2024
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?
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 entityOutcomes 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 karyotypeIntensive 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 patients
2023
A multicenter phase Ib trial of the histone deacetylase inhibitor entinostat in combination with pembrolizumab in patients with myelodysplastic syndromes/neoplasms or acute myeloid leukemia refractory to hypomethylating agents
Bewersdorf J, Shallis R, Sharon E, Park S, Ramaswamy R, Roe C, Irish J, Caldwell A, Wei W, Yacoub A, Madanat Y, Zeidner J, Altman J, Odenike O, Yerrabothala S, Kovacsovics T, Podoltsev N, Halene S, Little R, Piekarz R, Gore S, Kim T, Zeidan A. A multicenter phase Ib trial of the histone deacetylase inhibitor entinostat in combination with pembrolizumab in patients with myelodysplastic syndromes/neoplasms or acute myeloid leukemia refractory to hypomethylating agents. Annals Of Hematology 2023, 103: 105-116. PMID: 38036712, DOI: 10.1007/s00277-023-05552-4.Peer-Reviewed Original ResearchConceptsDose-limiting toxicityAcute myeloid leukemiaMarrow complete remissionPhase Ib trialAdverse eventsIb trialDose escalationNCI Cancer Therapy Evaluation ProgramAcute myeloid leukemia refractoryHematologic adverse eventsProtocol-defined responseDose level 1Anti-PD1 therapyAnti-PD1 antibodyDose-escalation designLimited clinical efficacySystems immunology approachHistone deacetylase inhibitor entinostatLeukemia refractoryMCR patientsComplete remissionRespiratory failureSuppressor cellsEscalation designClinical efficacyBlinatumomab in Combination with Immune Checkpoint Inhibitors (ICIs) of PD-1 and CTLA-4 in Adult Patients with Relapsed/Refractory (R/R) CD19 Positive B-Cell Acute Lymphoblastic Leukemia (ALL): Results of a Phase I Study
Webster J, Luskin M, Rimando J, Blackford A, Zeidan A, Sharon E, Streicher H, DeAngelo D, Luznik L, Gojo I. Blinatumomab in Combination with Immune Checkpoint Inhibitors (ICIs) of PD-1 and CTLA-4 in Adult Patients with Relapsed/Refractory (R/R) CD19 Positive B-Cell Acute Lymphoblastic Leukemia (ALL): Results of a Phase I Study. Blood 2023, 142: 966. DOI: 10.1182/blood-2023-191109.Peer-Reviewed Original ResearchImmune checkpoint inhibitorsMixed phenotype acute leukemiaRelapse-free survivalOverall survivalAcute lymphoblastic leukemiaComplete remissionPD-1Checkpoint inhibitorsExtramedullary diseaseAdverse eventsBM blastsGrade 3Positive B-cell acute lymphoblastic leukemiaResponse rateImmune-related adverse eventsB-cell acute lymphoblastic leukemiaMulti-center phase IPhase IDose-escalation schemaNon-hematologic toxicitiesMedian overall survivalDose-escalation studyPD-L1 expressionDuration of responseT cell subpopulationsMolecular Measurable Residual Disease (MRD) Clearance (≤1%) Is Associated with Improved Clinical Outcomes in Patients with Higher-Risk Myelodysplastic Neoplasms (HR-MDS): An Exploratory Analysis of Stimulus-MDS1 in Patients Receiving Sabatolimab or Placebo + Hypomethylating Agent (HMA)
Zeidan A, Fenaux P, Han X, James D, Malek K, Ramos P, Miyazaki Y, Platzbecker U. Molecular Measurable Residual Disease (MRD) Clearance (≤1%) Is Associated with Improved Clinical Outcomes in Patients with Higher-Risk Myelodysplastic Neoplasms (HR-MDS): An Exploratory Analysis of Stimulus-MDS1 in Patients Receiving Sabatolimab or Placebo + Hypomethylating Agent (HMA). Blood 2023, 142: 3236. DOI: 10.1182/blood-2023-180765.Peer-Reviewed Original ResearchProgression-free survivalTime-dependent Cox modelBest overall responseOverall survivalHypomethylating agentMRD cohortComplete remissionClinical outcomesVariant allelic frequencyMRD statusPartial remissionPrognostic valueMRD-1Next-generation sequencingLandmark analysisCox modelCR/PRInternational Prognostic Scoring SystemMarrow complete remissionHigh-risk MDSPrognostic scoring systemLow disease burdenMononuclear cell samplesLower hazard ratioPotential prognostic valueCharacteristics and Outcomes of Younger Adult Patients (Pts) with Myelodysplastic Syndromes (MDS): A Multicenter Retrospective Study
Abaza Y, Xie Z, Burkart M, Badar T, Desai P, Shallis R, Patel A, Cohen-Nowak A, Oh T, Walker C, Easwar N, Kewan T, Cannova J, Bell-Burdett K, Al Ali N, Sallman D, Roboz G, Carraway H, Zeidan A, Patnaik M, Altman J, Komrokji R. Characteristics and Outcomes of Younger Adult Patients (Pts) with Myelodysplastic Syndromes (MDS): A Multicenter Retrospective Study. Blood 2023, 142: 3232. DOI: 10.1182/blood-2023-188328.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeMedian overall survivalMulticenter retrospective studyRisk myelodysplastic syndromesOverall survivalYA groupMyelodysplastic syndromeYounger ptsComplete remissionAllo-SCTBaseline characteristicsFrontline therapyHematologic improvementMedian ageRetrospective studyCommon subtypeBlasts-2Exact testUpfront allogeneic stem cell transplantationIntermediate-risk myelodysplastic syndromesTherapy-related myelodysplastic syndromeAllogeneic stem cell transplantationDe novo myelodysplastic syndromeExcess blasts-2Marrow complete remissionEncouraging Efficacy Observed in Bexmab Study: A Phase 1/2 Study to Assess Safety and Efficacy of Bexmarilimab in Combination with Standard of Care in Myeloid Malignancies
Kontro M, Stein A, Pyörälä M, Rimpiläinen J, Siitonen T, Hollmén M, Fjaellskog M, Pawlitzky I, Zeidan A, Daver N. Encouraging Efficacy Observed in Bexmab Study: A Phase 1/2 Study to Assess Safety and Efficacy of Bexmarilimab in Combination with Standard of Care in Myeloid Malignancies. Blood 2023, 142: 2915. DOI: 10.1182/blood-2023-174912.Peer-Reviewed Original ResearchAcute myeloid leukemiaR AMLCLEVER-1Complete remissionStandard of careBex treatmentAdverse eventsBone marrow cellsHMA failureMarrow CRPatient BMPrior therapyPartial remissionAML patientsMedian numberT cellsDose levelsMyeloid malignanciesHigh-risk MDS patientsMarrow cellsMarrow complete remissionR AML patientsRisk MDS patientsNK cell numbersPhase 1/2 studySafety, Pharmacodynamic, and Anti-Tumor Activity of SL-172154 As Monotherapy and in Combination with Azacitidine (AZA) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) Patients (pts)
Daver N, Stein A, Bixby D, Chai-Ho W, Zeidner J, Maher K, Stevens D, Stahl M, Yee K, Curran E, Ito S, Sochacki A, Sallman D, Hernandez R, Metenou S, Ma B, Kato K, Zeidan A. Safety, Pharmacodynamic, and Anti-Tumor Activity of SL-172154 As Monotherapy and in Combination with Azacitidine (AZA) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) Patients (pts). Blood 2023, 142: 4278. DOI: 10.1182/blood-2023-173991.Peer-Reviewed Original ResearchR AMLAcute myeloid leukemiaTreatment-emergent AEsInfusion-related reactionsDose-limiting toxicityDose-escalation cohortsHR-MDSDose-dependent increaseComplete remissionObjective responseAnti-tumor activityBone marrowHypomethylating agentAllo-HCTAML ptsEvaluable ptsEscalation cohortsDose escalationRelapsed/Refractory Acute Myeloid LeukemiaMedian age 70 yearsMorphologic leukemia-free statePhase 1 dose escalationSIRPα-Fc fusion proteinRefractory acute myeloid leukemiaMarrow complete remissionPost Allogeneic Stem Cell Transplant Outcomes Following Response to Hypomethylating Agent Therapy in Myelodysplastic Syndromes Are Predicted By Persistent International Prognostic Scoring System-Molecular Risk
Frumm S, Kim H, Kelkar A, Ho V, Gooptu M, Gibson C, Koreth J, Shapiro R, Romee R, Nikiforow S, Antin J, Soiffer R, Rolles B, Shimony S, Bewersdorf J, Kewan T, Alhajahjeh A, Luskin M, Garcia J, Chen E, Lane A, Wadleigh M, Winer E, Stone R, DeAngelo D, Zeidan A, Lindsley C, Cutler C, Stahl M. Post Allogeneic Stem Cell Transplant Outcomes Following Response to Hypomethylating Agent Therapy in Myelodysplastic Syndromes Are Predicted By Persistent International Prognostic Scoring System-Molecular Risk. Blood 2023, 142: 3618. DOI: 10.1182/blood-2023-185220.Peer-Reviewed Original ResearchHematopoietic stem cell transplantBlood count recoveryPost-HCT outcomesComplete remissionTime of diagnosisMyelodysplastic syndromeOverall survivalHigh riskLower riskAgent therapyCount recoveryMolecular riskInternational Working Group response criteriaShorter median overall survivalResponse criteriaDana-Farber Cancer InstituteHCT comorbidity indexImportant prognostic impactTAC/sirolimusHost disease (GVHD) prophylaxisMedian overall survivalProgression-free survivalStem cell transplantBone marrow biopsyFuture prospective studiesImpact of Type of Hypomethylating Agent (HMA) Used on Outcomes of Patients (Pts) with Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) - a Large, Multicenter, Retrospective Analysis
Bewersdorf J, Kewan T, Blaha O, Stahl M, Al Ali N, DeZern A, Sekeres M, Uy G, Carraway H, Desai P, Griffiths E, Stein E, Brunner A, McMahon C, Zeidner J, Savona M, Stempel J, Chandhok N, Ramaswamy R, Roboz G, Rolles B, Wang E, Harris A, Amaya M, Hawkins H, Grenet J, Gurnari C, Shallis R, Xie Z, Maciejewski J, Sallman D, Della Porta M, Komrokji R, Zeidan A. Impact of Type of Hypomethylating Agent (HMA) Used on Outcomes of Patients (Pts) with Higher-Risk Myelodysplastic Syndromes/Neoplasms (HR-MDS) - a Large, Multicenter, Retrospective Analysis. Blood 2023, 142: 4613. DOI: 10.1182/blood-2023-178728.Peer-Reviewed Original ResearchCox multivariable regression modelOverall survivalHMA initiationHypomethylating agentMultivariable regression modelsTP53 mutationsAllo-HCTComplete remissionComplex karyotypePartner drugsBone marrowSurvival analysisAllogeneic hematopoietic cell transplantMultivariable Cox regression modelsTreatment typeOverall responseAdverse genetic featuresMedian overall survivalOutcomes of patientsHematopoietic cell transplantAdverse overall survivalKaplan-Meier methodCox regression modelLog-rank testPredictors of responseSpliceosome mutations are associated with clinical response in a phase 1b/2 study of the PLK1 inhibitor onvansertib in combination with decitabine in relapsed or refractory acute myeloid leukemia
Croucher P, Ridinger M, Becker P, Lin T, Silberman S, Wang E, Zeidan A. Spliceosome mutations are associated with clinical response in a phase 1b/2 study of the PLK1 inhibitor onvansertib in combination with decitabine in relapsed or refractory acute myeloid leukemia. Annals Of Hematology 2023, 102: 3049-3059. PMID: 37702821, PMCID: PMC10567832, DOI: 10.1007/s00277-023-05442-9.Peer-Reviewed Original ResearchAcute myeloid leukemiaR AML patientsAML patientsMyeloid leukemiaRefractory (R/R) AMLRelapsed/refractory (R/R) AMLHigher CR/CRi ratesCR/CRi rateRefractory acute myeloid leukemiaGene signaturePhase 1b/2 studyPhase 1b/2 trialPhase 1b trialBone marrow blastsSF mutationsBone marrow samplesCRi rateComplete remissionMarrow blastsClinical responseCount recoveryInitial safetyMarrow samplesPotential therapyMolecular predictorsVenetoclax plus azacitidine compared with intensive chemotherapy as induction for patients with acute myeloid leukemia: retrospective analysis of an electronic medical record database in the United States
Zeidan A, Pollyea D, Borate U, Vasconcelos A, Potluri R, Rotter D, Kiendrebeogo Z, Gaugler L, Prebet T, Strocchia M, Bonifacio G, Chen C. Venetoclax plus azacitidine compared with intensive chemotherapy as induction for patients with acute myeloid leukemia: retrospective analysis of an electronic medical record database in the United States. Annals Of Hematology 2023, 102: 749-754. PMID: 36732419, PMCID: PMC10285011, DOI: 10.1007/s00277-023-05109-5.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantAcute myeloid leukemiaRelapse-free survivalIntensive chemotherapyComplete remissionOverall survivalMyeloid leukemiaRetrospective analysisMedian relapse-free survivalElectronic medical record databaseFlatiron Health databaseMedian overall survivalStem cell transplantMedical record databaseElectronic health recordsHSCT ratesRelapse rateCell transplantControlled TrialsTreatment outcomesHealth databasesPatientsRecord databaseMonthsHealth recordsConsensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes
Zeidan A, Platzbecker U, Bewersdorf J, Stahl M, Adès L, Borate U, Bowen D, Buckstein R, Brunner A, Carraway H, Daver N, Díez-Campelo M, de Witte T, DeZern A, Efficace F, Garcia-Manero G, Garcia J, Germing U, Giagounidis A, Griffiths E, Hasserjian R, Hellström-Lindberg E, Iastrebner M, Komrokji R, Kulasekararaj A, Malcovati L, Miyazaki Y, Odenike O, Santini V, Sanz G, Scheinberg P, Stauder R, van de Loosdrecht A, Wei A, Sekeres M, Fenaux P. Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes. Blood 2023, 141: 2047-2061. PMID: 36724453, DOI: 10.1182/blood.2022018604.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHigh-risk MDSComplete remissionResponse criteriaInternational Working GroupClinical trialsHigh-risk myelodysplastic syndromeEnd pointMarrow complete remissionPartial hematologic recoveryClinical end pointsPatient-centered outcomesNovel investigational drugsVariable clinical presentationEvent end pointsHemoglobin thresholdHematologic recoveryCount recoveryClinical presentationClinical benefitMyelodysplastic syndromeIWG criteriaMyelodysplastic neoplasmsConsensus recommendationsInvestigational drugsNew agents
2022
Evaluating complete remission with partial hematologic recovery (CRh) as a response criterion in myelodysplastic syndromes (MDS)
Brunner A, Gavralidis A, Ali N, Hunter A, Komrokji R, Zeidan A, Sallman D. Evaluating complete remission with partial hematologic recovery (CRh) as a response criterion in myelodysplastic syndromes (MDS). Blood Cancer Journal 2022, 12: 153. PMID: 36379923, PMCID: PMC9666661, DOI: 10.1038/s41408-022-00748-9.Peer-Reviewed Original ResearchConceptsPartial hematologic recoveryMyelodysplastic syndromeHematologic recoveryResponse criteriaCR/CRhIWG 2006 criteriaDuration of therapyBest overall responseTime of therapyCR responseCRH responseDNMTi therapyOS associationComplete remissionMedian OSOverall survivalAdult patientsAllogeneic transplantsMedian ageMDS patientsMultivariable analysisClinical trialsSimilar survivalPatientsTherapyA phase 1b study of venetoclax and azacitidine combination in patients with relapsed or refractory myelodysplastic syndromes
Zeidan AM, Borate U, Pollyea DA, Brunner AM, Roncolato F, Garcia JS, Filshie R, Odenike O, Watson AM, Krishnadasan R, Bajel A, Naqvi K, Zha J, Cheng W, Zhou Y, Hoffman D, Harb JG, Potluri J, Garcia‐Manero G. A phase 1b study of venetoclax and azacitidine combination in patients with relapsed or refractory myelodysplastic syndromes. American Journal Of Hematology 2022, 98: 272-281. PMID: 36309981, PMCID: PMC10100228, DOI: 10.1002/ajh.26771.Peer-Reviewed Original ResearchConceptsMedian overall survivalMyelodysplastic syndromeOverall survivalTransfusion independenceHematological improvementTherapy failureHigh-risk myelodysplastic syndromeInternational Working Group criteriaHematological adverse eventsPhase 1b studyRefractory myelodysplastic syndromeStandard of careEfficacy of venetoclaxEffective therapeutic strategyFebrile neutropeniaMarrow CROral venetoclaxComplete remissionAdverse eventsMedian durationAzacitidine treatmentMedian timeMarrow responseMulticenter studyGroup criteriaImpact of pre-transplant induction and consolidation cycles on AML allogeneic transplant outcomes: a CIBMTR analysis in 3113 AML patients
Boyiadzis M, Zhang M, Chen K, Abdel-Azim H, Abid M, Aljurf M, Bacher U, Badar T, Badawy S, Battiwalla M, Bejanyan N, Bhatt V, Brown V, Castillo P, Cerny J, Copelan E, Craddock C, Dholaria B, Perez M, Ebens C, Gale R, Ganguly S, Gowda L, Grunwald M, Hashmi S, Hildebrandt G, Iqbal M, Jamy O, Kharfan-Dabaja M, Khera N, Lazarus H, Lin R, Modi D, Nathan S, Nishihori T, Patel S, Pawarode A, Saber W, Sharma A, Solh M, Wagner J, Wang T, Williams K, Winestone L, Wirk B, Zeidan A, Hourigan C, Litzow M, Kebriaei P, de Lima M, Page K, Weisdorf D. Impact of pre-transplant induction and consolidation cycles on AML allogeneic transplant outcomes: a CIBMTR analysis in 3113 AML patients. Leukemia 2022, 37: 1006-1017. PMID: 36310182, PMCID: PMC10148918, DOI: 10.1038/s41375-022-01738-3.Peer-Reviewed Original ResearchConceptsPrimary induction failureAllo-HCTRIC allo-HCTComplete remissionOverall survivalConsolidation therapyAML patientsDetectable MRDRelapse riskInduction cyclesAllogeneic hematopoietic cell transplantationConsolidation cyclesPre-transplant inductionFirst complete remissionAdult AML patientsReduced-intensity conditioningBetter overall survivalHematopoietic cell transplantationRisk of relapseAllogeneic transplant outcomesCIBMTR analysisImproved OSInduction failureTransplant outcomesCell transplantation
2021
Venetoclax and Azacitidine in the Treatment of Patients with Relapsed/Refractory Myelodysplastic Syndrome
Zeidan A, Borate U, Pollyea D, Brunner A, Roncolato F, Garcia J, Filshie R, Odenike O, Watson A, Krishnadasan R, Bajel A, Naqvi K, Zha J, Hogdal L, Zhou Y, Hoffman D, Kye S, Garcia-Manero G. Venetoclax and Azacitidine in the Treatment of Patients with Relapsed/Refractory Myelodysplastic Syndrome. Blood 2021, 138: 537. DOI: 10.1182/blood-2021-145646.Peer-Reviewed Original ResearchClinical Trials CommitteeHigh-risk myelodysplastic syndromeMedian overall survivalAdverse eventsMyelodysplastic syndromeTrials CommitteeOverall survivalMedian timeClinical trialsFebrile neutropeniaPrior therapyComplete remissionTransfusion independenceClinical outcomesBcl-2 inhibitorsMyeloid leukemiaEastern Cooperative Oncology Group performance statusGrade treatment-emergent adverse eventsInternational Working Group 2006 criteriaOverall median progression-free survivalIncomplete blood count recoveryMedian progression-free survivalOral BCL-2 inhibitorTreatment-emergent adverse eventsAllogeneic hematopoietic stem cellsEvaluating Complete Remission with Incomplete Hematologic Recovery (CRh) As a Response Criterion in Myelodysplastic Syndromes (MDS)
Brunner A, Gavralidis A, Al Ali N, Komrokji R, Zeidan A, Sallman D. Evaluating Complete Remission with Incomplete Hematologic Recovery (CRh) As a Response Criterion in Myelodysplastic Syndromes (MDS). Blood 2021, 138: 1522. DOI: 10.1182/blood-2021-151815.Peer-Reviewed Original ResearchClinical Trials CommitteeStable diseaseComplete remissionHMA therapyBone marrow blastsHematologic improvementProgressive diseaseMyelodysplastic syndromePartial remissionTrials CommitteeResponse criteriaMarrow blastsOverall survivalSpeakers bureauHigh-risk myelodysplastic syndromeAdvisory CommitteeIncomplete hematologic recoveryIWG 2006 criteriaMethod of KaplanRisk myelodysplastic syndromesOngoing prospective studyMoffitt Cancer CenterBest overall responsePrediction of OSMassachusetts General HospitalPhase 3 VERONA study of venetoclax with azacitidine to assess change in complete remission and overall survival in treatment-naïve higher-risk myelodysplastic syndromes.
Zeidan A, Garcia J, Fenaux P, Platzbecker U, Miyazaki Y, Xiao Z, Zhou Y, Naqvi K, Kye S, Garcia-Manero G. Phase 3 VERONA study of venetoclax with azacitidine to assess change in complete remission and overall survival in treatment-naïve higher-risk myelodysplastic syndromes. Journal Of Clinical Oncology 2021, 39: tps7054-tps7054. DOI: 10.1200/jco.2021.39.15_suppl.tps7054.Peer-Reviewed Original ResearchHematopoietic stem cell transplantOverall survivalAcute myeloid leukemiaCR rateComplete remissionTransfusion independenceCell transplantationDisease progressionMyeloid leukemiaDay 1B-cell lymphoma-2 inhibitorRed blood cell transfusion independenceHigh-risk myelodysplastic syndromeAllogenic stem cell transplantationCo-morbid patientsIWG 2006 criteriaPhase 1b studyPlatelet transfusion independenceMedian overall survivalFirst-line treatmentPhase 3 studyBone marrow blastsDe novo patientsHematopoietic cell transplantationStudy days 1