2024
Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them
Bidikian A, Bewersdorf J, Kewan T, Stahl M, Zeidan A. Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them. Cancers 2024, 16: 4092. PMID: 39682277, PMCID: PMC11640703, DOI: 10.3390/cancers16234092.Peer-Reviewed Original ResearchAcute promyelocytic leukemiaAdvent of all-trans retinoic acidEarly mortalityLong-term treatment toxicitiesArsenic trioxidePromyelocytic leukemiaClinical practiceIncidence of acute promyelocytic leukemiaRates of remissionLong-term outcomesTreatment of acute promyelocytic leukemiaLong-term survivalComprehensive patient evaluationResource-limited settingsTreatment toxicityAll-trans retinoic acidDelayed diagnosisPatient demographicsSignificant comorbiditiesTreatment initiationOlder patientsExpert centersClinical trialsTreatment outcomesReal-world settingsCost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML
Alhajahjeh A, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Goshua G, Bewersdorf J, Zeidan A. Cost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML. Leukemia & Lymphoma 2024, ahead-of-print: 1-9. PMID: 39560957, DOI: 10.1080/10428194.2024.2426073.Peer-Reviewed Original ResearchConventional care regimensOlder patientsTreatment of older patientsIDH2 inhibitor enasidenibIncremental cost-effectiveness ratioCost-effectiveness ratioProbabilistic sensitivity analysesR/R AMLRefractory/relapsed AMLEvent-freeCost-effective treatmentEnasidenibCare regimensAMLPatientsCost-effectiveIncremental costLife yearsConventional careR/RIncremental effectSurvivalTreatmentPhase I Study of Ruxolitinib in Combination with Abemaciclib for Patients with Primary or Post-Polycythemia Vera/Essential Thrombocythemia Myelofibrosis
Bewersdorf J, Derkach A, Zeidan A, Stein E, Mauro M, Podoltsev N, Rampal R. Phase I Study of Ruxolitinib in Combination with Abemaciclib for Patients with Primary or Post-Polycythemia Vera/Essential Thrombocythemia Myelofibrosis. Blood 2024, 144: 6659-6659. DOI: 10.1182/blood-2024-194918.Peer-Reviewed Original ResearchDose-limiting toxicityCancer Institute Common Terminology Criteria for Adverse EventsTreatment-related adverse eventsAdverse eventsDose levelsCombination therapySpleen volumeInhibitor abemaciclibGrade 3Patients discontinued treatment due to adverse eventsNational Cancer Institute Common Terminology Criteria for Adverse EventsPhase I dose-escalation trialTreatment due to adverse eventsCommon Terminology Criteria for Adverse EventsDisease progressionRecommended phase II doseMulticenter Phase IPlanned dose levelsGrade 3 thrombocytopeniaMedian overall survivalPhase II doseBone marrow blastsBone marrow fibrosisClinically significant bleedingData cut-offPhase Ib Study of PRT543, an Oral Protein Arginine Methyltransferase 5 (PRMT5) Inhibitor, in Patients with Relapsed or Refractory, Splicing Factor-Mutant Myeloid Malignancies
Bewersdorf J, Mi X, Lu B, Kuykendall A, Sallman D, Patel M, Stevens D, Philipovskiy A, Sutamtewagul G, Masarova L, Keiffer G, Verma A, Bhagwat N, Heiser D, Ro S, Hong W, Abdel-Wahab O, Stein E. Phase Ib Study of PRT543, an Oral Protein Arginine Methyltransferase 5 (PRMT5) Inhibitor, in Patients with Relapsed or Refractory, Splicing Factor-Mutant Myeloid Malignancies. Blood 2024, 144: 3215. DOI: 10.1182/blood-2024-198495.Peer-Reviewed Original ResearchTreatment-emergent adverse eventsPlatelet transfusion independenceProtein arginine methyltransferase 5Myeloid malignanciesSplicing factor mutationsSymmetric dimethyl arginineMedian PFSTransfusion independenceFollow-upOpen-label phase Ib studyRed blood cell transfusion-dependentPRMT5 inhibitionAdequate end-organ functionBaseline serum EPO levelsRecommended phase 2 doseMedian duration of treatmentGrade 5 eventsLower-risk MDSMDS/MPN overlap syndromesPhase 2 doseIntensive induction chemotherapyPeripheral blood mononuclear cellsHigh-risk MDSPhase Ib studyMedian follow-upMis-Splicing Derived Neoantigens and Cognate T Cell Receptors in Splicing Factor Mutant Myeloid Neoplasms
Kim W, Crosse E, De Neef E, Etxeberria I, Sabio E, Wang E, Bewersdorf J, 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, Meskauskaite Urben B, Takizawa M, Krause T, Molina H, Chaligne R, Koppikar P, Molldrem J, Gigoux M, Merghoub T, Daniyan A, Greenbaum B, Klebanoff C, Bradley R, Abdel-Wahab O. Mis-Splicing Derived Neoantigens and Cognate T Cell Receptors in Splicing Factor Mutant Myeloid Neoplasms. Blood 2024, 144: 343-343. DOI: 10.1182/blood-2024-198639.Peer-Reviewed Original ResearchCD8+ T cellsT cell receptorPrimary human T cellsHuman T cellsT cellsHLA-ILeukemic cellsSRSF2 mutationsHealthy donorsIsolated CD8+ T cellsGraft-versus-leukemia effectT cell cytotoxic functionTCR-T cell therapyVirus-reactive T cellsAllogeneic stem cell transplantationT cell-based immunotherapyT cell receptor clonotypesLyse leukemic cellsPatient PBMC samplesCognate T cell receptorsDonor T cellsHigh-risk MDSStem cell transplantationHLA class IMis-splicingArtificial-Intelligence, Data-Driven, Comprehensive Classification of Myeloid Neoplasms Based on Genomic, Morphological and Histological Features
Lanino L, D'Amico S, Maggioni G, Al Ali N, Wang Y, Gurnari C, Gagelmann N, Bewersdorf J, Ball S, Guglielmelli P, Meggendorfer M, Hunter A, Kubasch A, Travaglino E, Campagna A, Ubezio M, Russo A, Todisco G, Tentori C, Buizza A, Sauta E, Zampini M, Riva E, Asti G, Delleani M, Ficara F, Santoro A, Sala C, Dall'Olio D, Dall'Olio L, Kewan T, Casetti I, Awada H, Xicoy B, Vucinic V, Hou H, Chou W, Yao C, Lin C, Tien H, Consagra A, Sallman D, Kern W, Bernardi M, Chiusolo P, Borin L, Voso M, Pleyer L, Palomo L, Quintela D, Jerez A, Cornejo E, Martin P, Díaz-Beyá M, Pita A, Roldan V, Suarez D, Velasco E, Calabuig M, Garcia-Manero G, Loghavi S, Platzbecker U, Sole F, Diez-Campelo M, Maciejewski J, Kröger N, Fenaux P, Fontenay M, Santini V, Haferlach T, Germing U, Padron E, Robin M, Passamonti F, Solary E, Vannucchi A, Castellani G, Zeidan A, Komrokji R, Della Porta M. Artificial-Intelligence, Data-Driven, Comprehensive Classification of Myeloid Neoplasms Based on Genomic, Morphological and Histological Features. Blood 2024, 144: 1005. DOI: 10.1182/blood-2024-204826.Peer-Reviewed Original ResearchGenomic featuresSplicing mutationBiallelic inactivationAnalysis of genomic profilesBiallelic inactivation of TP53Clinical phenotypeGene expression profilesCNV analysisMorphological featuresInactivation of TP53Myeloid neoplasmsGenomic characterizationRNAseq dataMorphological dataMutation screeningExpression profilesMutationsJAK/STATGenomic profilingGenomeHierarchical importanceHeterogeneous phenotypesIntegrated analysisPhenotypeHematological phenotypeValidation of Recent Response Criteria (ELN-22, IWG-23 and PB-CR) in 1634 MDS/CMML/AML Patients Treated with HMA or HMA-Ven Using CPH Models and a CPH Deep Neural Network - Can or Should Response Criteria be Harmonized for Similarly Treated Patients?
Pleyer L, Vaisband M, Klammer P, Drost M, Angermann H, Keil F, Petzer V, Heibl S, Moritz J, Girschikofsky M, Stampfl-Mattersberger M, Pichler A, Hartmann B, Aschauer G, Schmitt C, Vallet S, Boros S, Pichler P, Hammerl-Steiner A, Renneberg F, Majjiga D, Russ G, Egle A, Leisch M, Melchardt T, Zaborsky N, Faber V, Bewersdorf J, Zeidan A, Hasenauer J, Greil R. Validation of Recent Response Criteria (ELN-22, IWG-23 and PB-CR) in 1634 MDS/CMML/AML Patients Treated with HMA or HMA-Ven Using CPH Models and a CPH Deep Neural Network - Can or Should Response Criteria be Harmonized for Similarly Treated Patients? Blood 2024, 144: 7511-7511. DOI: 10.1182/blood-2024-208073.Peer-Reviewed Original ResearchComposite complete remissionTime to next treatmentCox proportional hazardsHypomethylating agentsOverall survivalCox proportional hazards modelsMedian OSResponse CriteriaTreated ptsCycles of HMAHigher-risk MDSKaplan-Meier methodBone marrow evaluationProspective cohort studyStandard of careComplete remissionMarrow evaluationTreated patientsMultivariable adjustmentNext treatmentCohort studyClinical trialsClinical overlapHazard ratioDisease entityA Niche Driven Mechanism Determines Response to ATRA and a Mutation-Independent Therapeutic Approach for MDS and AML
Mosialou I, Ali A, Cuesta-Dominguez A, Labella R, Vgenopoulou V, Bisikirska B, Galan-Diez M, Wang A, Bewersdorf J, Liang C, Heiblig M, Jurcic J, Berman E, Rabadan R, Kornblau S, Garcia-Manero G, Raza A, Kousteni S. A Niche Driven Mechanism Determines Response to ATRA and a Mutation-Independent Therapeutic Approach for MDS and AML. Blood 2024, 144: 5788-5788. DOI: 10.1182/blood-2024-212307.Peer-Reviewed Original ResearchAcute myeloid leukemiaAcute myeloid leukemia patientsStandard of careComplete responseMyelodysplastic syndrome patientsMyelodysplastic syndromeOverall response rateResponse to ATRAAll-trans-retinoic acidB-cateninOsteoblast lineage cellsMyeloid malignanciesTreatment responseLineage cellsDuration of follow-upMonitoring of treatment responseResponse rateBone marrow biopsyAbsolute neutrophil countAdverse risk groupAcute myeloid leukemia cellsNotch signalingAssociated with complete inhibitionResponse to all-trans-retinoic acidResistance to standardImmune 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 inhibitorsCost-Effectiveness of Allogeneic Hematopoietic Stem Cell Transplantation Versus Consolidation Chemotherapy for Patients with Intermediate Risk Acute Myeloid Leukemia
Alhajahjeh A, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Zeidan A, Goshua G, Bewersdorf J. Cost-Effectiveness of Allogeneic Hematopoietic Stem Cell Transplantation Versus Consolidation Chemotherapy for Patients with Intermediate Risk Acute Myeloid Leukemia. Blood 2024, 144: 788. DOI: 10.1182/blood-2024-201535.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationUpfront hematopoietic stem cell transplantationDisease-free survivalAcute myeloid leukemiaIntermediate risk acute myeloid leukemiaConsolidation chemotherapyCurative therapeutic modalityOverall survivalOne-way sensitivity analysesEuropean LeukemiaNetIncremental net monetary benefitMyeloid leukemiaMortality associated with hematopoietic stem cell transplantationCost-effective strategyTherapeutic modalitiesFavorable risk AMLSalvage hematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationCycles of consolidation chemotherapyUS health system perspectiveDiagnosed AMLFollow-up of patientsSurvival analysisHigh-dose cytarabineLines of therapyCost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States
Potnis K, Ito S, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Zeidan A, Bewersdorf J, Goshua G. Cost-Effectiveness of Eltrombopag Plus Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone in Adults with Severe Aplastic Anemia in the United States. Blood 2024, 144: 3644. DOI: 10.1182/blood-2024-204312.Peer-Reviewed Original ResearchSevere aplastic anemiaTreatment of severe aplastic anemiaQuality-adjusted life yearsAplastic anemiaIncremental net monetary benefitDeterministic sensitivity analysisProbabilistic sensitivity analysesImmunosuppressive therapyNewly diagnosed severe aplastic anemiaCost-effectiveness of eltrombopagOral thrombopoietin receptor agonistPatients treated with eltrombopagUntreated severe aplastic anemiaHematopoietic stem cell transplantationCost-effective therapeutic strategyDevelopment of adverse eventsLonger-term follow-up dataRACE trialsLater-line therapyThrombopoietin receptor agonistsPhase I/II trialSecond-line therapyStem cell transplantationU.S. payer perspectiveFirst-line treatmentEfficacy and Safety of Pembrolizumab Added to Azacitidine Plus Venetoclax for Patients with Acute Myeloid Leukemia: Results from an Investigator-Initiated, Multi-Center, CTEP-Sponsored Randomized, Phase II Trial (BLAST AML-2)
Stempel J, Uy G, Dinner S, Gojo I, Reed D, Roy R, Byrd K, Yerrabothala S, Lai C, Doucette K, Caldwell A, Blaha O, Podoltsev N, Mendez L, Bewersdorf J, Kewan T, Wistuba I, Alatrash G, Haymaker C, Streicher H, Sharon E, Little R, Gore S, Radich J, Wood B, Zeidan A, Shallis R. Efficacy and Safety of Pembrolizumab Added to Azacitidine Plus Venetoclax for Patients with Acute Myeloid Leukemia: Results from an Investigator-Initiated, Multi-Center, CTEP-Sponsored Randomized, Phase II Trial (BLAST AML-2). Blood 2024, 144: 736. DOI: 10.1182/blood-2024-210370.Peer-Reviewed Original ResearchPhase II trialTreatment-related AEsII trialFrequent treatment-related AEsSafety run-in periodAllogeneic stem cell transplantationNo dose limiting toxicitiesRandomized phase II trialAML-2Multi-centerControl armSafety of pembrolizumabDose-limiting toxicityPD-1 inhibitionAnti-PD1 antibodyIncomplete count recoveryFLT3 wild-typeIntermediate cytogenetic riskStem cell transplantationAcute myeloid leukemiaActivated T cellsRun-in periodIntention-to-treat analysisCancer Immune MonitoringLong-term survivalToxicity and Efficacy of Isavuconazole Vs Voriconazole As Anti-Fungal Prophylaxis for Patients with Acute Myeloid Leukemia
Hunter C, Bewersdorf J, Mendez L, Podoltsev N, Zeidan A, Eighmy W, Roeder H, Malinis M, Shallis R. Toxicity and Efficacy of Isavuconazole Vs Voriconazole As Anti-Fungal Prophylaxis for Patients with Acute Myeloid Leukemia. Blood 2024, 144: 1480-1480. DOI: 10.1182/blood-2024-211250.Peer-Reviewed Original ResearchInvasive fungal infectionsAcute myeloid leukemiaAntifungal prophylaxisDiagnosed AMLDrug-drug interactionsTransaminase elevationVisual disturbancesRates of invasive fungal infectionsInvasive fungal infections incidenceBaseline ANCCD4 countMyeloid leukemiaAllogeneic stem cell transplantationAnti-fungal prophylaxisAcute myeloid leukemia diagnosisDuration of neutropeniaBaseline CD4 countLess-intensive therapyStem cell transplantationPatient baseline characteristicsSide effect profileCandida sppTriazole agentsWilcoxon rank sum testAntifungal voriconazoleRisk Factors and Predictors of Outcomes after Invasive Fungal Infection Among Patients with Acute Myeloid Leukemia
Hunter C, Bewersdorf J, Mendez L, Podoltsev N, Zeidan A, Eighmy W, Roeder H, Malinis M, Shallis R. Risk Factors and Predictors of Outcomes after Invasive Fungal Infection Among Patients with Acute Myeloid Leukemia. Blood 2024, 144: 4253. DOI: 10.1182/blood-2024-211426.Peer-Reviewed Original ResearchInvasive fungal infection diagnosisInvasive fungal infectionsAcute myeloid leukemiaInvasive Fungal Infection GroupAntifungal prophylaxis agentAntifungal prophylaxisCases of invasive fungal infectionsRates of invasive fungal infectionsFungal infectionsMyeloid leukemiaPrevent invasive fungal infectionsMycoses Study Group Education and Research ConsortiumRate of mortalityIFI patientsAllogeneic stem cell transplantationRisk factorsConsensus definitionAssociated with early mortalityNon-IFI groupSingle-gene mutationsPeriod of neutropeniaDays of neutropeniaAcute myeloid leukemia subtypesStem cell transplantationKaplan-Meier methodClinical Outcomes and Variability Based on Baseline Cytogenetic Risk of Patients with MDS Treated with Hypomethylating Agents: An Analysis from the International Consortium for MDS (icMDS) Validate Database
Getz T, Kewan T, Bewersdorf J, Stempel J, Lanino L, Wei W, Al Ali N, DeZern A, Sekeres M, Uy G, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, McMahon C, Shallis R, Zeidner J, Savona M, Ball S, Chandhok N, Logothetis C, Bidikian A, Roboz G, Rolles B, Wang E, Harris A, Amaya M, Hawkins H, Grenet J, Xie Z, Madanat Y, Abaza Y, Badar T, Haferlach T, Maciejewski J, Sallman D, Enjeti A, Al-Rabi K, Halahleh K, Hiwase D, Diez-Campelo M, Valcarcel D, Haferlach C, Pleyer L, Kotsianidis I, Pappa V, Santini V, Consagra A, Al-Kali A, Ogawa S, Nannya Y, Stahl M, Della Porta M, Komrokji R, Zeidan A. Clinical Outcomes and Variability Based on Baseline Cytogenetic Risk of Patients with MDS Treated with Hypomethylating Agents: An Analysis from the International Consortium for MDS (icMDS) Validate Database. Blood 2024, 144: 3219-3219. DOI: 10.1182/blood-2024-202075.Peer-Reviewed Original ResearchNon-complex karyotypeInternational Prognostic Scoring SystemCytogenetic risk groupHMA initiationComplex karyotypeIPSS-RHypomethylating agentsOverall survivalRisk groupsCytogenetic abnormalitiesAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationAssociated with worse survivalHypomethylating agent combinationsPoor-risk cytogeneticsPeripheral blood blastsTreated with azacitidinePrognostic Scoring SystemMeasured overall survivalStem cell transplantationKaplan-Meier methodLog-rank testOutcomes of PTTime-to-event analysisRisk of patientsComparative Analysis of Outcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients Harboring Very-High Risk Cytogenetics
Aguirre L, Bewersdorf J, Liu Y, Shallis R, Boussi L, Zucenka A, Garciaz S, Bystrom R, DeAngelo D, Stone R, Luskin M, Garcia J, Winer E, Ling K, Chen E, Wadleigh M, Stein E, Goldberg A, Zeidan A, Shimony S, Stahl M. Comparative Analysis of Outcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients Harboring Very-High Risk Cytogenetics. Blood 2024, 144: 4267-4267. DOI: 10.1182/blood-2024-202744.Peer-Reviewed Original ResearchMorphologic leukemia-free stateComposite complete remissionTreated with ICIntensive chemotherapyMonosomal karyotypeComplex karyotypeProgressive diseaseSurvival outcomesTreatment strategiesAllogeneic hematopoietic stem cell transplantationComposite complete remission rateTreated with intensive chemotherapyHematopoietic stem cell transplantationComparative analysis of outcomesDismal survival outcomesConventional cytotoxic chemotherapyAggressive disease biologyMulticenter retrospective cohortStem cell transplantationAnalyze survival outcomesKaplan-Meier methodLog-rank testAnalysis of outcomesCK-AMLCPX-351Outcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients with Chromosome 5 and 7 Abnormalities
Boussi L, Bewersdorf J, Liu Y, Shallis R, Aguirre L, Zucenka A, Garciaz S, Bystrom R, DeAngelo D, Stone R, Luskin M, Garcia J, Winer E, Chen E, Wadleigh M, Ling K, Zeidan A, Goldberg A, Stein E, Shimony S, Stahl M. Outcomes with HMA Plus Venetoclax Vs Intensive Chemotherapy in AML Patients with Chromosome 5 and 7 Abnormalities. Blood 2024, 144: 4281-4281. DOI: 10.1182/blood-2024-204467.Peer-Reviewed Original ResearchAcute myeloid leukemiaMedian OSTP53 co-mutationsTreated with ICIntensive chemotherapyComposite CRCo-mutationsComplete remissionOverall survivalPts ageAllogeneic stem cell transplantationSecondary acute myeloid leukemiaDiagnosed AMLAcute myeloid leukemia patientsAssociated with poor outcomesEstimate overall survivalStem cell transplantationKaplan-Meier methodLog-rank testPredictors of survivalCPX-351Monosomy 5MRD negativityInduction therapyComplex karyotypeThe 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 featuresLeukemia Physician's Perspective on Transplant in Patients with TP53 Mutated Acute Myeloid Leukemia
Moreno Vanegas Y, El Khatib S, Hisrich B, Coltoff A, Shallis R, Patel A, Bewersdorf J, Oh T, Abaza Y, Foucar C, Goldberg A, Duvall A, Atallah E, Litzow M, Badar T. Leukemia Physician's Perspective on Transplant in Patients with TP53 Mutated Acute Myeloid Leukemia. Blood 2024, 144: 1487-1487. DOI: 10.1182/blood-2024-205782.Peer-Reviewed Original ResearchAcute myeloid leukemiaTP53-mAllo-HCTComplex cytogeneticsHypomethylating agentsInternational Consensus ClassificationRefractory diseaseSecondary AMLOverall survivalMyeloid leukemiaTP53-mutated acute myeloid leukemiaDe novo acute myeloid leukemiaProgressive acute myeloid leukemiaSecondary acute myeloid leukemiaAllogeneic hematopoietic cell transplantationMedian overall survivalTP53 allelic stateIncomplete count recoveryLow-intensity regimensReviewed electronic medical recordsHematopoietic cell transplantationRate of relapseMulticenter observational studyOverall response ratePatient selection criteriaVenetoclax-Based Therapy Versus Intensive Chemotherapy Followed By Allogeneic-Stem Cell Transplantation for High-Risk Elderly Acute Myeloid Leukemia
Iat A, Bewersdorf J, Gilhodes J, Liu Y, Shallis R, Boussi L, Zucenka A, Bystrom R, DeAngelo D, Berton G, Soua A, Ling K, Aguirre L, Stone R, Luskin M, Garcia J, Winer E, Chen E, Wadleigh M, Goldberg A, Zeidan A, Cluzeau T, Shimony S, Stahl M, Garciaz S. Venetoclax-Based Therapy Versus Intensive Chemotherapy Followed By Allogeneic-Stem Cell Transplantation for High-Risk Elderly Acute Myeloid Leukemia. Blood 2024, 144: 1508. DOI: 10.1182/blood-2024-207045.Peer-Reviewed Original ResearchCumulative incidence of relapseRelapse-free survivalAcute myeloid leukemiaOverall response rateAdverse risk cytogeneticsAllo-SCTVEN-based therapyNon-relapse mortalityIntensive chemotherapyComposite CROverall survivalIC groupMedian OSMyeloid leukemiaElderly patientsTherapy-related acute myeloid leukemiaHigh-risk acute myeloid leukemiaAllogeneic stem-cell transplantationMedian time to relapseElderly acute myeloid leukemiaLong-term disease controlResponse rateFrequent cytogenetic alterationsProspective validation trialVIALE-A trial