2025
Hypomethylating Agent and Venetoclax Combination Is a Safe and Effective Alternative to Intensive Chemotherapy in Older (≥ 70 Years) Patients With Newly Diagnosed Favorable Risk Acute Myeloid Leukemia
Ball S, Jain A, Al Ali N, Aguirre L, Bewersdorf J, Kent A, Rose A, Hayden A, Siddon A, Lykon J, Madarang E, Swoboda D, Padron E, Sweet K, Sallman D, Lancet J, Carraway H, Watts J, Zeidan A, Pollyea D, Komrokji R. Hypomethylating Agent and Venetoclax Combination Is a Safe and Effective Alternative to Intensive Chemotherapy in Older (≥ 70 Years) Patients With Newly Diagnosed Favorable Risk Acute Myeloid Leukemia. American Journal Of Hematology 2025 PMID: 40772639, DOI: 10.1002/ajh.70031.Peer-Reviewed Original ResearchIntegrated Immune Landscape Analysis of RNA Splicing Factor-Mutant AML and Higher risk MDS Treated with Azacitidine ± 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. Integrated Immune Landscape Analysis of RNA Splicing Factor-Mutant AML and Higher risk MDS Treated with Azacitidine ± Durvalumab. Therapeutic Advances In Hematology 2025, 16: 20406207251347344. PMID: 40546817, PMCID: PMC12182606, DOI: 10.1177/20406207251347344.Peer-Reviewed Original ResearchThis study investigates the immune landscape of RNA splicing factor-mutant AML and MDS, showing no survival benefit from adding durvalumab to azacitidine treatment.Real-World Treatment Patterns, Clinical Outcomes, and Costs in Patients with Higher-Risk Myelodysplastic Syndromes Across France, Germany, and the United Kingdom
Drummond M, Finelli C, Kristo F, Kelkar S, Corman S, Raina R, Ashaye A, Dalal M, Haase D. Real-World Treatment Patterns, Clinical Outcomes, and Costs in Patients with Higher-Risk Myelodysplastic Syndromes Across France, Germany, and the United Kingdom. Journal Of Blood Medicine 2025, 16: 307-319. PMID: 40585910, PMCID: PMC12206415, DOI: 10.2147/jbm.s516558.Peer-Reviewed Original ResearchHigher-risk myelodysplastic syndromesReal-world treatment patternsTreatment patternsMyelodysplastic syndromeClinical outcomesProgression to acute myeloid leukemiaProgression-free survivalMedian patient ageMedian Follow-UpHigh-risk diseaseFirst-line treatmentKaplan-Meier methodAcute myeloid leukemiaHealthcare resource useFollow-up post-diagnosisIPSS-R.Partial remissionTransfusion-DependentOverall survivalPatient ageMyeloid leukemiaChart reviewClinical characteristicsAdjunctive therapyAdult patientsThe efficacy of METTL3 inhibition in pre-clinical models of MDS and AML.
Kenworthy C, Wei M, VanOudenhove J, Busarello E, Ramirez Amarilla C, Paul S, Cruz J, Baassiri A, Maul-Newby H, Biancon G, Tebaldi T, Halene S. The efficacy of METTL3 inhibition in pre-clinical models of MDS and AML. Journal Of Clinical Oncology 2025, 43 DOI: 10.1200/jco.2025.43.16_suppl.e18584.Peer-Reviewed Original ResearchAcute myeloid leukemiaMyelodysplastic syndromeInnate immune responseImmune signaturesTransplantation modelModel of myelodysplastic syndromeMurine models of leukemiaPreclinical anti-tumour efficacyImmune responseInnate immune cell subsetsSolid tumor clinical trialsSplicing factorsCompetitive transplant modelImmune checkpoint inhibitionMouse solid tumor modelsDouble-stranded RNAMyelodysplastic syndrome patientsInnate immune signaturesAnti-tumor efficacyImmune cell subsetsMyelodysplastic syndromes pathogenesisDriver genesSolid tumor modelsPre-clinical modelsModels of leukemiaMore than just another IDH inhibitor: Insights from the HMPL-306 phase 1 trial
Getz T, Bewersdorf J. More than just another IDH inhibitor: Insights from the HMPL-306 phase 1 trial. Med 2025, 6: 100600. PMID: 40516517, DOI: 10.1016/j.medj.2025.100600.Peer-Reviewed Original ResearchAn all-oral regimen of decitabine-cedazuridine (DEC-C) plus venetoclax (VEN) in patients (pts) with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive induction chemotherapy: Results from a phase 2 cohort of 101 pts.
Zeidan A, Griffiths E, Dinardo C, Mannis G, Montesinos P, Arnan M, Savona M, Odenike O, McCloskey J, Amin H, Fathi A, Bernal del Castillo T, Rodriguez-Macias G, Liesveld J, Im A, Oganesian A, Xu Q, Dijkstra M, Keer H, Roboz G. An all-oral regimen of decitabine-cedazuridine (DEC-C) plus venetoclax (VEN) in patients (pts) with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive induction chemotherapy: Results from a phase 2 cohort of 101 pts. Journal Of Clinical Oncology 2025, 43: 6504-6504. DOI: 10.1200/jco.2025.43.16_suppl.6504.Peer-Reviewed Original ResearchAcute myeloid leukemiaAll-oral regimenInduction chemotherapyEuropean LeukemiaNetCR rateMedian time to CRTreatment-emergent adverse eventsBCL-2 inhibitor venetoclaxDiagnosed AMLDiagnosed acute myeloid leukemiaMedian CR durationPhase 2 partAll-oral regimensIntensive induction chemotherapyPhase 1/2 trialBone marrow examinationMedian Follow-UpTime to CRConfidence intervalsDrug-drug interactionsPhase 2 cohortIntravenous decitabineMedian OSCR durationFebrile neutropeniaOverall survival (OS) and duration of response for transfusion independence (TI) in erythropoiesis stimulating agent (ESA)–naive patients (pts) with very low-, low-, or intermediate-risk myelodysplastic syndromes (MDS) treated with luspatercept (LUSPA) vs epoetin alfa (EA) in the COMMANDS trial.
Garcia-Manero G, Della Porta M, Zeidan A, Komrokji R, Pozharskaya V, Rose S, Keeperman K, Lai Y, Kalsekar S, Aggarwal B, Miteva D, Valcárcel D, Fenaux P, Shortt J, Platzbecker U, Santini V. Overall survival (OS) and duration of response for transfusion independence (TI) in erythropoiesis stimulating agent (ESA)–naive patients (pts) with very low-, low-, or intermediate-risk myelodysplastic syndromes (MDS) treated with luspatercept (LUSPA) vs epoetin alfa (EA) in the COMMANDS trial. Journal Of Clinical Oncology 2025, 43: 6512-6512. DOI: 10.1200/jco.2025.43.16_suppl.6512.Peer-Reviewed Original ResearchDuration of responseOverall survivalRBC-TIMyelodysplastic syndromeEpoetin alfaOS ratesTransfusion independenceTransfusion-DependentOS trendProgression to acute myeloid leukemiaIntermediate-risk myelodysplastic syndromesLower-risk myelodysplastic syndromesAcute myeloid leukemiaMedian cumulative durationStandard of careLuspatercept groupMedian OSDose escalationMedian followEligible ptsLR-MDSMedian durationSecondary endpointsMyeloid leukemiaClinical benefitQuANTUM-Wild: A phase 3, randomized, double-blind, placebo-controlled trial of quizartinib in combination with chemotherapy and as single-agent maintenance in FLT3 -ITD–negative acute myeloid leukemia (AML).
Montesinos P, Cheong J, Daver N, Fathi A, Levis M, Luger S, Miyamoto T, Oliva E, Perl A, Recher C, Schlenk R, Wang J, Zeidan A, Liu L, Duong Y, Imadalou K, Alexis K, Nahar A, Burns K, Erba H. QuANTUM-Wild: A phase 3, randomized, double-blind, placebo-controlled trial of quizartinib in combination with chemotherapy and as single-agent maintenance in FLT3 -ITD–negative acute myeloid leukemia (AML). Journal Of Clinical Oncology 2025, 43 DOI: 10.1200/jco.2025.43.16_suppl.tps6580.Peer-Reviewed Original ResearchAcute myeloid leukemiaRelapse-free survivalEvent-free survivalArm AOverall survivalComplete remissionDouble-blindArm CCases of acute myeloid leukemiaAcute myeloid leukemia blastsAcute myeloid leukemia casesFLT3-ITD-negativeHigh-dose cytarabineFLT3 gene mutationsType II FLT3 inhibitorsPlacebo-controlled trialProlonged overall survivalAllele frequency <5%Leukemic cell survivalArm BStandard chemotherapyEligible ptsInduction/consolidation chemotherapyPrimary endpointSecondary endpointsUnveiling the Mysteries of Molecular Testing in AML: A Guide for Oncologists [Podcast]
Zeidan A, Loghavi S. Unveiling the Mysteries of Molecular Testing in AML: A Guide for Oncologists [Podcast]. Blood And Lymphatic Cancer Targets And Therapy 2025, 15: 39-45. PMID: 40575712, PMCID: PMC12198350, DOI: 10.2147/blctt.s543541.Peer-Reviewed Original ResearchNext-generation sequencingAcute myeloid leukemiaSingle-gene PCRMolecular diagnosticsSingle-geneSanger sequencingGene fusionsMolecular testingRNA analysisMD Anderson Cancer CenterCapillary electrophoresisImproving clinical outcomesAML managementMyeloid leukemiaTargeted therapyClinical outcomesSequencePCRCancer CenterMolecular profilingPersonalized therapyAssay selectionClinical importanceTesting modalitiesSangerEfficacy of Total‐Body Irradiation‐based Intensified Myeloablative Regimens for Acute Leukemia—An International Collaborative Study
Arai Y, Brazauskas R, He N, Al‐Homsi A, Chhabra S, Battiwalla M, Yanada M, Steinberg A, Perez M, Hong S, Kanda J, Bashey A, Frangoul H, Badawy S, Verdonck L, Lazarus H, Yared J, Hashem H, Sharma A, Aljurf M, Dias A, Abid M, Wirk B, Freytes C, Zeidan A, Gergis U, Beitinjaneh A, Askar M, Pu J, Lehmann L, Rangarajan H, Wood W, Hashmi S, Yano S, Kako S, Ozawa Y, Doki N, Kanda Y, Fukuda T, Katayama Y, Ichinohe T, Tanaka J, Teshima T, Okamoto S, Atsuta Y, Saber W. Efficacy of Total‐Body Irradiation‐based Intensified Myeloablative Regimens for Acute Leukemia—An International Collaborative Study. EJHaem 2025, 6: e70061. PMID: 40438703, PMCID: PMC12118588, DOI: 10.1002/jha2.70061.Peer-Reviewed Original ResearchTreatment-related mortalityAcute lymphoblastic leukemiaAcute myeloid leukemiaHematopoietic stem cell transplantationOverall survivalConditioning regimensAllogeneic hematopoietic stem cell transplantationMyeloablative allogeneic hematopoietic stem cell transplantationConditioning regimen of cyclophosphamideMyeloablative conditioning regimensRegimen of cyclophosphamideTotal body irradiationStem cell transplantationRisk of relapseMyeloablative regimensCell transplantationAcute leukemiaLymphoblastic leukemiaMyeloid leukemiaAdult patientsCY/TBIClinical trial registrationRegimensInternational collaborative studyRelapseLong-Term Transfusion Independence with Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Lower-Risk Myelodysplastic Syndromes in the COMMANDS Trial
Garcia-Manero G, Santini V, Zeidan A, Komrokji R, Pozharskaya V, Rose S, Keeperman K, Lai Y, Kalsekar S, Aggarwal B, Miteva D, Valcárcel D, Fenaux P, Shortt J, Della Porta M, Platzbecker U. Long-Term Transfusion Independence with Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Lower-Risk Myelodysplastic Syndromes in the COMMANDS Trial. Advances In Therapy 2025, 42: 3576-3589. PMID: 40377899, PMCID: PMC12182481, DOI: 10.1007/s12325-025-03208-5.Peer-Reviewed Original ResearchConceptsLower-risk myelodysplastic syndromesEfficacy of erythropoiesis-stimulating agentsRBC-TITransfusion independenceRate of red blood cellsEpoetin alfaTransfusion-DependentMyelodysplastic syndromeClinical benefitRates of treatment-emergent adverse eventsESA-naiveTreatment-emergent adverse eventsLack of clinical benefitResultsAt data cutoffHigh-risk MDSErythropoiesis-stimulating agentsAcute myeloid leukemiaData cutoffIntroductionThe efficacyMethodsEligible patientsConsent withdrawalMyeloid leukemiaSerum erythropoietinRed blood cellsAdverse eventsNavigating the dynamic landscape of lower-risk MDS: Advances and emerging insights
Mina A, Madanat Y, Abaza Y, Zeidan A. Navigating the dynamic landscape of lower-risk MDS: Advances and emerging insights. Blood Reviews 2025, 101301. PMID: 40450506, DOI: 10.1016/j.blre.2025.101301.Peer-Reviewed Original ResearchAcute myeloid leukemiaLR-MDSErythropoiesis stimulating agentsIncreased risk of transformation to acute myeloid leukemiaMolecular International Prognostic Scoring SystemRisk of transformation to acute myeloid leukemiaTransformation to acute myeloid leukemiaAllogeneic hematopoietic stem cell transplantationInternational Prognostic Scoring SystemHematopoietic stem cell transplantationClonal myeloid malignanciesPrognostic scoring systemStem cell transplantationPredominant clinical manifestationLowered riskClinical trial designIneffective hematopoiesisCurative optionMyeloid malignanciesCell transplantationIndolent natureTransfusion supportImprove patient outcomesMyeloid leukemiaClinical manifestationsSynergistic Activity of Combined FLT3-ITD and MDM2 Inhibition With Quizartinib and Milademetan in FLT3-ITD Mutant/TP53 Wild Type Acute Myeloid Leukemias.
Zhang W, Li L, Muftuoglu M, Basyal M, Togashi N, Iwanaga K, Tanzawa F, Numata M, Bixby D, Erba H, Podoltsev N, Schiller G, Kumar P, Lesegretain A, Isoyama T, Seki T, Daver N, Andreeff M. Synergistic Activity of Combined FLT3-ITD and MDM2 Inhibition With Quizartinib and Milademetan in FLT3-ITD Mutant/TP53 Wild Type Acute Myeloid Leukemias. Clinical Cancer Research 2025, of1-of15. PMID: 40327322, DOI: 10.1158/1078-0432.ccr-24-2764.Peer-Reviewed Original ResearchAcute myeloid leukemiaMurine double minute 2Patient-derived xenograftsPatient-derived xenograft modelsFLT3-ITDMyeloid leukemiaMDM2 inhibitionCell linesInhibition of FLT3-ITDFLT3-mutant acute myeloid leukemiaDose-escalation clinical trialDevelopment of resistant diseaseFLT3 internal tandem duplicationOverexpression of murine double minute 2FLT3-ITD acute myeloid leukaemiaFMS-like tyrosine kinase 3Phase 1 clinical trialIncomplete hematologic recoveryRelapsed/refractory AML patientsTP53 wild typeReduced tumor burdenPreliminary clinical dataAcute myeloid leukemia cell linesMutations of FMS-like tyrosine kinase 3Expression of prosurvivalRandomized phase 2 study to assess the role of single-agent nivolumab to maintain remission in acute myeloid leukemia
Pyzer A, Dillon L, Sharon E, Karrison T, Zha Y, Fulton N, Gui G, Andrew G, Streicher H, Sweet K, Yaghmour G, Liu J, Jonas B, Schimmer A, Grant S, Zeidan A, Hildebrandt G, Lowrey C, Mattison R, Palmisiano N, Salhotra A, Tzachanis D, Baer M, Lin T, Patel P, Chen H, Stadler W, Odenike O, Larson R, Gajewski T, Hourigan C, Stock W, Liu H. Randomized phase 2 study to assess the role of single-agent nivolumab to maintain remission in acute myeloid leukemia. Blood Advances 2025, 9: 2144-2152. PMID: 39928953, PMCID: PMC12051614, DOI: 10.1182/bloodadvances.2024015176.Peer-Reviewed Original ResearchRandomized phase II studyProgression-free survivalPhase II studyNivolumab armAdverse eventsObservation armOverall survivalII studyMedian duration of follow-upProgression-free survival curvesDuration of follow-upEfficacy of nivolumabIncomplete hematologic recoverySingle-agent nivolumabEvaluation of adverse eventsAcute myeloid leukemiaIncreased AEsAML chemotherapyMedian OSNivolumab administrationNivolumab maintenanceHematologic recoveryDisease relapseOpen-labelMedian durationInhibition of MIF with an Allosteric Inhibitor Triggers Cell Cycle Arrest in Acute Myeloid Leukemia
Pantouris G, Khurana L, Tilstam P, Benner A, Cho T, Lelaidier M, Perrée M, Rosenbaum Z, Leng L, Foss F, Bhandari V, Verma A, Bucala R, Lolis E. Inhibition of MIF with an Allosteric Inhibitor Triggers Cell Cycle Arrest in Acute Myeloid Leukemia. ACS Omega 2025, 10: 17441-17452. PMID: 40352549, PMCID: PMC12059935, DOI: 10.1021/acsomega.4c10969.Peer-Reviewed Original ResearchAcute myeloid leukemiaMacrophage migration inhibitory factorCell cycle arrestNational Cancer InstituteMyeloid leukemiaMicroenvironment of acute myeloid leukemiaMIF receptorMultiple AML cell linesCycle arrestAcute myeloid leukemia pathogenesisInhibition of macrophage migration inhibitory factorPromote tumor cell survivalAcute myeloid leukemia cell survivalAcute myeloid leukemia cellsCell survivalCell linesAcute myeloid leukemia cell line HL-60Triggered cell cycle arrestTumor cell survivalAML cell linesMigration inhibitory factorMIF inhibitorExtract mechanistic insightsG0/G1 cell cycle arrestProliferation of AML cellsReal‐world treatment patterns and outcomes with oral azacitidine maintenance therapy in patients with acute myeloid leukemia
Leber B, Ruiz M, Elgendy H, Pettersson F, Prebet T, Vigil C, Parikh R, Korgaonkar S, Bello F, Davis K, Gaugler L, Strocchia M, Sieluk J, Li Y, Schuh A. Real‐world treatment patterns and outcomes with oral azacitidine maintenance therapy in patients with acute myeloid leukemia. Cancer 2025, 131: e35845. PMID: 40233158, DOI: 10.1002/cncr.35845.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaRelapse-free survivalOral-AZAMaintenance therapyMyeloid leukemiaClinical characteristicsTreatment patternsFollow-upSafety outcomes of patientsHematopoietic stem cell transplantationReal-world treatment patternsMedian Follow-UpStem cell transplantationEuropean LeukemiaNet classificationKaplan-Meier methodologyOverall survival outcomesOutcomes of patientsMedical record reviewInclusion of patientsOral azacitidineConsolidation therapyInduction therapyInduction regimensOverall survivalCell transplantationStreamline: Retrospective Cohort Study of FMS-Like Tyrosine Kinase 3-Mutated Acute Myeloid Leukemia – Real-World Treatment Patterns and Clinical Outcomes of Patients in First Relapse or Refractory Diagnosis
Zeidan A, Yu R, Wang Y, Lan Z, Grinblatt D, Elsouda D, Spalding J, Block A, Touya M, Walker M, Pandya B. Streamline: Retrospective Cohort Study of FMS-Like Tyrosine Kinase 3-Mutated Acute Myeloid Leukemia – Real-World Treatment Patterns and Clinical Outcomes of Patients in First Relapse or Refractory Diagnosis. Acta Haematologica 2025, 1-12. PMID: 40228486, PMCID: PMC12112888, DOI: 10.1159/000545384.Peer-Reviewed Original ResearchAcute myeloid leukemiaReal-world overall survivalAcute myeloid leukemia diagnosisReal-world time to next treatmentTreatment patternsFLT3-TKIMyeloid leukemiaClinical outcomesMedian real-world overall survivalTime to next treatmentClinical outcomes of patientsFLT3 tyrosine kinase inhibitorsReal-world treatment patternsAcute myeloid leukemia patientsFMS-like tyrosine kinase 3R/R AML patientsHigh-intensity chemotherapyOutcomes of patientsRetrospective cohort studyTyrosine kinase 3Retrospective longitudinal studyFirst relapseOverall survivalAML patientsFLT3 mutationsRetrospective Analysis and Characterization of Avascular Necrosis By Bone Location in Pediatric Leukemia/Lymphoma Patients.
Alayleh A, Naz H, Taylor V, Johnson T, Farook S, Hofmann G, Obilo C, Pham N, Harbacheck K, Laureano T, Smith S, Chao K, Goodman S, Shea K. Retrospective Analysis and Characterization of Avascular Necrosis By Bone Location in Pediatric Leukemia/Lymphoma Patients. Journal Of Pediatric Orthopaedics 2025 PMID: 40214168, DOI: 10.1097/bpo.0000000000002963.Peer-Reviewed Original ResearchAcute lymphoblastic leukemiaAvascular necrosisLeukemia/lymphoma patientsProximal tibial metaphysisSteroid therapyDistal femoral metaphysisHigh-dose steroid therapyMultivariate logistic regression analysisHigh-dose steroidsTibial metaphysisAcute myeloid leukemiaDiagnosis of avascular necrosisFemoral metaphysisEarly detectionBone locationEarly joint replacementLogistic regression analysisImprove functional outcomesAVN diagnosisLong bonesJoint collapseSteroid treatmentEarly-onset osteoarthritisMedian ageMyeloid leukemiaInvestigation of NPM1 mutation frequency in cutaneous blastic plasmacytoid dendritic cell neoplasms
Hamdan H, Siddon A, Ramia de Cap M, Germans S, Cantu M, Fuda F, Vandergriff T, Aggarwal N, Weinberg O. Investigation of NPM1 mutation frequency in cutaneous blastic plasmacytoid dendritic cell neoplasms. Human Pathology 2025, 158: 105766. PMID: 40216030, DOI: 10.1016/j.humpath.2025.105766.Peer-Reviewed Original ResearchConceptsBlastic plasmacytoid dendritic cell neoplasmBPDCN patientsBPDCN casesCutaneous blastic plasmacytoid dendritic cell neoplasmAcute myeloid leukemiaPlasmacytoid dendritic cell neoplasmDendritic cell neoplasmNPM1 mutationsMyeloid sarcomaCell neoplasmsSarcoma patientsImmunohistochemical stainingAbsence of NPM1 mutationsFrequency of NPM1 mutationsBone marrow of patientsLow white blood cell countCutaneous myeloid sarcomaWhite blood cell countMarrow of patientsTime of diagnosisHigher hemoglobin levelsBlood cell countPercentage of involvementSRSF2 mutationsComplex karyotypePLK1 Inhibition Induces Synthetic Lethality in Fanconi Anemia Pathway–Deficient Acute Myeloid Leukemia
Sheth A, Chan K, Liu S, Wan J, Angus S, Rhodes S, Mitchell D, Davis C, Ridinger M, Croucher P, Zeidan A, Wijeratne A, Qian S, Tran N, Potchanant E. PLK1 Inhibition Induces Synthetic Lethality in Fanconi Anemia Pathway–Deficient Acute Myeloid Leukemia. Cancer Research Communications 2025, 5: 648-667. PMID: 40111122, PMCID: PMC12011380, DOI: 10.1158/2767-9764.crc-24-0260.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaSporadic acute myeloid leukemiasMyeloid leukemiaOS of AMLMitotic centromeresPathway mutationsFA pathway-deficient cellsLack of predictive biomarkersPLK1 inhibitionFanconi anemiaSurvival of acute myeloid leukemiaPLK1 inhibitorsIdentification of patientsSynthetic lethal therapeutic strategyMitotic kinase Plk1Tumor suppression networkOverall survivalPredictive biomarkersClinical successPatient populationKinase Plk1Mitotic chromosomesTherapeutic strategiesMitotic collapseSynthetic lethality
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