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 groupVenetoclax
2022
Synthetic introns enable splicing factor mutation-dependent targeting of cancer cells
North K, Benbarche S, Liu B, Pangallo J, Chen S, Stahl M, Bewersdorf J, Stanley R, Erickson C, Cho H, Pineda J, Thomas J, Polaski J, Belleville A, Gabel A, Udy D, Humbert O, Kiem H, Abdel-Wahab O, Bradley R. Synthetic introns enable splicing factor mutation-dependent targeting of cancer cells. Nature Biotechnology 2022, 40: 1103-1113. PMID: 35241838, PMCID: PMC9288984, DOI: 10.1038/s41587-022-01224-2.Peer-Reviewed Original ResearchConceptsBreast cancerExpression of herpes simplex virus thymidine kinaseHerpes simplex virus thymidine kinaseCancer cellsPancreatic cancer cells in vitroWild-type cellsCancer cells in vitroCancer gene therapyTargeting of cancer cellsTumor-specific changesUveal melanoma cellsTreatment in vivoSynthetic intronChange-of-function mutationsCells in vitroUveal melanomaSF3B1 mutationsHSV-tkGene therapyTumor cellsIsogenic wild-type cellsMelanoma cellsRNA splicing factorsCancerHost survival
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
One plus one does not always equal two, especially with regard to hypomethylating agents: the question of synergy of azacitidine and lenalidomide for treatment of relapsed acute myeloid leukemia and myelodysplastic syndromes post allogeneic hematopoietic stem cell transplant
Bewersdorf JP, Stahl M, Zeidan AM. One plus one does not always equal two, especially with regard to hypomethylating agents: the question of synergy of azacitidine and lenalidomide for treatment of relapsed acute myeloid leukemia and myelodysplastic syndromes post allogeneic hematopoietic stem cell transplant. Expert Review Of Hematology 2019, 12: 575-578. PMID: 31225770, DOI: 10.1080/17474086.2019.1635005.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAntigens, CDAzacitidineCD8-Positive T-LymphocytesClinical Trials as TopicDrug Therapy, CombinationFemaleHematopoietic Stem Cell TransplantationHumansLenalidomideLeukemia, Myeloid, AcuteLymphocyte Activation Gene 3 ProteinMaleMiddle AgedMyelodysplastic SyndromesProgrammed Cell Death 1 ReceptorRecurrenceTransplantation, HomologousConceptsAcute myeloid leukemiaHematopoietic stem cell transplantStem cell transplantMyelodysplastic syndromeMyeloid leukemiaAllo-HSCTCell transplantAllogeneic hematopoietic stem cell transplantResponse rateAllogeneic stem cell transplantationNovel salvage therapiesPhase Ib studyStem cell transplantationOverall response rateCycles of treatmentCases of graftHigh response rateMechanism of actionCombination lenalidomideSalvage therapyHost diseaseMost patientsCurative treatmentCombination therapyDisease stageRBC 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
Single agent blinatumumab as frontline therapy for an 85-year-old patient with B cell precursor acute lymphoblastic leukemia
Kim TK, Xu ML, Podoltsev NA, Prebet T, Barbarotta L, Amin K, Kasberg S, Roche K, Stahl M, Gore SD, Zeidan AM. Single agent blinatumumab as frontline therapy for an 85-year-old patient with B cell precursor acute lymphoblastic leukemia. Annals Of Hematology 2016, 95: 1895-1898. PMID: 27468851, PMCID: PMC10955604, DOI: 10.1007/s00277-016-2761-4.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAged, 80 and overAntibodies, BispecificAntineoplastic AgentsAntineoplastic Combined Chemotherapy ProtocolsBone MarrowBreast NeoplasmsCarcinoma, Transitional CellCytarabineFemaleHumansLymphoma, B-CellMercaptopurineMethotrexateNeoplasms, Second PrimaryPrecursor B-Cell Lymphoblastic Leukemia-LymphomaPrednisoneRemission InductionSalvage TherapyUrinary Bladder NeoplasmsVincristine
2011
Divergent CFTR orthologs respond differently to the channel inhibitors CFTRinh-172, glibenclamide, and GlyH-101
Stahl M, Stahl K, Brubacher MB, Forrest JN. Divergent CFTR orthologs respond differently to the channel inhibitors CFTRinh-172, glibenclamide, and GlyH-101. American Journal Of Physiology - Cell Physiology 2011, 302: c67-c76. PMID: 21940661, PMCID: PMC3328903, DOI: 10.1152/ajpcell.00225.2011.Peer-Reviewed Original ResearchConceptsCystic fibrosis transmembrane conductance regulatorGlyH-101Expression studiesChannel proteinsCFTR chloride channel proteinFibrosis transmembrane conductance regulatorChloride channel proteinSite-directed mutagenesisShark rectal glandAmino acid residuesTransmembrane conductance regulatorDifferent orthologsSingle amino acidPrevious site-directed mutagenesisOocyte expression studiesThree-dimensional structureRegulatory regionsXenopus laevis oocytesCFTR proteinAdditional residuesConductance regulatorOrthologsAcid residuesRectal glandSpecific inhibitor
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