2024
Phase 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-offOral 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 numberEncouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study
Kontro M, Stein A, Pyörälä M, Rimpiläinen J, Siitonen T, Rannikko J, Mickos J, Turpin R, Hakoniemi M, Berns B, Aakko S, Pawlitzky I, Hollmén M, Zeidan A, Daver N. Encouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study. Blood 2024, 144: 4265. DOI: 10.1182/blood-2024-206804.Peer-Reviewed Original ResearchMedian overall survival estimatesMyelodysplastic syndrome patientsTreatment-emergent adverse eventsMyelodysplastic syndromeBone marrowMarrow CRStable diseaseHematologic improvementPartial responseClever-1HLA-DROutcome of high-risk myelodysplastic syndromeHuman leukocyte antigen-DR isotypePhase 1 dose-escalationResponse of stable diseaseAllogeneic stem cell transplantationHigh-risk myelodysplastic syndromeHigher-risk myelodysplastic syndromesSimon 2-stage designTreatment of myelodysplastic syndromesResponse rateBayesian optimal intervalMedian overall survivalPrimary refractory diseaseRefractory myelodysplastic syndromeHypomethylating 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
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
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 subpopulationsCharacteristics 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 remissionTreatment Patterns and Outcomes of Patients with Acute Myeloid Leukemia (AML) from 2013 to 2022: A Connect ® Myeloid Registry Study
Grinblatt D, Roboz G, Pollyea D, Sekeres M, Scott B, Seiter K, Zeidan A, Patel J, Garcia-Manero G, Komrokji R, Savona M, Degutis I, Kiselev P, Yu E, Heydendael W, Qiu Y, Vergara S, McBride A, Erba H. Treatment Patterns and Outcomes of Patients with Acute Myeloid Leukemia (AML) from 2013 to 2022: A Connect ® Myeloid Registry Study. Blood 2023, 142: 593. DOI: 10.1182/blood-2023-189963.Peer-Reviewed Original ResearchAcute myeloid leukemiaYears of ageMedian overall survivalProportion of patientsOverall survivalRate of transplantGroup 2Group 1Molecular testingTreatment patternsAML cohortMedian (95% CI) OSMyeloid diseasesReal-world clinical practiceWorld Health Organization criteriaIntensive chemotherapy regimensLow-intensity chemotherapyObservational cohort studyOutcomes of patientsFirst-line treatmentKaplan-Meier methodProspective observational studyYear of diagnosisDate of diagnosisTreatment of patientsReclassification of Ascertain (ASTX727-02) Myelodysplastic Syndrome (MDS) Patients: Outcomes Including Clinical Response, Overall Survival (OS), and Leukemia Free Survival (LFS) Based on IPSS-R and IPSS-M Scoring Systems
Garcia-Manero G, McCloskey J, Griffiths E, Zeidan A, Yee K, Al-Kali A, Deeg H, Patel P, Sabloff M, Keating M, Zhu N, Gabrail N, Fazal S, Maly J, Odenike O, Kantarjian H, DeZern A, O'Connell C, Roboz G, Busque L, Buckstein R, Amin H, Randhawa J, Leber B, Lee S, Chan W, Souza S, Sano Y, Keer H, Savona M. Reclassification of Ascertain (ASTX727-02) Myelodysplastic Syndrome (MDS) Patients: Outcomes Including Clinical Response, Overall Survival (OS), and Leukemia Free Survival (LFS) Based on IPSS-R and IPSS-M Scoring Systems. Blood 2023, 142: 4619. DOI: 10.1182/blood-2023-188258.Peer-Reviewed Original ResearchInternational Prognosis Scoring SystemLow-risk MDSHigh-risk MDSLeukemia-free survivalIPSS-R scoreOverall survivalMDS subjectsClinical outcomesPatient outcomesC-indexConcordance indexScoring systemMDS/CMMLMedian overall survivalDifferent risk stratification systemsHarrell's concordance indexMyelodysplastic syndrome patientsHigh-risk populationRisk stratification systemHigh-risk categoryHR categoriesCycle 2Different risk categoriesTreatment discontinuationClinical responsePost 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 studiesValidation 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 HSCTImpact 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 responseValidation 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 Implications of TP53 Mutations/Allelic State in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Treated with Hypomethylating Agents (HMA)- a Multicenter, Retrospective Analysis from the Validate Database
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, Cochran H, Ramaswamy R, Singh A, Roboz G, Rolles B, Wang E, Harris A, Shallis R, Xie Z, Padron E, Maciejewski J, Della Porta M, Komrokji R, Sallman D, Zeidan A. Clinical Implications of TP53 Mutations/Allelic State in Patients (Pts) with Myelodysplastic Syndromes/Neoplasms (MDS) Treated with Hypomethylating Agents (HMA)- a Multicenter, Retrospective Analysis from the Validate Database. Blood 2023, 142: 1002. DOI: 10.1182/blood-2023-186875.Peer-Reviewed Original ResearchOverall response rateMedian overall survivalOverall survivalComplete responseHMA initiationHMA therapyMultivariable Cox proportional hazards regression modelsCox proportional hazards regression modelHigh-risk disease featuresComplex karyotypeProportional hazards regression modelsWorse overall survivalLog-rank testHazards regression modelsSignificant differencesLogistic regression modelsAllogenic HSCTBM biopsyHMA cyclesMDS-EBTherapy initiationMedian ageRegression modelsCR ratePoor outcomeIntensive 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 treatmentIntensive Induction Chemotherapy Vs Hypomethylating Agents + Venetoclax (HMA/VEN) in NPM1-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 Vs Hypomethylating Agents + Venetoclax (HMA/VEN) in NPM1-Mutant Newly Diagnosed Acute Myeloid Leukemia (AML) - a Multicenter Cohort Study. Blood 2023, 142: 2964. DOI: 10.1182/blood-2023-174285.Peer-Reviewed Original ResearchNPM1 mutant acute myeloid leukemiaIntensive induction chemotherapyAcute myeloid leukemiaComposite complete responseMedian overall survivalOverall survivalComplete responseAllo-SCTPt ageAbnormal cytogeneticsCohort studyMyelodysplastic syndromePolymerase chain reactionClinical trialsMyeloid leukemiaNPM1 mutationsLarge multicenter retrospective cohort studyTime-varying covariatesMulticenter retrospective cohort studyAllogeneic stem cell transplantationPrior myelodysplastic syndromeMulticenter cohort studyRetrospective cohort studyStem cell transplantationPrior chemotherapy exposureClinical Outcomes in Patients With Refractory Anemia With Excess Blasts (RAEB) Who Receive Hypomethylating Agents (HMAs)
Zeidan A, Mearns E, Ng C, Shah A, Lamarre N, Yellow-Duke A, Alrawashdh N, Yang B, Cheng W, Bui C, Svensson A. Clinical Outcomes in Patients With Refractory Anemia With Excess Blasts (RAEB) Who Receive Hypomethylating Agents (HMAs). Clinical Lymphoma Myeloma & Leukemia 2023, 24: 177-186. PMID: 37996264, DOI: 10.1016/j.clml.2023.10.010.Peer-Reviewed Original ResearchEvent-free survivalAcute myeloid leukemiaMedian overall survivalOverall survivalHypomethylating agentExcess blastsRefractory anemiaReal-world settingMedian event-free survivalFirst-line therapyHematopoietic cell transplantationEligible patientsClinical outcomesCancer RegistryCell transplantationClinical benefitMedicare databaseClinical effectivenessAML progressionClinical trialsPatient outcomesMyeloid leukemiaPatientsOverall populationSignificant differencesVenetoclax 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 records
2022
A 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 criteriaVenetoclax-based salvage therapy in patients with relapsed/refractory acute myeloid leukemia previously treated with FLT3 or IDH1/2 inhibitors
Bewersdorf JP, Shallis RM, Derkach A, Goldberg AD, Stein A, Stein EM, Marcucci G, Zeidan AM, Shimony S, DeAngelo DJ, Stone RM, Aldoss I, Ball BJ, Stahl M. Venetoclax-based salvage therapy in patients with relapsed/refractory acute myeloid leukemia previously treated with FLT3 or IDH1/2 inhibitors. Leukemia & Lymphoma 2022, 64: 188-196. PMID: 36287540, PMCID: PMC9905301, DOI: 10.1080/10428194.2022.2136952.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaSalvage therapyIDH1/2 inhibitorsIDH2 inhibitorsMyeloid leukemiaResponse rateRefractory acute myeloid leukemiaEffective salvage therapyLow-dose cytarabineMedian overall survivalRetrospective cohort studyOverall response rateLow response rateCohort studyOverall survivalAML patientsTreatment optionsFLT3 inhibitorsITD mutationPatientsTherapyFLT3Little dataVenetoclaxInhibitors