2024
Cost-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 therapyClinical 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 patientsOutcomes 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 featuresVenetoclax-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 trialImpact 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 therapyValidation 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 Molecular-Based Ecosystem to Improve Personalized Medicine in Patients with Chronic Myelomonocytic Leukemia (CMML)
Lanino L, Hunter A, Gagelmann N, Robin M, Sala C, Dall'Olio D, Gurnari C, Dall'Olio L, Wang Y, Pleyer L, Xicoy B, Montalban-Bravo G, Shih L, Haque T, Abdel-Wahab O, Geissler K, Bataller A, Bazinet A, Meggendorfer M, Casetti I, Sauta E, Travaglino E, Palomo L, Zamora L, Quintela D, Jerez A, Cornejo E, Garcia Martin P, Díaz-Beyá M, Avendaño Pita A, Roldan V, Fiallo Suarez D, Cerezo Velasco E, Calabuig M, Such E, Sanz G, Kubasch A, Castilla-Llorente C, Bulabois C, Souchet L, Awada H, Bernardi M, Chiusolo P, Curti A, Giaccone L, Onida F, Borin L, Passamonti F, Diral E, Vucinic V, Bergonzi G, Voso M, Hou H, Chou W, Yao C, Lin C, Tien H, Campagna A, Ubezio M, Russo A, Todisco G, Maggioni G, Tentori C, Buizza A, Asti G, Zampini M, Riva E, Delleani M, Consagra A, Ficara F, Santoro A, Carota L, Sanavia T, Rollo C, Kiwan A, VanOudenhove J, Fariselli P, Al Ali N, Sallman D, Kern W, Garcia-Manero G, Thota S, Griffiths E, Follo M, Finelli C, Platzbecker U, Sole F, Diez-Campelo M, Maciejewski J, Bejar R, Thol F, Kröger N, Fenaux P, Itzykson R, Graubert T, Fontenay M, Zeidan A, Komrokji R, Santini V, Haferlach T, Germing U, D'Amico S, Castellani G, Patnaik M, Solary E, Padron E, Della Porta M. A Molecular-Based Ecosystem to Improve Personalized Medicine in Patients with Chronic Myelomonocytic Leukemia (CMML). Blood 2024, 144: 1003-1003. DOI: 10.1182/blood-2024-200104.Peer-Reviewed Original ResearchChronic myelomonocytic leukemiaLeukemia-free survivalMyeloid neoplasmsProportion of patientsOverall survivalMolecular-based toolsMolecular informationEvaluation of mutation statusInfluence disease phenotypeGenomic overlapScoring systemGenomic associationsGenomic featuresSplicing machineryConcordance indexGenomic characterizationChronic myelomonocytic leukemia patientsMedian leukemia-free survivalProbability of disease relapseAllogeneic stem cell transplantationSignal transductionGenomic heterogeneityRisk of disease progressionMulti-color flow cytometryMutation screeningOral Decitabine/Cedazuridine in Patients with MDS and TP53 Mutations: A Propensity Score Matching Analysis from the Phase II and III Trials
Urrutia S, Sasaki K, Bataller A, Kantarjian H, Montalban-Bravo G, McCloskey J, Griffiths E, Yee K, Zeidan A, Savona M, Oganesian A, Sano Y, Keer H, Garcia-Manero G. Oral Decitabine/Cedazuridine in Patients with MDS and TP53 Mutations: A Propensity Score Matching Analysis from the Phase II and III Trials. Blood 2024, 144: 661-661. DOI: 10.1182/blood-2024-193274.Peer-Reviewed Original ResearchMedian overall survivalHematopoietic stem cell transplantationHypomethylating agentsTP53 mutationsOverall survivalImproved survivalMedian follow-up timeIPSS-R scoreComplete response rateStem cell transplantationECOG performance statusPropensity score matching analysisFollow-up timeScore matching analysisIPSS-RComplex cytogeneticsCell transplantationPerformance statusDecitabine/cedazuridineTP53wtCo-mutationsLandmark analysisPoor outcomeTP53mutMedian numberEvaluating Molecular and Clinical Predictors in Myelodysplastic Syndromes through Machine Learning Integration
Mosquera Orgueira A, Perez Encinas M, Pérez Míguez C, Crucitti D, Piñeiro Fiel M, Díaz Varela N, Mora E, Díaz-Beyá M, Montoro M, Pomares H, Ramos Ortega F, Tormo M, Jerez A, Nomdedeu J, de Miguel Sanchez C, Arenillas L, Carcel Corella P, Cedena Romero M, Xicoy B, Rivero M, Del Orbe Barreto R, Bewersdorf J, Stahl M, Stempel J, Kewan T, Zeidan A, Diez-Campelo M, Valcarcel D. Evaluating Molecular and Clinical Predictors in Myelodysplastic Syndromes through Machine Learning Integration. Blood 2024, 144: 6683-6683. DOI: 10.1182/blood-2024-198114.Peer-Reviewed Original ResearchLeukemia-free survivalPredictors of OSMyelodysplastic syndromeOverall survivalIPSS-MC-indexFLT3-ITDPredictors of leukemia-free survivalRevised International Prognostic Scoring SystemPrognostication of myelodysplastic syndromesInternational Prognostic Scoring SystemPrognostic Scoring SystemCox regression analysisMolecular dataClinical trial designIPSS-RGenetic variabilityMLL-PTDRandom survival forestPrognostic landscapePrognostic significanceLaboratory parametersAffected prognosisClinical predictorsClinical indicesTrial in Progress: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 2 Study of AK117/Placebo in Combination with Azacitidine in Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes (AK117-205)
Zeidan A, Tong H, Xiao Z, Baratam P, Abboud R, Benton C, Zeidner J, Borate U, Chai-Ho W, Lu Y, Yang J, Hu M, Li B, Xia M, Sallman D. Trial in Progress: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 2 Study of AK117/Placebo in Combination with Azacitidine in Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes (AK117-205). Blood 2024, 144: 6705-6705. DOI: 10.1182/blood-2024-200541.Peer-Reviewed Original ResearchAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationEastern Cooperative Oncology GroupIPSS-R scoreEvent-free survivalStem cell transplantationAcute myeloid leukemiaOverall survivalIPSS-RTransfusion independenceComplete responseCR rateDouble-blindCell transplantationHR-MDSMyeloproliferative neoplasmsAdverse eventsChimeric antigen receptor T cellsTransformation to acute myeloid leukemiaAnemia ratesMulticenter phase 2 studyTreated with hypomethylating agentsHigher-risk myelodysplastic syndromesSeverity of adverse eventsAdequate organ functionTrial in Progress: The Phase 3, Randomized, Double-Blind, Placebo-Controlled QuANTUM-Wild Study of Quizartinib in Combination With Chemotherapy and as Single-Agent Maintenance in Newly Diagnosed, FLT3 -ITD-Negative Acute Myeloid Leukemia
Montesinos P, Cheong J, Daver N, Fathi A, Levis M, Luger S, Miyamoto T, Oliva E, Perl A, Récher C, Schlenk R, Wang J, Zeidan A, Liu L, Duong Y, Imadalou K, Alexis K, Nahar A, Burns K, Erba H. Trial in Progress: The Phase 3, Randomized, Double-Blind, Placebo-Controlled QuANTUM-Wild Study of Quizartinib in Combination With Chemotherapy and as Single-Agent Maintenance in Newly Diagnosed, FLT3 -ITD-Negative Acute Myeloid Leukemia. Blood 2024, 144: 1504.3-1504.3. DOI: 10.1182/blood-2024-205101.Peer-Reviewed Original ResearchFMS-like tyrosine kinase 3Acute myeloid leukemiaRelapse-free survivalEvent-free survivalArm AOverall survivalEligible ptsFLT3-ITDMonotherapy maintenanceAllo-HSCTDouble-blindFLT3 mutationsArm CMyeloid leukemiaAssociated with significantly prolonged overall survivalFLT3 activationFms-like tyrosine kinase 3 ligandFMS-like tyrosine kinase 3-internal tandem duplicationAllogeneic hematopoietic stem cell transplantationFms-like tyrosine kinase 3-mutatedRelapsed/refractory acute myeloid leukemiaBone marrow blast countFms-like tyrosine kinase 3 expressionCases of acute myeloid leukemiaHematopoietic stem cell transplantationEnhancing Personalized Prognostic Assessment of Myelodysplastic Syndromes through a Multimodal and Explainable Deep Data Fusion Approach (MAGAERA)
Sauta E, Sartori F, Lanino L, Asti G, D'Amico S, Delleani M, Riva E, Zampini M, Zazzetti E, Bicchieri M, Maggioni G, Campagna A, Todisco G, Tentori C, Ubezio M, Russo A, Buizza A, Ficara F, Crisafulli L, Brindisi M, Ventura D, Pinocchio N, Rahal D, Lancellotti C, Bonometti A, Di Tommaso L, Savevski V, Santoro A, Derus N, Dall'Olio D, Santini V, Sole F, Platzbecker U, Fenaux P, Diez-Campelo M, Komrokji R, Garcia-Manero G, Haferlach T, Kordasti S, Zeidan A, Castellani G, Sanavia T, Fariselli P, Della Porta M. Enhancing Personalized Prognostic Assessment of Myelodysplastic Syndromes through a Multimodal and Explainable Deep Data Fusion Approach (MAGAERA). Blood 2024, 144: 105-105. DOI: 10.1182/blood-2024-205413.Peer-Reviewed Original ResearchPersonalized medicine programsMyelodysplastic syndrome patientsMyelodysplastic syndromeOverall survivalConcordance indexClinical outcomesMay-Grunwald-GiemsaHypomethylating agentsBone marrowAnalysis of hematological malignanciesSomatic mutation screeningEvaluation of T lymphocytesResponse to hypomethylating agentsCD34+ bone marrowStudies of myelodysplastic syndromesGenomic featuresMDS populationRNA-seqPrediction of patient outcomeGenomic characterizationHarrell's concordance indexPredicting clinical outcomesHematoxylin and eosin (H&EMorphological dataMulti-omicsArtificial Intelligence-Powered Digital Pathology to Improve Diagnosis and Personalized Prognostic Assessment in Patient with Myeloid Neoplasms
Asti G, Curti N, Maggioni G, Carlini G, Lanino L, Campagna A, D'Amico S, Sauta E, Delleani M, Bonometti A, Lancellotti C, Rahal D, Ubezio M, Todisco G, Tentori C, Russo A, Crespi A, Figini G, Buizza A, Riva E, Zampini M, Brindisi M, Ficara F, Crisafulli L, Ventura D, Pinocchio N, Zazzetti E, Bicchieri M, Grondelli M, Forcina Barrero A, Savevski V, Santoro A, Santini V, Sole F, Platzbecker U, Fenaux P, Diez-Campelo M, Komrokji R, Haferlach T, Kordasti S, Di Tommaso L, Zeidan A, Loghavi S, Garcia-Manero G, Castellani G, Della Porta M. Artificial Intelligence-Powered Digital Pathology to Improve Diagnosis and Personalized Prognostic Assessment in Patient with Myeloid Neoplasms. Blood 2024, 144: 3598-3598. DOI: 10.1182/blood-2024-206248.Peer-Reviewed Original ResearchLeukemia-free survivalMyeloid neoplasmsOverall survivalConcordance indexGenomic informationBone marrowPredictive of overall survivalMD Anderson Cancer CenterCell typesProportion of patientsHarrell's concordance indexSomatic gene mutationsMorphological featuresHumanitas Research HospitalGenomic dataMGG smearsPersonalized risk assessmentRUNX1 mutationsBM aspiratesClinically relevant informationClinical entityBiopsy dataMN patientsPrognostic assessmentWhole slide imagesMDS-157 Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Santini V, Komrokji R, Sekeres M, Savona M, Fenaux P, Madanat Y, Oliva E, Buckstein R, Annaášová A, Germing U, Mittelman M, Thepot S, Riggs J, Dougherty S, Berry T, Navada S, Xia Q, Sun L, Zeidan A, Platzbecker U. MDS-157 Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s386-s387. DOI: 10.1016/s2152-2650(24)01345-4.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesNon-del(5q) lower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsRBC-TIOverall survivalRBC-TDNon-del(5qClinical benefitRed blood cellsHemoglobin increaseRBC transfusion dependenceStratified log-rank testMedian follow-upKaplan-Meier methodLog-rank testWithdrawal of consentMedian OSOS ratesHemoglobin riseMyelodysplastic syndromeOS analysisHemoglobin levelsAssess OSPlaceboImetelstatOverall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Santini V, Komrokji R, Sekeres M, Savona M, Fenaux P, Madanat Y, Oliva E, Buckstein R, Jonášová A, Germing U, Mittelman M, Thepot S, Riggs J, Dougherty S, Berry T, Navada S, Xia Q, Sun L, Zeidan A, Platzbecker U. Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s192. DOI: 10.1016/s2152-2650(24)00647-5.Peer-Reviewed Original ResearchOverall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Santini V, Komrokji R, Sekeres M, Savona M, Fenaux P, Madanat Y, Oliva E, Buckstein R, Jonášová A, Germing U, Mittelman M, Thepot S, Riggs J, Dougherty S, Berry T, Navada S, Xia Q, Sun L, Zeidan A, Platzbecker U. Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s153. DOI: 10.1016/s2152-2650(24)00388-4.Peer-Reviewed Original ResearchCost-effectiveness of adding quizartinib to induction chemotherapy for patients with FLT3-mutant acute myeloid leukemia
Bewersdorf J, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Stahl M, Stein E, Huntington S, Goshua G, Zeidan A. Cost-effectiveness of adding quizartinib to induction chemotherapy for patients with FLT3-mutant acute myeloid leukemia. Leukemia & Lymphoma 2024, 65: 1136-1144. PMID: 38648559, PMCID: PMC11265977, DOI: 10.1080/10428194.2024.2344052.Peer-Reviewed Original ResearchQuality-adjusted life yearsCompletion of consolidation therapyFLT3-mutant acute myeloid leukemiaAllogeneic hematopoietic cell transplantationIncremental cost-effectiveness ratioProbabilistic sensitivity analysesImproved overall survivalHematopoietic cell transplantationPartitioned survival analysis modelAcute myeloid leukemiaCost-effectiveness ratioFLT3 inhibitor quizartinibHealth economic implicationsConsolidation therapyInduction chemotherapyAverage wholesale priceOverall survivalCell transplantationContinuous therapyMyeloid leukemiaITD mutationQuizartinibIncremental costCost-effective optionLife yearsHypomethylating 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 Were Treated with Hypomethylating Agents (HMA)
Kewan T, Bewersdorf J, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, Amaya M, Zeidner J, Savona M, Stempel J, Chandhok N, Logothetis C, Ramaswamy R, Rose A, Roboz G, Rolles B, Wang E, 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 Were Treated with Hypomethylating Agents (HMA). Blood 2023, 142: 3240. DOI: 10.1182/blood-2023-186340.Peer-Reviewed Original ResearchComplete remission rateOverall response rateOutcome of ptsMedian overall survivalOverall survivalHypomethylating agentHMA initiationHR-MDSC-indexRisk groupsScoring systemInternational Prognostic Scoring SystemResponse criteriaPrognostic scoring systemHigh-risk diseaseLarge multicenter cohortHigh-risk groupHarrell's C-indexLog-rank testPrediction of outcomeDifferent scoring systemsSubsequent validation studiesHMA cyclesMedian followAllogeneic HSCT