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 evasion
2024
A 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 inhibitorsThe 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 featuresTP53 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 survivalPrognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases
Badar T, Nanaa A, Atallah E, Shallis R, Craver E, Li Z, Goldberg A, Saliba A, Patel A, Bewersdorf J, Duvall A, Burkart M, Bradshaw D, Abaza Y, Stahl M, Palmisiano N, Murthy S, Zeidan A, Kota V, Patnaik M, Litzow M. Prognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases. Haematologica 2024, 109: 3533-3542. PMID: 38813716, PMCID: PMC11532685, DOI: 10.3324/haematol.2024.285000.Peer-Reviewed Original ResearchAcute myeloid leukemiaMyelodysplastic syndromeComplex cytogeneticsMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationLower-risk myelodysplastic syndromesHematopoietic stem cell transplantationHigher-risk myelodysplastic syndromesOutcomes of SHStem cell transplantationAllo-HCTTP53 alterationsPrognostic impactMyeloid malignanciesTP53 mutationsCell transplantationFLT3-ITDIDH1 mutationMultivariate analysisSupportive careUS academic institutionsNeoplastic diseasePatientsSuperior EFSPredicting outcomePhase 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
Intensive Induction Chemotherapy Vs Hypomethylating Agents + Venetoclax (HMA/VEN) in NPM1-Mutant Newly Diagnosed Acute Myeloid Leukemia (AML) - a Multicenter Cohort Study
Bewersdorf J, Shimony S, Shallis R, Liu Y, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Lindsley R, Chen E, Ramos J, Stein A, DeAngelo D, Neuberg D, Stone R, Ball B, Stahl M. Intensive Induction Chemotherapy Vs Hypomethylating Agents + Venetoclax (HMA/VEN) in NPM1-Mutant Newly Diagnosed Acute Myeloid Leukemia (AML) - a Multicenter Cohort Study. Blood 2023, 142: 2964. DOI: 10.1182/blood-2023-174285.Peer-Reviewed Original ResearchNPM1 mutant acute myeloid leukemiaIntensive induction chemotherapyAcute myeloid leukemiaComposite complete responseMedian overall survivalOverall survivalComplete responseAllo-SCTPt ageAbnormal cytogeneticsCohort studyMyelodysplastic syndromePolymerase chain reactionClinical trialsMyeloid leukemiaNPM1 mutationsLarge multicenter retrospective cohort studyTime-varying covariatesMulticenter retrospective cohort studyAllogeneic stem cell transplantationPrior myelodysplastic syndromeMulticenter cohort studyRetrospective cohort studyStem cell transplantationPrior chemotherapy exposureSafety and Preliminary Efficacy of DFV890 in Adult Patients with Myeloid Diseases: A Phase 1b Study
Garcia-Manero G, Ooi M, Lao Z, Gill H, Abaza Y, Stahl M, Haque T, DeZern A, Greenberg P, Pelletier M, Singh A, Carreon D, Beaulieu V, Woo J. Safety and Preliminary Efficacy of DFV890 in Adult Patients with Myeloid Diseases: A Phase 1b Study. Blood 2023, 142: 3250. DOI: 10.1182/blood-2023-174642.Peer-Reviewed Original ResearchChronic myelomonocytic leukemiaCMML-specific prognostic scoring systemErythropoiesis-stimulating agentsDose-expansion partPrognostic Scoring SystemMyelodysplastic syndromeHypomethylating agentsLR-MDSEastern Cooperative Oncology Group performance statusIntermediate risk myelodysplastic syndromePredictive markers of efficacyDose of study treatmentInternational Prognostic Scoring SystemIL-18Colony-stimulating growth factorScoring systemAllogeneic hematopoietic transplantationBone marrow aspirate/biopsyPhase 1b studyProgression-free survivalTime to progressionPotential curative optionRisk myelodysplastic syndromesDuration of responseBone marrow assessmentSubunit-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 C, Luskin M, Stahl M, Tothova Z. Subunit-Specific Analysis of Cohesin-Mutant Myeloid Malignancies Reveals Distinct Ontogeny and Outcomes. Blood 2023, 142: 999. DOI: 10.1182/blood-2023-187519.Peer-Reviewed Original ResearchAcute myeloid leukemiaDana-Farber Cancer InstituteProgression free survivalMyelodysplasia related changesMyelodysplastic syndromeOverall survivalTP53 mutationsSTAG2 mutationsRAD21 mutationsMDS/myeloproliferative neoplasmPrognostic impactClinical outcomesDana-Farber Cancer Institute cohortPrognostic impact of TP53 mutationsFollow-upDe novo acute myeloid leukemiaImpact of TP53 mutationsAllogeneic stem cell transplantationCohesin mutationsSecondary acute myeloid leukemiaRisk of leukemic transformationSubtype of myelodysplastic syndromeAnalysis of overall survivalAssociated with myelodysplastic syndromeAML-free survivalMyeloid Neoplasia in Patients with Hereditary Hemoglobinopathies and Hemolytic Anemias (HHAs)
Osei M, Merz L, Chien K, Hammond D, Lindsley C, Luskin M, Stahl M, How J, Wazir M, Marneth A, Shimony S, Mullally A, DeAngelo D, Garcia-Manero G, Achebe M, Ebert B, Weeks L. Myeloid Neoplasia in Patients with Hereditary Hemoglobinopathies and Hemolytic Anemias (HHAs). Blood 2023, 142: 2427. DOI: 10.1182/blood-2023-172992.Peer-Reviewed Original ResearchAcute myeloid leukemiaSickle cell diseaseChronic myelomonocytic leukemiaMyeloid neoplasiaHigh-risk diseasePh+ CMLPh-MPNsSickle cell traitHereditary spherocytosisThalassemia traitMyelodysplastic syndromeMN diagnosisMN patientsHemolytic anemiaELN criteriaHereditary hemoglobinopathiesPh+ chronic myelogenous leukemiaVery high riskCases of acute myeloid leukemiaPh-negative myeloproliferative neoplasmsCell diseaseTP53-mutant acute myeloid leukemiaHigh riskAcute myeloid leukemia casesDiagnosis of myeloid malignancyEpidemiology 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 significancePathophysiologyWhy do we not have more drugs approved for MDS? A critical viewpoint on novel drug development in MDS
Frumm S, Shimony S, Stone R, DeAngelo D, Bewersdorf J, Zeidan A, Stahl M. Why do we not have more drugs approved for MDS? A critical viewpoint on novel drug development in MDS. Blood Reviews 2023, 60: 101056. PMID: 36805300, DOI: 10.1016/j.blre.2023.101056.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromeDNA methyltransferase inhibitorLow risk (LR) MDSHigh-risk myelodysplastic syndromeHigh transfusion needsRisk myelodysplastic syndromesCurrent treatment landscapeErythropoiesis-stimulating agentsNovel drug developmentHR-MDSTreatment landscapeTransfusion needsTargetable mutationsClinical trialsMDS pathogenesisNew agentsRing sideroblastsMore drugsUnmet needTherapy developmentDrug developmentMethyltransferase inhibitorFactor mutationsApprovalInflammation
2022
International Working Group (IWG) Response Criteria and Association with Overall Survival in Patients with Myelodysplastic Syndromes Treated with Hypomethylating Agents - a Systematic Review and Meta-Analysis
Bewersdorf J, Grimshaw A, Platzbecker U, Fenaux P, Sekeres M, Zeidan A, Stahl M. International Working Group (IWG) Response Criteria and Association with Overall Survival in Patients with Myelodysplastic Syndromes Treated with Hypomethylating Agents - a Systematic Review and Meta-Analysis. Blood 2022, 140: 9811-9812. DOI: 10.1182/blood-2022-158622.Peer-Reviewed Original ResearchPrognostication in myelodysplastic syndromes (neoplasms): Molecular risk stratification finally coming of age
Xie Z, Chen E, Stahl M, Zeidan A. Prognostication in myelodysplastic syndromes (neoplasms): Molecular risk stratification finally coming of age. Blood Reviews 2022, 59: 101033. PMID: 36357283, DOI: 10.1016/j.blre.2022.101033.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromePrognostic modelManagement of MDSInternational Prognostic Scoring SystemMolecular prognostic modelPrognostic scoring systemMolecular risk stratificationHeterogeneous clinical outcomesBone marrow failure disordersMarrow failure disordersRevised IPSSClinical outcomesRisk prognosticationRisk stratificationTraditional risk modelsScoring systemMDS pathobiology
2021
Venetoclax combination therapy in acute myeloid leukemia and myelodysplastic syndromes
Shimony S, Stone R, Stahl M. Venetoclax combination therapy in acute myeloid leukemia and myelodysplastic syndromes. Current Opinion In Hematology 2021, 29: 63-73. PMID: 34966123, DOI: 10.1097/moh.0000000000000698.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaVenetoclax-based combination therapiesCombination therapyMyelodysplastic syndromeMyeloid leukemiaNewly diagnosed acute myeloid leukemiaAcute myeloid leukemia patientsVenetoclax combination therapyDeep molecular responseIncomplete count recoveryLow dose cytarabineBcl-2 inhibitorsHigh-risk populationRelapsed myelodysplastic syndromeProlonged cytopeniasDose cytarabineCount recoveryHypomethylating agentsRemission rateTargeted therapyTherapyBcl-2Therapeutic possibilitiesLong-term effectsPatient outcomesClinical Predictors of Outcome in Adult Patients with Acute Leukemias and Myelodysplastic Syndrome and COVID-19 Infection: Report from the American Society of Hematology Research Collaborative (ASH RC) Data Hub
Desai P, Goldberg A, Anderson K, Narendra V, Neuberg D, Radhakrishnan V, Redd R, Roboz G, Sehn L, Sekeres M, Stahl M, Tallman M, Thompson J, Tucker E, Wood W, Hicks L. Clinical Predictors of Outcome in Adult Patients with Acute Leukemias and Myelodysplastic Syndrome and COVID-19 Infection: Report from the American Society of Hematology Research Collaborative (ASH RC) Data Hub. Blood 2021, 138: 280. PMCID: PMC8701680, DOI: 10.1182/blood-2021-146016.Peer-Reviewed Original ResearchAcute myeloid leukemiaAcute lymphocytic leukemiaEntity's Board of DirectorsMyelodysplastic syndromeTime of COVID-19 diagnosisCurrent equity holderActive diseaseBoard of directorsCOVID-19 severityCOVID-19 infectionCOVID-19 registryICU careCOVID-19 diagnosisHematologic malignanciesBristol Myers SquibbPublicly-traded companiesResponse groupMultivariate analysisPredictors of severe infectionTypes of hematological malignanciesHospitalized patientsAssociated with advanced ageAssociated with neutropeniaAssociated with increased mortalitySevere COVID-19 infectionHypomethylating Agents and FLT3 Inhibitors As Maintenance Treatment for Acute Myeloid Leukemia and Myelodysplastic Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation–A Systematic Review and Meta-Analysis
Bewersdorf JP, Allen C, Mirza AS, Grimshaw AA, Giri S, Podoltsev NA, Gowda L, Cho C, Tallman MS, Zeidan AM, Stahl M. Hypomethylating Agents and FLT3 Inhibitors As Maintenance Treatment for Acute Myeloid Leukemia and Myelodysplastic Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation–A Systematic Review and Meta-Analysis. Transplantation And Cellular Therapy 2021, 27: 997.e1-997.e11. PMID: 34551341, PMCID: PMC9533376, DOI: 10.1016/j.jtct.2021.09.005.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaRelapse-free survivalMaintenance therapyMyelodysplastic syndromeFLT3 inhibitorsAllo-HCTHazard ratioMaintenance treatmentClinical trialsMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationControl groupSystematic reviewAllogeneic hematopoietic cell transplantMeasurable residual disease statusHematopoietic stem cell transplantationMeasurable residual disease testingNon-relapse mortalityHematopoietic cell transplantPre-emptive treatmentResidual disease statusStem cell transplantationFavorable safety profileSame patient cohortRandom-effects model
2020
Safety and Efficacy of Maintenance Treatment Following Allogeneic Hematopoietic Cell Transplant in Acute Myeloid Leukemia and Myelodysplastic Syndrome - a Systematic Review and Meta-Analysis
Bewersdorf J, Tallman M, Cho C, Zeidan A, Stahl M. Safety and Efficacy of Maintenance Treatment Following Allogeneic Hematopoietic Cell Transplant in Acute Myeloid Leukemia and Myelodysplastic Syndrome - a Systematic Review and Meta-Analysis. Blood 2020, 136: 34-35. DOI: 10.1182/blood-2020-136671.Peer-Reviewed Original ResearchAcute myeloid leukemiaRelapse-free survivalAllogeneic hematopoietic cell transplantHematopoietic cell transplantFLT3 inhibitorsMyelodysplastic syndromeAllo-HCTOverall survivalRFS ratesChronic GVHDMaintenance therapyCell transplantPatient selectionClinical trialsMyeloid leukemiaADC therapeuticsSystematic reviewMinimal residual disease testingAdvisory CommitteeDaiichi SankyoHigh-risk geneticsRate of GVHDPre-emptive treatmentCareful patient selectionPrognosis of patients
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