kewan T, Halene S, Haferlach T, Fox B, Zeidan A. Immune Landscape and Outcomes of Patients with RNA Splicing Factor-Mutant Acute Myeloid Leukemia and Myelodysplastic Syndromes Treated with Azacitidine +/- the Anti-PD-L1 Antibody Durvalumab. Blood 2024, 144: 4585. DOI: 10.1182/blood-2024-194929.Peer-Reviewed Original ResearchAcute myeloid leukemiaAnti-PD-L1 antibody durvalumabOverall response rateMyelodysplastic syndromeComplete responseBM aspiratesMyeloid leukemiaInternational Working GroupBone marrowMyelodysplastic syndromes treated with azacitidineAcute myeloid leukemia ptsWild-type acute myeloid leukemiaSecondary acute myeloid leukemiaResponse criteriaAnti-PD-L1Immune checkpoint inhibitorsTreated with azacitidineOutcomes of patientsAny-cause deathGeneration of neoantigensVariant allele frequencySusceptible to treatmentMarrow CRAdverse cytogeneticsCheckpoint inhibitorsCost-Effectiveness of Allogeneic Hematopoietic Stem Cell Transplantation Versus Consolidation Chemotherapy for Patients with Intermediate Risk Acute Myeloid Leukemia
Alhajahjeh A, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Zeidan A, Goshua G, Bewersdorf J. Cost-Effectiveness of Allogeneic Hematopoietic Stem Cell Transplantation Versus Consolidation Chemotherapy for Patients with Intermediate Risk Acute Myeloid Leukemia. Blood 2024, 144: 788. DOI: 10.1182/blood-2024-201535.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationUpfront hematopoietic stem cell transplantationDisease-free survivalAcute myeloid leukemiaIntermediate risk acute myeloid leukemiaConsolidation chemotherapyCurative therapeutic modalityOverall survivalOne-way sensitivity analysesEuropean LeukemiaNetIncremental net monetary benefitMyeloid leukemiaMortality associated with hematopoietic stem cell transplantationCost-effective strategyTherapeutic modalitiesFavorable risk AMLSalvage hematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationCycles of consolidation chemotherapyUS health system perspectiveDiagnosed AMLFollow-up of patientsSurvival analysisHigh-dose cytarabineLines of therapyOutcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients with Chromosome 5 and 7 Abnormalities
Boussi L, Bewersdorf J, Liu Y, Shallis R, Aguirre L, Zucenka A, Garciaz S, Bystrom R, DeAngelo D, Stone R, Luskin M, Garcia J, Winer E, Chen E, Wadleigh M, Ling K, Zeidan A, Goldberg A, Stein E, Shimony S, Stahl M. Outcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients with Chromosome 5 and 7 Abnormalities. Blood 2024, 144: 4281-4281. DOI: 10.1182/blood-2024-204467.Peer-Reviewed Original ResearchAcute myeloid leukemiaMedian OSTP53 co-mutationsTreated with ICIntensive chemotherapyComposite CRCo-mutationsComplete remissionOverall survivalPts ageAllogeneic stem cell transplantationSecondary acute myeloid leukemiaDiagnosed AMLAcute myeloid leukemia patientsAssociated with poor outcomesEstimate overall survivalStem cell transplantationKaplan-Meier methodLog-rank testPredictors of survivalCPX-351Monosomy 5MRD negativityInduction therapyComplex karyotypeThe Prognostic and Predictive Value of RUNX1 Mutations in Newly Diagnosed Acute Myeloid Leukemia - an International Multicenter Cohort Study
Bystrom R, Bewersdorf J, Liu Y, Schaefer E, Shallis R, Boussi L, Zucenka A, Garciaz S, Aguirre L, DeAngelo D, Stone R, Luskin M, Garcia J, Winer E, Chen E, Wadleigh M, Ling K, Stein E, Goldberg A, Zeidan A, Shimony S, Stahl M. The Prognostic and Predictive Value of RUNX1 Mutations in Newly Diagnosed Acute Myeloid Leukemia - an International Multicenter Cohort Study. Blood 2024, 144: 2947-2947. DOI: 10.1182/blood-2024-205581.Peer-Reviewed Original ResearchAcute myeloid leukemiaComposite complete responseMedian OSRUNX1 mutationsComplete responseIntensive chemotherapyOverall survivalMyelodysplastic syndromeAllo-SCTIntermediate riskPredictive valueMyeloid leukemiaInternational multicenter retrospective cohort studyTreatment strategiesCohort studyNewly diagnosed acute myeloid leukemiaAllogeneic stem cell transplantationMulticenter retrospective cohort studyNext-generation sequencingAntecedent MDSConcomitant TP53 mutationIncomplete count recoveryTreated with ICStem cell transplantationAdverse risk featuresVenetoclax-Based Therapy Versus Intensive Chemotherapy Followed By Allogeneic-Stem Cell Transplantation for High-Risk Elderly Acute Myeloid Leukemia
Iat A, Bewersdorf J, Gilhodes J, Liu Y, Shallis R, Boussi L, Zucenka A, Bystrom R, DeAngelo D, Berton G, Soua A, Ling K, Aguirre L, Stone R, Luskin M, Garcia J, Winer E, Chen E, Wadleigh M, Goldberg A, Zeidan A, Cluzeau T, Shimony S, Stahl M, Garciaz S. Venetoclax-Based Therapy Versus Intensive Chemotherapy Followed By Allogeneic-Stem Cell Transplantation for High-Risk Elderly Acute Myeloid Leukemia. Blood 2024, 144: 1508. DOI: 10.1182/blood-2024-207045.Peer-Reviewed Original ResearchCumulative incidence of relapseRelapse-free survivalAcute myeloid leukemiaOverall response rateAdverse risk cytogeneticsAllo-SCTVEN-based therapyNon-relapse mortalityIntensive chemotherapyComposite CROverall survivalIC groupMedian OSMyeloid leukemiaElderly patientsTherapy-related acute myeloid leukemiaHigh-risk acute myeloid leukemiaAllogeneic stem-cell transplantationMedian time to relapseElderly acute myeloid leukemiaLong-term disease controlResponse rateFrequent cytogenetic alterationsProspective validation trialVIALE-A trialDual Bclxl and BCL2 Inhibition with Navitoclax (NAV), Venetoclax (VEN), and Decitabine (DEC) for Advanced Myeloid Neoplasms (MN): Safety and Biological Activity in a Phase 1 Study
Chen E, Liu Y, Bell H, Galinsky I, Luskin M, Winer E, Stahl M, Vedula R, Volpe V, DeAngelo D, Quillen K, Ryan J, Minihane E, Hersch M, Leonard R, Neuberg D, Stone R, Letai A, Lane A, Garcia J. Dual Bclxl and BCL2 Inhibition with Navitoclax (NAV), Venetoclax (VEN), and Decitabine (DEC) for Advanced Myeloid Neoplasms (MN): Safety and Biological Activity in a Phase 1 Study. Blood 2024, 144: 4580-4580. DOI: 10.1182/blood-2024-198284.Peer-Reviewed Original ResearchAcute myeloid leukemiaAdvanced myeloid neoplasmsDose-limiting toxicityPhase 1 studyIncreased apoptotic primingMyeloid neoplasmsNavitoclax doseOverall survivalDNA methyltransferase inhibitorHematologic recoveryApoptotic primingTherapy-related acute myeloid leukemiaDose levelsHematologic dose-limiting toxicityHigh-risk myeloid neoplasmsRecommended phase 2 doseSecondary acute myeloid leukemiaNewly-diagnosed acute myeloid leukemiaResponse rateIncomplete hematologic recoveryPartial hematologic recoveryPhase 2 doseBCL2 inhibitor venetoclaxImmature platelet fractionMyelodysplastic syndrome/myeloproliferative neoplasmImpact of Molecular Ontogeny on Hematological Recovery in AML Patients Treated with Hypomethylating Agent Plus Venetoclax Therapy
Zhang J, Shimony S, Liu Y, Inyang E, Chen E, Aguirre L, Bystrom R, Rolles B, Stone R, DeAngelo D, Tiao E, Stahl M. Impact of Molecular Ontogeny on Hematological Recovery in AML Patients Treated with Hypomethylating Agent Plus Venetoclax Therapy. Blood 2024, 144: 5175-5175. DOI: 10.1182/blood-2024-200358.Peer-Reviewed Original ResearchAcute myeloid leukemiaAbsolute neutrophil countTP53-mutated acute myeloid leukemiaDe novo acute myeloid leukemiaMorphologic leukemia-free stateTP53-MTDe novo groupHypomethylating agentsCount recoveryTP53 mutationsRed blood cellsNewly diagnosed acute myeloid leukemiaMedian absolute neutrophil countHigh-risk myelodysplastic syndromePresence of TP53 mutationsDiagnosed AMLCycle 3Acute myeloid leukemia patientsCycle 1Dose of venetoclaxAssociated with prior historyCycle lengthIncomplete count recoveryBlood cellsCycles of treatmentImpact of Social Determinants of Health on Overall Survival in Patients with AML By European Leukemianet Risk Group
Shimony S, Wang Y, Cronin A, Walsh T, Charles A, Slopen N, Stahl M, Bystrom R, Luskin M, Abel G, DeAngelo D, Stone R, Lindsley R, Hantel A. Impact of Social Determinants of Health on Overall Survival in Patients with AML By European Leukemianet Risk Group. Blood 2024, 144: 5171-5171. DOI: 10.1182/blood-2024-203644.Peer-Reviewed Original ResearchNational Area Deprivation IndexSocial determinants of healthDeterminants of healthHSCT ratesImpact of social determinants of healthMeasures of socioeconomic disadvantageNon-Hispanic (NHArea Deprivation IndexRisk groupsImpact of SDoHElectronic medical recordsHigher levels of disadvantageHematopoietic stem cell transplantationOutcomes of overall survivalLevels of disadvantageDana-Farber Cancer InstituteCensus block group levelBetween-group comparisonsDeprivation indexIntermediate risk patientsNH AsiansSocial determinantsAcute myeloid leukemiaBlock group levelNH BlackFinal Analysis of a Phase I Study of Escalating Doses of the BCL-2 Inhibitor Venetoclax in Combination with Daunorubcin/Cytarabine Induction and High Dose Cytarabine Consolidation in Previously Untreated Adults with Acute Myeloid Leukemia
Stone R, DeAngelo D, Letai A, Galinsky I, Weiner H, Halpin A, Noyes L, Smith H, Lombardi C, Leonard R, Fell G, Flammand Y, Ryan J, Konopleva M, Wadleigh M, Stahl M, Chen E, Volpe V, Winer E, Garcia J, Luskin M, Patel A. Final Analysis of a Phase I Study of Escalating Doses of the BCL-2 Inhibitor Venetoclax in Combination with Daunorubcin/Cytarabine Induction and High Dose Cytarabine Consolidation in Previously Untreated Adults with Acute Myeloid Leukemia. Blood 2024, 144: 4261. DOI: 10.1182/blood-2024-203683.Peer-Reviewed Original ResearchMaximum tolerated doseAcute myeloid leukemiaMeasurable residual diseaseBCL-2 inhibitor venetoclaxAra-CExpansion cohortInhibitor venetoclaxANC recoverySeptic deathMyeloid leukemiaEscalation phaseDose levelsMRD negativityNegative measurable residual diseaseUntreated acute myeloid leukemiaRate of complete remissionUntreated adultsBcl-2High dose cytarabineTumor lysis syndromeInitiation of therapyPhase I studyAra-C doseCore binding factor rearrangementsCytarabine consolidationHMA/Ven Treatment Modifications and Associated Outcomes in IDH -Mutant AML
Chin K, Derkach A, Famulare C, Gupta G, Borge P, Geyer M, Goldberg A, Haque T, Park J, Roeker L, Tallman M, Stahl M, Stein E. HMA/Ven Treatment Modifications and Associated Outcomes in IDH -Mutant AML. Blood 2024, 144: 5962-5962. DOI: 10.1182/blood-2024-211733.Peer-Reviewed Original ResearchAcute myeloid leukemiaMedian overall survivalIsocitrate dehydrogenase-mutantCommon Terminology Criteria for Adverse EventsMeasurable residual diseaseOverall survivalTreatment modificationNo significant differenceCR/CRi rateMRD negativityHypomethylating agentsSignificant differenceLong-term survival outcomesSingle-center retrospective studyToxicity associated with treatmentMutant acute myeloid leukemiaCycle lengthChemotherapy-ineligible patientsGrade 3 neutropeniaMRD-negativity ratesTreatment cycle characteristicsAssociated with higher responseEvent-free survivalKaplan-Meier methodologyEfficacious treatment regimensHMA/VEN treatment modifications and associated outcomes in IDH-mutant AML
Chin K, Derkach A, Famulare C, Gupta G, Borge P, Geyer M, Goldberg A, Haque T, Park J, Roeker L, Tallman M, Stahl M, Stein E. HMA/VEN treatment modifications and associated outcomes in IDH-mutant AML. Leukemia & Lymphoma 2024, 66: 270-278. PMID: 39397429, DOI: 10.1080/10428194.2024.2411436.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaTreatment modificationHypomethylating agentsResponse rateAssociated with lower response ratesMedian overall survivalIDH-mutated acute myeloid leukemiaLong-term toxicityCR/CRi rateSignificant neutropeniaFebrile neutropeniaInduction chemotherapyOverall survivalMyeloid leukemiaLow response rateSurvival rateAffect survivalNeutropeniaVenetoclaxSurvivalED visitsPatientsR/RMonthsReal-world settingsIntensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML
Bewersdorf J, Shimony S, Shallis R, Liu Y, Berton G, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Bystrom R, Lindsley R, Chen E, Perez J, Stein A, Pullarkat V, Aldoss I, DeAngelo D, Neuberg D, Stone R, Garciaz S, Ball B, Stahl M. Intensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML. Blood Advances 2024, 8: 4845-4855. PMID: 38941537, PMCID: PMC11416634, DOI: 10.1182/bloodadvances.2024012858.Peer-Reviewed Original ResearchIntensive induction chemotherapyAcute myeloid leukemiaNPM1-Mutant Acute Myeloid LeukemiaInduction chemotherapyHypomethylating agentsMulticenter retrospective cohort study of patientsPatients treated with ICAllogeneic stem cell transplantationRetrospective cohort study of patientsMulticenter retrospective cohort studyCohort study of patientsComposite complete remissionStem cell transplantationYears-oldFLT3-ITD mutationStudy of patientsStandard of careNormal cytogeneticsComplete remissionCell transplantationNPM1 mutationsMyeloid leukemiaFLT3-ITDYounger patientsOlder patientsBeyond HMAs: Novel Targets and Therapeutic Approaches
Getz T, Bewersdorf J, Kewan T, Stempel J, Bidikian A, Shallis R, Stahl M, Zeidan A. Beyond HMAs: Novel Targets and Therapeutic Approaches. Seminars In Hematology 2024, 61: 358-369. PMID: 39389839, DOI: 10.1053/j.seminhematol.2024.08.001.Peer-Reviewed Original ResearchAcute myeloid leukemiaDelays progression to acute myeloid leukemiaHeterogeneous group of clonal hematopoietic disordersGroup of clonal hematopoietic disordersMolecular International Prognostic Scoring SystemRandomized phase 3 clinical trialProgression to acute myeloid leukemiaInternational Prognostic Scoring SystemLower-risk MDS patientsRisk stratification of patientsPhase 3 clinical trialsCombination of azacitidineHypomethylating agent combinationsCurrent treatment landscapeFirst line therapyPrognostic Scoring SystemBiomarker-directed therapiesClonal hematopoietic disordersLack of therapeutic agentsStratification of patientsEarly phase trialsErythropoiesis stimulating agentsTreated with therapiesVariable clinical featuresStandard of careSubunit-specific analysis of cohesin-mutant myeloid malignancies reveals distinct ontogeny and outcomes
Jann J, Hergott C, Winkler M, Liu Y, Braun B, Charles A, Copson K, Barua S, Meggendorfer M, Nadarajah N, Shimony S, Winer E, Wadleigh M, Stone R, DeAngelo D, Garcia J, Haferlach T, Lindsley R, Luskin M, Stahl M, Tothova Z. Subunit-specific analysis of cohesin-mutant myeloid malignancies reveals distinct ontogeny and outcomes. Leukemia 2024, 38: 1992-2002. PMID: 39033241, PMCID: PMC11347381, DOI: 10.1038/s41375-024-02347-y.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaDana-Farber Cancer InstituteMyelodysplastic neoplasmsCohesin complex componentSubunit specificityAssociated with secondary AMLCohesin complexDe novo acute myeloid leukemiaSecondary acute myeloid leukemiaComplex mutationsCohesinGenetic driversGenetic characteristicsSTAG2 mutationsCo-occurrenceSubunit mutationsMutationsMyeloid malignanciesPrognostic significanceAdverse prognosisPrognostic classificationMyeloid leukemiaClinical characteristicsDana-FarberOntogenyAcute myeloid leukemia (AML) with chromosome 3 inversion: biology, management, and clinical outcome
Alhajahjeh A, Bewersdorf J, Bystrom R, Zeidan A, Shimony S, Stahl M. Acute myeloid leukemia (AML) with chromosome 3 inversion: biology, management, and clinical outcome. Leukemia & Lymphoma 2024, 65: 1541-1551. PMID: 38962996, DOI: 10.1080/10428194.2024.2367040.Peer-Reviewed Original ResearchAcute myeloid leukemiaIntensive chemotherapyHypomethylating agentsMyeloid leukemiaAllogeneic stem cell transplantationAcute myeloid leukemia casesAcute myeloid leukemia subtypesStem cell transplantationComplex hematological malignancyCurrent treatment modalitiesRare genetic anomalyCell transplantationHematologic malignanciesTreatment modalitiesClinical outcomesTreatment responseInv(3Genetic alterationsLeukemia developmentTreatment strategiesCellular processesGenetic anomaliesLeukemiaFusion geneClinical implicationsPrognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases
Badar T, Nanaa A, Atallah E, Shallis R, Craver E, Li Z, Goldberg A, Saliba A, Patel A, Bewersdorf J, Duvall A, Burkart M, Bradshaw D, Abaza Y, Stahl M, Palmisiano N, Murthy S, Zeidan A, Kota V, Patnaik M, Litzow M. Prognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases. Haematologica 2024, 109: 3533-3542. PMID: 38813716, PMCID: PMC11532685, DOI: 10.3324/haematol.2024.285000.Peer-Reviewed Original ResearchAcute myeloid leukemiaMyelodysplastic syndromeComplex cytogeneticsMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationLower-risk myelodysplastic syndromesHematopoietic stem cell transplantationHigher-risk myelodysplastic syndromesOutcomes of SHStem cell transplantationAllo-HCTTP53 alterationsPrognostic impactMyeloid malignanciesTP53 mutationsCell transplantationFLT3-ITDIDH1 mutationMultivariate analysisSupportive careUS academic institutionsNeoplastic diseasePatientsSuperior EFSPredicting outcome
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