2025
Acute 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 ResearchMeSH KeywordsAlgorithmsDisease ManagementHumansLeukemia, Myeloid, AcuteNeoplasm, ResidualPrognosisRisk AssessmentConceptsAcute 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
HMA/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 settingsSubunit-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-FarberOntogenyTP53 Mutations in Acute Leukemias and Myelodysplastic Syndromes: Insights and Treatment Updates.
Santini V, Stahl M, Sallman D. TP53 Mutations in Acute Leukemias and Myelodysplastic Syndromes: Insights and Treatment Updates. American Society Of Clinical Oncology Educational Book 2024, 44: e432650. PMID: 38768424, DOI: 10.1200/edbk_432650.Peer-Reviewed Original ResearchMeSH KeywordsHumansLeukemia, Myeloid, AcuteMolecular Targeted TherapyMutationMyelodysplastic SyndromesTumor Suppressor Protein p53ConceptsMissense mutationsResistant to current chemotherapyLoss-of-function effectPhase II trialPhase III trialsAnti-CD47 antibodyAnti-CD123 antibodyResponse to treatmentPressure of chemotherapyApoptosis systemP53 functionChromosome lossTP53 mutationsTruncating mutationsInhibitor therapyMyelodysplastic syndromeOverall survivalII trialIII trialsSelection pressureAnti-CD123Acute leukemiaAML casesDismal diseaseImproved survivalMolecular ontogeny underlies the benefit of adding venetoclax to hypomethylating agents in newly diagnosed AML patients
Shimony S, Garcia J, Keating J, Chen E, Luskin M, Stahl M, Neuberg D, DeAngelo D, Stone R, Lindsley R. Molecular ontogeny underlies the benefit of adding venetoclax to hypomethylating agents in newly diagnosed AML patients. Leukemia 2024, 38: 1494-1500. PMID: 38538860, PMCID: PMC11216982, DOI: 10.1038/s41375-024-02230-w.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedAged, 80 and overAntineoplastic Combined Chemotherapy ProtocolsBridged Bicyclo Compounds, HeterocyclicDNA MethylationFemaleHematopoietic Stem Cell TransplantationHumansLeukemia, Myeloid, AcuteMaleMiddle AgedMutationPrognosisRemission InductionSulfonamidesSurvival RateTumor Suppressor Protein p53Young AdultConceptsAcute myeloid leukemiaDiagnosed AML patientsHypomethylating agentsAML patientsTP53-mutated acute myeloid leukemiaPatients treated with intensive chemotherapyAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationComposite complete remissionStem cell transplantationGroup of patientsMolecular ontogenyMedian OSOS benefitComplete remissionIntensive chemotherapyCell transplantationClinicopathological variablesMyeloid leukemiaClinical benefitClinical impactSplicing mutationPatientsSecondary groupVenetoclaxPhase 1b trial of tagraxofusp in combination with azacitidine with or without venetoclax in acute myeloid leukemia
Lane A, Garcia J, Raulston E, Garzon J, Galinsky I, Baxter E, Leonard R, DeAngelo D, Luskin M, Reilly C, Stahl M, Stone R, Vedula R, Wadleigh M, Winer E, Mughal T, Brooks C, Gupta I, Stevenson K, Neuberg D, Ren S, Keating J, Konopleva M, Stein A, Pemmaraju N. Phase 1b trial of tagraxofusp in combination with azacitidine with or without venetoclax in acute myeloid leukemia. Blood Advances 2024, 8: 591-602. PMID: 38052038, PMCID: PMC10837492, DOI: 10.1182/bloodadvances.2023011721.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaAgent azacitidineMyelodysplastic syndromeMyeloid leukemiaTreatment of blastic plasmacytoid dendritic cell neoplasmHigh-risk acute myeloid leukemiaBlastic plasmacytoid dendritic cell neoplasmDNA hypomethylating agent azacitidineRecommended phase 2 doseHigh-risk myelodysplastic syndromeAdverse-risk acute myeloid leukaemiaBCL-2 inhibitor venetoclaxPlasmacytoid dendritic cell neoplasmInterleukin-3Bcl-2Median overall survivalPhase 1b studyProgression-free survivalPhase 1b trialHypomethylating agent azacitidineDendritic cell neoplasmAntiapoptotic molecule Bcl-2Recombinant interleukin-3Interleukin-3 receptorExpansion cohort
2023
Diagnostic Precision or Pitfalls: How to Apply the New Acute Myeloid Leukemia Classification Systems?
Carlson K, Cunningham A, Stahl M, Winer E, Michaelis L. Diagnostic Precision or Pitfalls: How to Apply the New Acute Myeloid Leukemia Classification Systems? Acta Haematologica 2023, 147: 122-133. PMID: 38071966, DOI: 10.1159/000535607.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaAML classificationWorld Health OrganizationMyelodysplasia-related acute myeloid leukemiaClassification of acute myeloid leukemiaRevised 4th editionWorld Health Organization blue bookTreatment decision-makingMyeloid leukemiaClassification systemTherapy-relatedClinical trialsBlast thresholdHematologic oncologistsClinical scenariosTreatment decisionsDiagnostic precisionHealth OrganizationWhen to use which molecular prognostic scoring system in the management of patients with MDS?
Kewan T, Bewersdorf J, Gurnari C, Xie Z, Stahl M, Zeidan A. When to use which molecular prognostic scoring system in the management of patients with MDS? Best Practice & Research Clinical Haematology 2023, 36: 101517. PMID: 38092484, DOI: 10.1016/j.beha.2023.101517.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsClinical Decision-MakingHumansLeukemia, Myeloid, AcuteMutationMyelodysplastic SyndromesPrognosisConceptsInternational Prognostic Scoring SystemPrognostic scoring systemAcute myeloid leukemiaScoring systemRisk stratificationRecurrent molecular alterationsHigh-risk patientsAppropriate risk stratificationManagement of patientsRecurrent genetic mutationsIntensive therapyMyeloid leukemiaTreatment strategiesPrognostic toolDisease pathogenesisMolecular alterationsHematopoietic cancersClinical decisionHeterogeneous groupGenetic mutationsNext-generation sequencingPrognostic systemPatientsVariable propensitySubsequent revisionFLT3-ITD does not predict inferior prognosis in acute myeloid leukemia patients age ≥60 years
Shimony S, Fell G, Chen E, Tsai H, Wadleigh M, Winer E, Garcia J, Luskin M, Stahl M, Neuberg D, DeAngelo D, Lindsley R, Stone R. FLT3-ITD does not predict inferior prognosis in acute myeloid leukemia patients age ≥60 years. Blood Advances 2023, 7: 5354-5358. PMID: 37163357, PMCID: PMC10509660, DOI: 10.1182/bloodadvances.2023009748.Peer-Reviewed Original ResearchTargeting apoptosis dysregulation in myeloid malignancies - The promise of a therapeutic revolution
Santinelli E, Pascale M, Xie Z, Badar T, Stahl M, Bewersdorf J, Gurnari C, Zeidan A. Targeting apoptosis dysregulation in myeloid malignancies - The promise of a therapeutic revolution. Blood Reviews 2023, 62: 101130. PMID: 37679263, DOI: 10.1016/j.blre.2023.101130.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAntineoplastic AgentsApoptosisHumansLeukemia, Myeloid, AcuteMyeloproliferative DisordersConceptsAcute myeloid leukemiaMyeloid malignanciesLeukemia cell survivalBcl-2 signalingPro-apoptotic agentsClinical studiesMyeloid leukemiaMyeloid neoplasmsTherapeutic revolutionMolecular alterationsMyeloid neoplasiaTherapeutic landscapeSpecific drugsApoptosis dysregulationSynergistic efficacyNew drugsMechanism of apoptosisDrugsMalignancyCombination strategiesCell survivalAttractive targetApoptotic pathwayTreatmentApoptosisSafety and efficacy of FLAG-Ida-based therapy combined with venetoclax for the treatment for newly diagnosed and relapsed/refractory patients with AML – A systematic review
Sherban A, Fredman D, Shimony S, Yeshurun M, Raanani P, Stahl M, Gafter-Gvili A, Wolach O. Safety and efficacy of FLAG-Ida-based therapy combined with venetoclax for the treatment for newly diagnosed and relapsed/refractory patients with AML – A systematic review. Leukemia Research 2023, 133: 107368. PMID: 37598660, DOI: 10.1016/j.leukres.2023.107368.Peer-Reviewed Original ResearchMeSH KeywordsAntineoplastic Combined Chemotherapy ProtocolsBridged Bicyclo Compounds, HeterocyclicCytarabineHumansIdarubicinLeukemia, Myeloid, AcuteConceptsAcute myeloid leukemiaIntensive chemotherapyEfficacy outcomesHigh-risk acute myeloid leukemiaResponse to treatmentFLAG-IdaRelapsed/refractory patientsMyeloid leukemiaTreated patientsVenetoclaxSafety outcomesSystematic reviewEfficacyInfection ratePatientsTreatmentOutcomesChemotherapyLeukemiaSafetyTherapyReviewInfectionA systematic reviewIncidence and predictors of anthracycline-related left ventricular dysfunction in acute myeloid leukemia
Stahl M, Giblin G, Liu Y, Winer E, Garcia J, Chen E, Wadleigh M, Ling K, Lindsley R, Shimony S, Copson K, Charles A, DeAngelo D, Stone R, Nohria A, Luskin M. Incidence and predictors of anthracycline-related left ventricular dysfunction in acute myeloid leukemia. Leukemia Research 2023, 132: 107351. PMID: 37451200, DOI: 10.1016/j.leukres.2023.107351.Peer-Reviewed Original ResearchMeSH KeywordsAdultAnthracyclinesAntibiotics, AntineoplasticHumansIncidenceLeukemia, Myeloid, AcuteStroke VolumeVentricular Dysfunction, LeftVentricular Function, LeftConceptsAcute myeloid leukemiaInduction chemotherapyVentricular dysfunctionMyeloid leukemiaAllogeneic stem cell transplantationGroup of AML patientsGenetic predictorsBaseline cardiovascular comorbiditiesInclusion criteriaCumulative anthracycline doseStem cell transplantationVentricular ejection fractionConsecutive adult patientsLeft ventricular dysfunctionDana-Farber Cancer InstituteMyeloid mutationsAllo-SCTAnthracycline dosePost-remissionAML patientsCell transplantationEchocardiographic assessmentEjection fractionPrimary endpointJAK2 mutationEpidemiology and Pathogenesis of Myelodysplastic Syndrome
Rotter L, Shimony S, Ling K, Chen E, Shallis R, Zeidan A, Stahl M. Epidemiology and Pathogenesis of Myelodysplastic Syndrome. The Cancer Journal 2023, 29: 111-121. PMID: 37195766, DOI: 10.1097/ppo.0000000000000665.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsEpigenesis, GeneticHematopoiesisHumansLeukemia, Myeloid, AcuteMutationMyelodysplastic SyndromesConceptsMyelodysplastic syndromeClonal hematopoiesisMDS pathophysiologyFrank MDSOutcomes of patientsOngoing clinical trialsAcute myeloid leukemiaIndividualized therapeutic approachesRisk assessment toolVariable cytopeniasOverall incidenceUnderlying pathophysiologyClinical trialsIneffective hematopoiesisMyeloid leukemiaNovel therapiesClonal cytopeniaTherapeutic modalitiesClonal disorderTherapeutic approachesUnique molecular profileEpidemiological assessmentDisease evolutionUnknown significancePathophysiologyEvolution of Therapeutic Benefit Measurement Criteria in Myelodysplastic Syndromes/Neoplasms
Stempel J, Xie Z, Bewersdorf J, Stahl M, Zeidan A. Evolution of Therapeutic Benefit Measurement Criteria in Myelodysplastic Syndromes/Neoplasms. The Cancer Journal 2023, 29: 203-211. PMID: 37195777, DOI: 10.1097/ppo.0000000000000666.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsInternational Working Group response criteriaResponse criteriaPhase III clinical trialsIWG 2006 criteriaRisk of progressionPatient-focused outcomesClonal myeloid neoplasmsAcute myeloid leukemiaTherapeutic response assessmentPatient-centered responsesNovel drug developmentHematologic recoveryProgressive cytopeniasClinical trialsIWG criteriaLong-term benefitsMyeloid leukemiaIneffective hematopoiesisMyeloid neoplasmsResponse assessmentDisease severitySurvival of TP53-mutated acute myeloid leukemia patients receiving allogeneic stem cell transplantation after first induction or salvage therapy: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND)
Badar T, Atallah E, Shallis R, Saliba A, Patel A, Bewersdorf J, Grenet J, Stahl M, Duvall A, Burkart M, Palmisiano N, Bradshaw D, Kubiak M, Dinner S, Goldberg A, Abaza Y, Murthy G, Kota V, Litzow M. Survival of TP53-mutated acute myeloid leukemia patients receiving allogeneic stem cell transplantation after first induction or salvage therapy: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases (COMMAND). Leukemia 2023, 37: 799-806. PMID: 36807649, DOI: 10.1038/s41375-023-01847-7.Peer-Reviewed Original ResearchConceptsEvent-free survivalAllo-HSCTOverall survivalChronic GVHDAllogeneic hematopoietic stem cell transplantAllogeneic stem cell transplantationMedian event-free survivalHematopoietic stem cell transplantAcute myeloid leukemia patientsMedian overall survivalPost allo-HSCTReduced intensity conditioningStem cell transplantStem cell transplantationLong-term outcomesMyeloid leukemia patientsMulti-center studyAcute graftComplete remissionHost diseaseSalvage therapyComplex cytogeneticsMyeloablative conditioningMedian ageCell transplantAcute myeloid leukemia: 2023 update on diagnosis, risk‐stratification, and management
Shimony S, Stahl M, Stone R. Acute myeloid leukemia: 2023 update on diagnosis, risk‐stratification, and management. American Journal Of Hematology 2023, 98: 502-526. PMID: 36594187, DOI: 10.1002/ajh.26822.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaTherapeutic landscape of acute myeloid leukemiaPatient acute myeloid leukemiaInternational Consensus ClassificationMalignant myeloid cellsStem cell cancerTherapeutic decision makingMarrow stem cellsCell cancerTherapeutic algorithmMyeloid leukemiaMyeloid cellsRisk stratificationWHO systemTherapeutic complexMutation profilesDisease characteristicsMolecular findingsConsensus classificationTherapeutic landscapeStem cellsRisk classificationLeukemiaIntegration of molecular analysisTrial results
2022
Disparities in receiving disease‐directed therapy, allogeneic stem cell transplantation in non‐Hispanic Black patients with TP53‐mutated acute myeloid leukemia
Badar T, Litzow M, Shallis R, Patel A, Saliba A, Burkart M, Bewersdorf J, Stahl M, De Camargo Correia G, Murthy S, Abaza Y, Duvall A, Bradshaw D, Kota V, Dinner S, Goldberg A, Palmisiano N, Al Kali A, Atallah E. Disparities in receiving disease‐directed therapy, allogeneic stem cell transplantation in non‐Hispanic Black patients with TP53‐mutated acute myeloid leukemia. Cancer 2022, 129: 934-945. PMID: 36545710, DOI: 10.1002/cncr.34604.Peer-Reviewed Original ResearchMeSH KeywordsBlack PeopleHealthcare DisparitiesHematopoietic Stem Cell TransplantationHumansLeukemia, Myeloid, AcuteRetrospective StudiesTumor Suppressor Protein p53White PeopleConceptsAcute myeloid leukemiaAllogeneic stem cell transplantationNHB patientsStem cell transplantationNHW patientsCell transplantationMyeloid leukemiaTherapy-related acute myeloid leukemiaNon-Hispanic black patientsCurative-intent therapyLow-intensity chemotherapyBest supportive careComplete response rateMedian patient ageProportion of patientsSubset of patientsDisease-directed therapyHigher proportionIntensive chemotherapyPatient ageSupportive careBlack patientsClinical outcomesTreatment disparitiesRetrospective studySafety and efficacy of CPX-351 in younger patients (< 60 years) with secondary acute myeloid leukemia
Przespolewski A, Goldberg A, Talati C, Fazal S, Vachhani P, Sanikommu S, Thota S, Waksal J, Ball B, Famulare C, Stahl M, Baron J, Griffiths E, Thompson J, Sweet K, Wang E. Safety and efficacy of CPX-351 in younger patients (< 60 years) with secondary acute myeloid leukemia. Blood 2022, 141: 1489-1493. PMID: 36493344, DOI: 10.1182/blood.2022016678.Peer-Reviewed Original ResearchCost-effectiveness of azacitidine and ivosidenib in newly diagnosed older, intensive chemotherapy-ineligible patients with IDH1-mutant acute myeloid leukemia
Bewersdorf J, Patel K, Goshua G, Shallis R, Podoltsev N, Stahl M, Stein E, Huntington S, Zeidan A. Cost-effectiveness of azacitidine and ivosidenib in newly diagnosed older, intensive chemotherapy-ineligible patients with IDH1-mutant acute myeloid leukemia. Leukemia & Lymphoma 2022, 64: 454-461. PMID: 36493798, PMCID: PMC9957935, DOI: 10.1080/10428194.2022.2140288.Peer-Reviewed Original ResearchMeSH KeywordsAzacitidineCost-Benefit AnalysisHumansIsocitrate DehydrogenaseLeukemia, Myeloid, AcutePyridinesQuality-Adjusted Life YearsUnited StatesMolecular predictors of immunophenotypic measurable residual disease clearance in acute myeloid leukemia
Stahl M, Derkach A, Farnoud N, Bewersdorf J, Robinson T, Famulare C, Cho C, Devlin S, Menghrajani K, Patel M, Cai S, Miles L, Bowman R, Geyer M, Dunbar A, Epstein‐Peterson Z, McGovern E, Schulman J, Glass J, Taylor J, Viny A, Stein E, Getta B, Arcila M, Gao Q, Barker J, Shaffer B, Papadopoulos E, Gyurkocza B, Perales M, Abdel‐Wahab O, Levine R, Giralt S, Zhang Y, Xiao W, Pai N, Papaemmanuil E, Tallman M, Roshal M, Goldberg A. Molecular predictors of immunophenotypic measurable residual disease clearance in acute myeloid leukemia. American Journal Of Hematology 2022, 98: 79-89. PMID: 36251406, PMCID: PMC10080561, DOI: 10.1002/ajh.26757.Peer-Reviewed Original ResearchMeSH KeywordsHematopoietic Stem Cell TransplantationHumansLeukemia, Myeloid, AcuteNeoplasm, ResidualPrognosisRemission InductionStem Cell TransplantationTransplantation, HomologousConceptsMeasurable residual diseaseAcute myeloid leukemiaAllo-SCTInduction chemotherapyPersistent diseaseMyeloid leukemiaAllogeneic stem cell transplantationStem cell transplantationMonosomy 5Residual diseaseDisease clearanceKaryotypic abnormalitiesPrognostic factorsCell transplantationMolecular predictorsMRD clearanceRemissionClinical trialsNext-generation sequencingChemotherapyPatientsMutation patternsTherapyLeukemiaMRD
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