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 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 leukemia
2024
Global Disparities in the Characteristics and Outcomes of Leukemia Clinical Trials: A Cross-Sectional Study of the ClinicalTrials.gov Database.
Alhajahjeh A, Rotter L, Stempel J, Grimshaw A, Bewersdorf J, Blaha O, Kewan T, Podoltsev N, Shallis R, Mendez L, Stahl M, Zeidan A. Global Disparities in the Characteristics and Outcomes of Leukemia Clinical Trials: A Cross-Sectional Study of the ClinicalTrials.gov Database. JCO Global Oncology 2024, 10: e2400316. PMID: 39621951, DOI: 10.1200/go-24-00316.Peer-Reviewed Original ResearchHMA/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 settingsRisk of bleeding in patients with essential thrombocythemia and extreme thrombocytosis
Venkat R, Redd R, Harris A, Aryee M, Marneth A, Kamaz B, Kim C, Wazir M, Weeks L, Stahl M, DeAngelo D, Lindsley R, Luskin M, Hobbs G, How J. Risk of bleeding in patients with essential thrombocythemia and extreme thrombocytosis. Blood Advances 2024, 8: 6043-6054. PMID: 39293089, PMCID: PMC11635702, DOI: 10.1182/bloodadvances.2024013777.Peer-Reviewed Original ResearchConceptsRisk of bleedingClinically relevant nonmajor bleedingEssential thrombocythemiaBleeding riskPlatelet countCumulative incidenceDana-Farber Cancer Institute and Massachusetts General HospitalAssociated with acquired von Willebrand syndromeCumulative incidence of thrombosisCumulative incidence of bleedingIncreased bleeding riskIncidence of bleedingReduced bleeding riskVon Willebrand syndromeIncidence of thrombosisNonmajor bleedingDNMT3A mutationsMassachusetts General HospitalThrombotic eventsDana-FarberDiabetes mellitusBleedingPatientsRisk factorsTreatment decisionsSubunit-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 ResearchConceptsMissense 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 StatementsConceptsInternational 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 revisionE7820, an anti-cancer sulfonamide, degrades RBM39 in patients with splicing factor mutant myeloid malignancies: a phase II clinical trial
Bewersdorf J, Stahl M, Taylor J, Mi X, Chandhok N, Watts J, Derkach A, Wysocki M, Lu S, Bourcier J, Hogg S, Rahman J, Chaudhry S, Totiger T, Abdel-Wahab O, Stein E. E7820, an anti-cancer sulfonamide, degrades RBM39 in patients with splicing factor mutant myeloid malignancies: a phase II clinical trial. Leukemia 2023, 37: 2512-2516. PMID: 37814121, PMCID: PMC10681888, DOI: 10.1038/s41375-023-02050-4.Peer-Reviewed Original ResearchFLT3-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 StatementsConceptsAcute 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 pathwayTreatmentApoptosisClassification, risk stratification and response assessment in myelodysplastic syndromes/neoplasms (MDS): A state-of-the-art report on behalf of the International Consortium for MDS (icMDS)
Stahl M, Bewersdorf J, Xie Z, Porta M, Komrokji R, Xu M, Abdel-Wahab O, Taylor J, Steensma D, Starczynowski D, Sekeres M, Sanz G, Sallman D, Roboz G, Platzbecker U, Patnaik M, Padron E, Odenike O, Nimer S, Nazha A, Majeti R, Loghavi S, Little R, List A, Kim T, Hourigan C, Hasserjian R, Halene S, Griffiths E, Gore S, Greenberg P, Figueroa M, Fenaux P, Efficace F, DeZern A, Daver N, Churpek J, Carraway H, Buckstein R, Brunner A, Boultwood J, Borate U, Bejar R, Bennett J, Wei A, Santini V, Savona M, Zeidan A. Classification, risk stratification and response assessment in myelodysplastic syndromes/neoplasms (MDS): A state-of-the-art report on behalf of the International Consortium for MDS (icMDS). Blood Reviews 2023, 62: 101128. PMID: 37704469, DOI: 10.1016/j.blre.2023.101128.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsInternational consensus classificationResponse assessmentWorld Health Organization classificationPatient-centered careRisk assessment toolClinical outcomesRisk stratificationOrganization classificationCentered careTherapeutic benefitTherapeutic outcomesConsensus classificationResponse criteriaInternational ConsortiumNeoplasmsLife assessmentAssessment toolOutcomesReportAssessmentPrognosticationCareSafety 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 ResearchConceptsAcute 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 ResearchConceptsAcute 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 StatementsConceptsMyelodysplastic 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 severityCurrent landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS)
Bewersdorf J, Xie Z, Bejar R, Borate U, Boultwood J, Brunner A, Buckstein R, Carraway H, Churpek J, Daver N, Porta M, DeZern A, Fenaux P, Figueroa M, Gore S, Griffiths E, Halene S, Hasserjian R, Hourigan C, Kim T, Komrokji R, Kuchroo V, List A, Loghavi S, Majeti R, Odenike O, Patnaik M, Platzbecker U, Roboz G, Sallman D, Santini V, Sanz G, Sekeres M, Stahl M, Starczynowski D, Steensma D, Taylor J, Abdel-Wahab O, Xu M, Savona M, Wei A, Zeidan A. Current landscape of translational and clinical research in myelodysplastic syndromes/neoplasms (MDS): Proceedings from the 1st International Workshop on MDS (iwMDS) Of the International Consortium for MDS (icMDS). Blood Reviews 2023, 60: 101072. PMID: 36934059, DOI: 10.1016/j.blre.2023.101072.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsImmune checkpoint inhibitorsSpecific molecular alterationsNovel animal modelInnate immune systemCheckpoint inhibitorsImmune dysregulationMDS patientsClinical trialsNovel therapiesTherapeutic strategiesAnimal modelsGermline predispositionImmune systemMolecular alterationsClinical researchInternational ConsortiumNeoplasmsClinical workGenetic landscapeInternational WorkshopPatientsCurrent landscapePathogenesisTherapyDisease
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