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 patientsMore 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 ResearchA phase I study of asciminib in combination with dasatinib, prednisone, and blinatumomab for Ph-positive acute leukemia in adults.
Luskin M, Murakami M, Keating J, Wang E, McMasters M, Flamand Y, Winer E, Stahl M, Smith H, Jaeckle S, Weizer C, Fleming J, Saygin C, Stock W, DeAngelo D. A phase I study of asciminib in combination with dasatinib, prednisone, and blinatumomab for Ph-positive acute leukemia in adults. Journal Of Clinical Oncology 2025, 43: 6509-6509. DOI: 10.1200/jco.2025.43.16_suppl.6509.Peer-Reviewed Original ResearchPh+ acute lymphoblastic leukemiaAcute lymphoblastic leukemiaDose-limiting toxicityChronic myeloid leukemiaDose reductionAcute leukemiaPh-positive acute leukemiaTreatment of Ph+ acute lymphoblastic leukemiaDasatinib dose reductionT-cell engagersPhase I studyPhase 1 studyPh+ acute leukemiaSafety of dasatinibEfficacy of dasatinibExpansion cohortBlast crisisMedian WBCToxicity gradeMedian agePhysician's discretionPleural effusionMedian timeMyeloid leukemiaLymphoblastic leukemiaOverall 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 benefitUnveiling 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 prosurvivalMis-splicing-derived neoantigens and cognate TCRs in splicing factor mutant leukemias
Kim W, Crosse E, De Neef E, Etxeberria I, Sabio E, Wang E, Bewersdorf J, Lin K, Lu S, Belleville A, Fox N, Castro C, Zhang P, Fujino T, Lewis J, Rahman J, Zhang B, Winick J, Lewis A, Stanley R, DeWolf S, Urben B, Takizawa M, Krause T, Molina H, Chaligne R, Koppikar P, Molldrem J, Gigoux M, Merghoub T, Daniyan A, Chandran S, Greenbaum B, Klebanoff C, Bradley R, Abdel-Wahab O. Mis-splicing-derived neoantigens and cognate TCRs in splicing factor mutant leukemias. Cell 2025, 188: 3422-3440.e24. PMID: 40273911, PMCID: PMC12204805, DOI: 10.1016/j.cell.2025.03.047.Peer-Reviewed Original ResearchConceptsT cell receptorT cellsCurative allogeneic stem cell transplantationVirus-reactive T cellsAllogeneic stem cell transplantationCD8<sup>+</sup> T cellsCognate T cell receptorsStem cell transplantationBlood of patientsImpaired cytotoxic functionPeptide-major histocompatibility complexMyeloid malignanciesCell transplantationActive cancerCytotoxic functionMyeloid leukemiaHealthy donorsPublic neoantigensNeoantigensHistocompatibility complexSplicing alterationsLeukemiaMis-splicing eventsRNA splicing factorsPatientsInhibition 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 leukemiaPLK1 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 lethalityCARDIOVASCULAR EVENTS IN PATIENTS WITH ACUTE MYELOID LEUKEMIA TREATED WITH VENETOCLAX: A MULTICENTER STUDY
Filimon S, Ghamari A, Vakilpour A, Patel R, Achar R, Akhter N, Shallis R, DeCara J, Anazco F, Altman J, Smith A, Matthews A, Lai C, Brunner A, Farina K, Gilman H, Neilan T, Upadhyay D, Patel A, Tremblay D, Scherrer-Crosbie M. CARDIOVASCULAR EVENTS IN PATIENTS WITH ACUTE MYELOID LEUKEMIA TREATED WITH VENETOCLAX: A MULTICENTER STUDY. Journal Of The American College Of Cardiology 2025, 85: 2865. DOI: 10.1016/s0735-1097(25)03349-2.Peer-Reviewed Original ResearchTP53‐Mutated Acute Myeloid Leukemia and Blast Phase Myeloproliferative Neoplasm: Distinct Mutation Leads to Poorer Prognosis
Chen D, Cantu M, Siddon A, Weinberg O. TP53‐Mutated Acute Myeloid Leukemia and Blast Phase Myeloproliferative Neoplasm: Distinct Mutation Leads to Poorer Prognosis. European Journal Of Haematology 2025, 115: 57-63. PMID: 40152319, PMCID: PMC12134709, DOI: 10.1111/ejh.14421.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaTP53-mutated acute myeloid leukemiaBlast phase myeloproliferative neoplasmsTP53 mutationsClinical outcomesMPN-BPMyeloproliferative neoplasmsMyeloid leukemiaAssociated with prognostic significanceMissense mutationsLow-risk groupSplice site variantOverall survivalAssociated with relatively better prognosisMyeloid neoplasmsPrognostic significanceBetter prognosisTP53 mutantsTherapeutic responseRisk stratificationRetrospective analysisExon 5TP53Exon 6Poorer PrognosisContemporary 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, 25: 435-456. 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
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