2024
Mis-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-splicingClinical 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 featuresImpact of Response to Hypomethylating Agent-Based Therapy on Survival Outcomes in the Context of Baseline Clinical-Molecular Risk and Transplant Status in Patients with Myelodysplastic Syndromes/Neoplasms (MDS): An Analysis from the International Consortium for MDS (icMDS) Validate Database
Rolles B, Bewersdorf J, Kewan T, Blaha O, Stempel J, Lanino L, 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, Frumm S, Barua S, Chandhok N, Logothetis C, Bidikian A, Getz T, Roboz G, Wang E, Harris A, Amaya M, Hawkins H, Ball S, 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, Della Porta M, Komrokji R, Zeidan A, Stahl M. Impact of Response to Hypomethylating Agent-Based Therapy on Survival Outcomes in the Context of Baseline Clinical-Molecular Risk and Transplant Status in Patients with Myelodysplastic Syndromes/Neoplasms (MDS): An Analysis from the International Consortium for MDS (icMDS) Validate Database. Blood 2024, 144: 664-664. DOI: 10.1182/blood-2024-208034.Peer-Reviewed Original ResearchComposite complete responseAllo-HCTOverall survivalComplete responseIPSS-MHMA therapyMedian OSResponse criteriaAllogeneic stem cell transplantationPartial hematologic recoveryClinical response criteriaStem cell transplantationHypomethylating agent-based therapyAgent-based therapyClinical practiceCox regression analysisResponse to treatmentAvailability of donorsDecitabine monotherapyImpact OSOS benefitHematologic recoveryAzacitidine monotherapyCell transplantationCombination 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 methodTargeted therapies for myelodysplastic syndromes/neoplasms (MDS): current landscape and future directions
Bidikian A, Bewersdorf J, Shallis R, Getz T, Stempel J, Kewan T, Stahl M, Zeidan A. Targeted therapies for myelodysplastic syndromes/neoplasms (MDS): current landscape and future directions. Expert Review Of Anticancer Therapy 2024, 24: 1131-1146. PMID: 39367718, DOI: 10.1080/14737140.2024.2414071.Peer-Reviewed Original ResearchErythropoiesis-stimulating agentsTargeted therapyLR-MDSHR-MDSHypoxia-inducible factorAllogeneic hematopoietic stem cell transplantationLandscape of targeted therapiesHematopoietic stem cell transplantationHeterogeneous group of hematologic malignanciesGroup of hematologic malignanciesMolecular prognostic toolsDuration of responseStem cell transplantationTrial designClinical trial designHypomethylating agentsCell transplantationHematologic malignanciesImprove patient outcomesRNA splicing machineryImmune evasionPrognostic toolTGF-betaTherapyEffective treatmentIntensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML
Bewersdorf J, Shimony S, Shallis R, Liu Y, Berton G, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Bystrom R, Lindsley R, Chen E, Perez J, Stein A, Pullarkat V, Aldoss I, DeAngelo D, Neuberg D, Stone R, Garciaz S, Ball B, Stahl M. Intensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML. Blood Advances 2024, 8: 4845-4855. PMID: 38941537, PMCID: PMC11416634, DOI: 10.1182/bloodadvances.2024012858.Peer-Reviewed Original ResearchIntensive induction chemotherapyAcute myeloid leukemiaNPM1-Mutant Acute Myeloid LeukemiaInduction chemotherapyHypomethylating agentsMulticenter retrospective cohort study of patientsPatients treated with ICAllogeneic stem cell transplantationRetrospective cohort study of patientsMulticenter retrospective cohort studyCohort study of patientsComposite complete remissionStem cell transplantationYears-oldFLT3-ITD mutationStudy of patientsStandard of careNormal cytogeneticsComplete remissionCell transplantationNPM1 mutationsMyeloid leukemiaFLT3-ITDYounger patientsOlder patientsAcute myeloid leukemia (AML) with chromosome 3 inversion: biology, management, and clinical outcome
Alhajahjeh A, Bewersdorf J, Bystrom R, Zeidan A, Shimony S, Stahl M. Acute myeloid leukemia (AML) with chromosome 3 inversion: biology, management, and clinical outcome. Leukemia & Lymphoma 2024, 65: 1541-1551. PMID: 38962996, DOI: 10.1080/10428194.2024.2367040.Peer-Reviewed Original ResearchAcute myeloid leukemiaIntensive chemotherapyHypomethylating agentsMyeloid leukemiaAllogeneic stem cell transplantationAcute myeloid leukemia casesAcute myeloid leukemia subtypesStem cell transplantationComplex hematological malignancyCurrent treatment modalitiesRare genetic anomalyCell transplantationHematologic malignanciesTreatment modalitiesClinical outcomesTreatment responseInv(3Genetic alterationsLeukemia developmentTreatment strategiesCellular processesGenetic anomaliesLeukemiaFusion geneClinical implicationsPrognostic 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 outcomeTreatment of Myelodysplastic Syndromes for Older Patients: Current State of Science, Challenges, and Opportunities
Kewan T, Stahl M, Bewersdorf J, Zeidan A. Treatment of Myelodysplastic Syndromes for Older Patients: Current State of Science, Challenges, and Opportunities. Current Hematologic Malignancy Reports 2024, 19: 138-150. PMID: 38632155, DOI: 10.1007/s11899-024-00733-y.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationLower-risk MDSErythropoiesis-stimulating agentsHypomethylating agentsIPSS-MHR-MDSRisk stratificationMolecular International Prognostic Scoring SystemRisk of leukemia progressionTreated with hypomethylating agentsInternational Prognostic Scoring SystemTreatment of myelodysplastic syndromesOral hypomethylating agentsHigh-risk MDSPrognostic Scoring SystemStem cell transplantationEnhanced risk stratificationRecent FindingsRecent advancesRefine treatment strategiesQuality-of-life improvementAssociated with treatmentTreatment decision-makingIntensive chemotherapyMDS patientsHypomethylating agents plus venetoclax compared with intensive induction chemotherapy regimens in molecularly defined secondary AML
Shimony S, Bewersdorf J, Shallis R, Liu Y, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Lindsley R, Chen E, Ramos Perez J, Stein A, DeAngelo D, Neuberg D, Stone R, Ball B, Stahl M. Hypomethylating agents plus venetoclax compared with intensive induction chemotherapy regimens in molecularly defined secondary AML. Leukemia 2024, 38: 762-768. PMID: 38378841, DOI: 10.1038/s41375-024-02175-0.Peer-Reviewed Original ResearchAssociated with improved OSHypomethylating agentsCPX-351Overall survivalSplicing factor mutationsCo-mutationsAllogeneic hematopoietic stem cell transplantationAssociated with better OSAssociated with worse OSSecondary acute myeloid leukemiaHematopoietic stem cell transplantationMedian overall survivalStem cell transplantationPatients aged >Acute myeloid leukemiaTreated with daunorubicinLiposomal daunorubicinMonosomal karyotypeNRAS/KRAS mutationsImproved OSSecondary AMLMyeloid diseasesMyeloid neoplasmsAML patientsAML treatment
2023
Validation of the Molecular International Prognostic Scoring System (IPSS-M) in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Who Underwent Allogenic Stem Cell Transplantation (HSCT)
Kewan T, Bewersdorf J, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Uy G, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, Amaya M, Zeidner J, Savona M, Stempel J, Chandhok N, Logothetis C, Cochran H, Rose A, Roboz G, Wang E, Rolles B, Harris A, Shallis R, Xie Z, Padron E, Maciejewski J, Sallman D, Della Porta M, Komrokji R, Zeidan A. Validation of the Molecular International Prognostic Scoring System (IPSS-M) in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Who Underwent Allogenic Stem Cell Transplantation (HSCT). Blood 2023, 142: 4980. DOI: 10.1182/blood-2023-186578.Peer-Reviewed Original ResearchOverall survivalHigh-risk groupUnderwent HSCTC-indexRisk groupsHigh riskDonor typeInternational Prognostic Scoring SystemAllogenic stem cell transplantationTime of HSCTMedian overall survivalReduced-intensity conditioningSignificant OS differencePrognostic scoring systemRisk stratification toolTime of diagnosisStem cell transplantationSingle-institution analysisLog-rank testHarrell's C-indexDifferent overall survivalCox proportional hazardsHMA cyclesHaploidentical donorsMaintenance therapyClinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Myelodysplastic Syndromes (MDS)
Tentori C, Gregorio C, Robin M, Gagelmann N, Gurnari C, Ball S, Berrocal J, Lanino L, D'Amico S, Spreafico M, Maggioni G, Travaglino E, Sauta E, Meggendorfer M, Zhao L, Bernardi M, Di Grazia C, Vago L, Rivoli G, Borin L, Chiusolo P, Giaccone L, Voso M, Bewersdorf J, Nibourel O, Díaz-Beyá M, Jerez A, Hernandez F, Kennedy K, Xicoy B, Ubezio M, Campagna A, Russo A, Todisco G, Mannina D, Bramanti S, Zampini M, Riva E, Bicchieri M, Asti G, Viviani F, Buizza A, Tinterri B, Bacigalupo A, Rambaldi A, Passamonti F, Ciceri F, Savevski V, Santoro A, Al Ali N, Sallman D, Sole F, Garcia-Manero G, Germing U, Kordasti S, Santini V, Sanz G, Kern W, Kubasch A, Platzbecker U, Diez-Campelo M, Maciejewski J, Ades L, Fenaux P, Haferlach T, Zeidan A, Castellani G, Komrokji R, Ieva F, Della Porta M. Clinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Myelodysplastic Syndromes (MDS). Blood 2023, 142: 197. DOI: 10.1182/blood-2023-182194.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationStem cell transplantationMyelodysplastic syndromeProlonged life expectancyClinical outcomesOptimal timingCell transplantationLife expectancyValidation cohortImmediate transplantationTransplantation policyRisks of HSCTImmediate hematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationAge groupsDiagnosis of MDSConventional prognostic scoresPost-HSCT outcomesLow-risk diseaseTiming of transplantationDisease-modifying therapiesEarly disease stagesPatient's life expectancyAverage survival timeDifferent time pointsIntensive Induction Chemotherapy (IC) Vs Hypomethylating Agents + Venetoclax (HMA/VEN) in IDH1- or IDH2-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 (IC) Vs Hypomethylating Agents + Venetoclax (HMA/VEN) in IDH1- or IDH2-Mutant Newly Diagnosed Acute Myeloid Leukemia (AML) - a Multicenter Cohort Study. Blood 2023, 142: 1589. DOI: 10.1182/blood-2023-174283.Peer-Reviewed Original ResearchIntensive induction chemotherapyAcute myeloid leukemiaComposite complete responseMedian overall survivalOverall survivalComplete responseIDH2 mutationsAllo-SCTLarge multicenter retrospective cohort studyMulticenter retrospective cohort studyAllogeneic stem cell transplantationSecondary acute myeloid leukemiaComparable overall survivalIDH1 inhibitor ivosidenibHigh rateRetrospective cohort studyStem cell transplantationLog-rank testStandard of careLarge academic centerYears of ageEffect of treatmentIDH-mutant AMLMultivariable stepwiseFit patientsWhat Is the Optimal Treatment Modality in Molecularly Defined Secondary AML? a Multicenter Cohort Study
Shimony S, Bewersdorf J, Shallis R, Liu Y, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Chen E, Ramos J, Lindsley R, Stein A, DeAngelo D, Neuberg D, Stone R, Ball B, Stahl M. What Is the Optimal Treatment Modality in Molecularly Defined Secondary AML? a Multicenter Cohort Study. Blood 2023, 142: 1478. DOI: 10.1182/blood-2023-172763.Peer-Reviewed Original ResearchAcute myeloid leukemiaComposite complete responseStem cell transplantationOverall survivalCPX-351Complete responseTreatment modalitiesMonosomal karyotypeCohort studyOdds ratioTreatment groupsLarge multicenter retrospective cohort studyMulticenter retrospective cohort studyAllogeneic stem cell transplantationSecondary acute myeloid leukemiaIncomplete count recoveryImproved overall survivalMedian overall survivalMulticenter cohort studyRetrospective cohort studyWorse overall survivalOptimal treatment modalityOptimal treatment selectionLog-rank testEffect of treatment