2025
Imetelstat in myeloid malignancies: current data and future directions
Bidikian A, Bewersdorf J, Kewan T, Podoltsev N, Stahl M, Zeidan A. Imetelstat in myeloid malignancies: current data and future directions. Expert Review Of Anticancer Therapy 2025, ahead-of-print: 1-12. PMID: 40116730, DOI: 10.1080/14737140.2025.2482721.Peer-Reviewed Original ResearchMyelodysplastic syndromeLR-MDSClinical trialsPotential disease-modifying propertiesLow-risk myelodysplastic syndromesElevated liver enzymesLR-MDS patientsTreat myelodysplastic syndromeSearch of PubMedTransfusion independenceEssential thrombocythemiaInfusion reactionsMyeloid malignanciesDisease-modifying propertiesCombination therapySurvival benefitEffective telomerase inhibitorImetelstatTelomerase reactivationPatient populationLiver enzymesMyelofibrosisCancer cellsMalignancyConference abstractsContemporary 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 statusAsciminib plus dasatinib and prednisone for Philadelphia chromosome–positive acute leukemia
Luskin M, Murakami M, Keating J, Flamand Y, Winer E, Garcia J, Stahl M, Stone R, Wadleigh M, Jaeckle S, Hagopian E, Weinstock D, Liegel J, McMasters M, Wang E, Stock W, DeAngelo D. Asciminib plus dasatinib and prednisone for Philadelphia chromosome–positive acute leukemia. Blood 2025, 145: 577-589. PMID: 39374521, DOI: 10.1182/blood.2024025800.Peer-Reviewed Original ResearchConceptsAcute leukemiaPhiladelphia chromosome-positive acute leukemiaRecommended phase 2 dosePh+ acute lymphoblastic leukemiaHematopoietic stem cell transplantationDe novo ALLHematologic remission rateLymphoid blast crisisMaximum tolerated doseStem cell transplantationPhase 1 studyChronic myeloid leukemiaMulticolor flow cytometryAcute lymphoblastic leukemiaVaso-occlusive eventsCytogenetic remissionBlast crisisSymptomatic pancreatitisTolerated doseRemission ratePh+ ALLCell transplantationMedian ageEnzyme elevationLymphoblastic leukemiaCardiovascular Risk Factors in Myeloproliferative Neoplasms: Associations with Survival and Thrombotic Outcomes
How J, Leiva O, Redd R, Marneth A, DeAngelo D, Dieli-Conwright C, El-Jawahri A, Kamaz B, Kim C, Lindsley C, Luskin M, Stahl M, Wazir M, Weeks L, Hobbs G. Cardiovascular Risk Factors in Myeloproliferative Neoplasms: Associations with Survival and Thrombotic Outcomes. Blood Vessels Thrombosis & Hemostasis 2025, 100051. DOI: 10.1016/j.bvth.2025.100051.Peer-Reviewed Original ResearchMyeloproliferative neoplasm patientsCardiovascular risk factorsEssential thrombocythemiaMyeloproliferative neoplasmsPolycythemia veraThrombotic outcomesThrombotic riskRisk factorsDiagnosis of hematological malignanciesMyeloproliferative neoplasm subtypeIncreased risk of deathType 2 diabetes mellitusMyeloproliferative neoplasms diagnosisRetrospective cohort analysisSex-matched controlsManaging cardiovascular risk factorsAssociated with survivalImprove patient morbidityRisk of deathMF patientsHematologic malignanciesMPN diagnosisPatient morbidityDana-FarberArterial/venous thrombosis
2024
Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them
Bidikian A, Bewersdorf J, Kewan T, Stahl M, Zeidan A. Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them. Cancers 2024, 16: 4092. PMID: 39682277, PMCID: PMC11640703, DOI: 10.3390/cancers16234092.Peer-Reviewed Original ResearchAcute promyelocytic leukemiaAdvent of all-trans retinoic acidEarly mortalityLong-term treatment toxicitiesArsenic trioxidePromyelocytic leukemiaClinical practiceIncidence of acute promyelocytic leukemiaRates of remissionLong-term outcomesTreatment of acute promyelocytic leukemiaLong-term survivalComprehensive patient evaluationResource-limited settingsTreatment toxicityAll-trans retinoic acidDelayed diagnosisPatient demographicsSignificant comorbiditiesTreatment initiationOlder patientsExpert centersClinical trialsTreatment outcomesReal-world settingsCost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML
Alhajahjeh A, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Goshua G, Bewersdorf J, Zeidan A. Cost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML. Leukemia & Lymphoma 2024, 66: 488-496. PMID: 39560957, DOI: 10.1080/10428194.2024.2426073.Peer-Reviewed Original ResearchConventional care regimensOlder patientsTreatment of older patientsIDH2 inhibitor enasidenibIncremental cost-effectiveness ratioCost-effectiveness ratioProbabilistic sensitivity analysesR/R AMLRefractory/relapsed AMLEvent-freeCost-effective treatmentEnasidenibCare regimensAMLPatientsCost-effectiveIncremental costLife yearsConventional careR/RIncremental effectSurvivalTreatmentA 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 therapyCost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States
Potnis K, Ito S, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Zeidan A, Bewersdorf J, Goshua G. Cost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States. Blood 2024, 144: 3644. DOI: 10.1182/blood-2024-204312.Peer-Reviewed Original ResearchSevere aplastic anemiaTreatment of severe aplastic anemiaQuality-adjusted life yearsAplastic anemiaIncremental net monetary benefitDeterministic sensitivity analysisProbabilistic sensitivity analysesImmunosuppressive therapyNewly diagnosed severe aplastic anemiaCost-effectiveness of eltrombopagOral thrombopoietin receptor agonistPatients treated with eltrombopagUntreated severe aplastic anemiaHematopoietic stem cell transplantationCost-effective therapeutic strategyDevelopment of adverse eventsLonger-term follow-up dataRACE trialsLater-line therapyThrombopoietin receptor agonistsPhase I/II trialSecond-line therapyStem cell transplantationU.S. payer perspectiveFirst-line treatmentComparative Analysis of Outcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients Harboring Very-High Risk Cytogenetics
Aguirre L, Bewersdorf J, Liu Y, Shallis R, Boussi L, Zucenka A, Garciaz S, Bystrom R, DeAngelo D, Stone R, Luskin M, Garcia J, Winer E, Ling K, Chen E, Wadleigh M, Stein E, Goldberg A, Zeidan A, Shimony S, Stahl M. Comparative Analysis of Outcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients Harboring Very-High Risk Cytogenetics. Blood 2024, 144: 4267-4267. DOI: 10.1182/blood-2024-202744.Peer-Reviewed Original ResearchMorphologic leukemia-free stateComposite complete remissionTreated with ICIntensive chemotherapyMonosomal karyotypeComplex karyotypeProgressive diseaseSurvival outcomesTreatment strategiesAllogeneic hematopoietic stem cell transplantationComposite complete remission rateTreated with intensive chemotherapyHematopoietic stem cell transplantationComparative analysis of outcomesDismal survival outcomesConventional cytotoxic chemotherapyAggressive disease biologyMulticenter retrospective cohortStem cell transplantationAnalyze survival outcomesKaplan-Meier methodLog-rank testAnalysis of outcomesCK-AMLCPX-351Outcomes 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 BlackHMA/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 regimens
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