2025
Asciminib 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
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 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-FarberOntogenyMolecular 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
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)
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 transplant
2021
Multicenter evaluation of efficacy and toxicity of venetoclax‐based combinations in patients with accelerated and blast phase myeloproliferative neoplasms
King A, Weis T, Derkach A, Ball S, Pandey M, Mauro M, Goldberg A, Stahl M, Famulare C, Tallman M, Wang E, Kuykendall A, Rampal R. Multicenter evaluation of efficacy and toxicity of venetoclax‐based combinations in patients with accelerated and blast phase myeloproliferative neoplasms. American Journal Of Hematology 2021, 97: e7-e10. PMID: 34674293, DOI: 10.1002/ajh.26381.Peer-Reviewed Original ResearchPlasmacytoid dendritic cell expansion defines a distinct subset of RUNX1-mutated acute myeloid leukemia
Xiao W, Chan A, Waarts M, Mishra T, Liu Y, Cai S, Yao J, Gao Q, Bowman R, Koche R, Csete I, DelGaudio N, Derkach A, Baik J, Yanis S, Famulare C, Patel M, Arcila M, Stahl M, Rampal R, Tallman M, Zhang Y, Dogan A, Goldberg A, Roshal M, Levine R. Plasmacytoid dendritic cell expansion defines a distinct subset of RUNX1-mutated acute myeloid leukemia. Blood 2021, 137: 1377-1391. PMID: 32871587, PMCID: PMC7955409, DOI: 10.1182/blood.2020007897.Peer-Reviewed Original ResearchConceptsBlastic plasmacytoid dendritic cell neoplasmPlasmacytoid dendritic cellsAcute myeloid leukemiaPDC expansionPDC-AMLRUNX1-mutated acute myeloid leukemiaRUNX1 mutationsLeukemic blastsDendritic cellsMyeloid leukemiaCross-lineage antigen expressionPlasmacytoid dendritic cell differentiationPlasmacytoid dendritic cell neoplasmPatient-derived xenograft modelsAcute myeloid leukemia casesMature plasmacytoid dendritic cellsPlasmacytoid dendritic cell precursorsDendritic cell expansionChronic myelomonocytic leukemiaDendritic cell neoplasmPotential treatment approachTranscriptional programsCD123 targetingLeukemic burdenCell neoplasms
2020
Phase 1 dose escalation trial of volasertib in combination with decitabine in patients with acute myeloid leukemia
Cortes J, Podoltsev N, Kantarjian H, Borthakur G, Zeidan AM, Stahl M, Taube T, Fagan N, Rajeswari S, Uy GL. Phase 1 dose escalation trial of volasertib in combination with decitabine in patients with acute myeloid leukemia. International Journal Of Hematology 2020, 113: 92-99. PMID: 32951163, DOI: 10.1007/s12185-020-02994-8.Peer-Reviewed Original ResearchMeSH KeywordsAgedAntineoplastic Combined Chemotherapy ProtocolsCell Cycle ProteinsDecitabineDose-Response Relationship, DrugFebrile NeutropeniaFeeding and Eating DisordersFemaleGene ExpressionHumansLeukemia, Myeloid, AcuteMaleMolecular Targeted TherapyProtein Serine-Threonine KinasesProto-Oncogene ProteinsPteridinesTreatment OutcomeConceptsAcute myeloid leukemiaMyeloid leukemiaCommon treatment-emergent adverse eventsPhase 1 dose-escalation trialTreatment-emergent adverse eventsMTD of volasertibObjective response rateAdverse event profileDose-escalation trialPhase 1 trialAnti-leukemic activityPolo-like kinase 1Febrile neutropeniaEscalation trialAdverse eventsCell cycle kinase inhibitorsAML patientsEvent profilePoor prognosisResponse ratePatientsVolasertibDecitabineKinase inhibitorsNumerous cancers
2019
Association of provider experience and clinical outcomes in patients with myelodysplastic syndromes receiving hypomethylating agents
Zeidan AM, Hu X, Zhu W, Stahl M, Wang R, Huntington SF, Giri S, Bewersdorf JP, Podoltsev NA, Gore SD, Ma X, Davidoff AJ. Association of provider experience and clinical outcomes in patients with myelodysplastic syndromes receiving hypomethylating agents. Leukemia & Lymphoma 2019, 61: 397-408. PMID: 31570040, PMCID: PMC7732188, DOI: 10.1080/10428194.2019.1663423.Peer-Reviewed Original ResearchConceptsMyelodysplastic syndromeOverall survivalHMA cyclesHMA therapyProvider experienceCox proportional hazards modelOlder MDS patientsMedian overall survivalPercent of patientsSEER-Medicare dataMultivariate logistic regressionProportional hazards modelHMA initiationClinical outcomesMDS patientsClinical trialsMedian numberHMA treatmentHazards modelPatientsSignificant associationLogistic regressionPopulation-level survivalSurvivalSyndromeRBC transfusion independence among lower risk MDS patients receiving hypomethylating agents: a population-level analysis
Zeidan AM, Zhu W, Stahl M, Wang R, Huntington SF, Giri S, Podoltsev NA, Gore SD, Ma X, Davidoff AJ. RBC transfusion independence among lower risk MDS patients receiving hypomethylating agents: a population-level analysis. Leukemia & Lymphoma 2019, 60: 3181-3187. PMID: 31170846, DOI: 10.1080/10428194.2019.1622700.Peer-Reviewed Original ResearchConceptsRBC transfusion independenceLR-MDS patientsTransfusion independenceHMA initiationRBC transfusionClinical effectivenessReal-life clinical effectivenessRed blood cell transfusionLower-risk myelodysplastic syndromesLow-risk MDS patientsRisk MDS patientsBlood cell transfusionRisk myelodysplastic syndromesHMA therapyLR-MDSCell transfusionMost patientsDisease courseMDS patientsMedicare databaseMyelodysplastic syndromePopulation-level estimatesLower oddsTransfusionPatients
2016
Selecting initial treatment of acute myeloid leukaemia in older adults
Podoltsev NA, Stahl M, Zeidan AM, Gore SD. Selecting initial treatment of acute myeloid leukaemia in older adults. Blood Reviews 2016, 31: 43-62. PMID: 27745715, DOI: 10.1016/j.blre.2016.09.005.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsAcute myeloid leukemiaTreatment-related mortalityInduction chemotherapyComplete remissionInitial treatmentMyeloid leukemiaNovel agentsOlder adultsMedian overall survivalGroup of patientsPhase III developmentDisease-related characteristicsMechanism of actionCPX-351Overall survivalLow-intensity treatmentClinical trialsTreatment outcomesPatientsRemissionChemotherapyLeukemiaMortalityTreatmentAdultsClinical response to ruxolitinib in CSF3R T618-mutated chronic neutrophilic leukemia
Stahl M, Xu ML, Steensma DP, Rampal R, Much M, Zeidan AM. Clinical response to ruxolitinib in CSF3R T618-mutated chronic neutrophilic leukemia. Annals Of Hematology 2016, 95: 1197-1200. PMID: 27068405, PMCID: PMC7479634, DOI: 10.1007/s00277-016-2664-4.Peer-Reviewed Original Research
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