2023
STIMULUS-MDS2 design and rationale: a phase III trial with the anti-TIM-3 sabatolimab (MBG453) + azacitidine in higher risk MDS and CMML-2
Zeidan A, Giagounidis A, Sekeres M, Xiao Z, Sanz G, Van Hoef M, Ma F, Hertle S, Santini V. STIMULUS-MDS2 design and rationale: a phase III trial with the anti-TIM-3 sabatolimab (MBG453) + azacitidine in higher risk MDS and CMML-2. Future Oncology 2023, 19: 631-642. PMID: 37083373, DOI: 10.2217/fon-2022-1237.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAntimetabolites, AntineoplasticAzacitidineClinical Trials, Phase III as TopicHumansLeukemia, Myeloid, AcuteLeukemia, Myelomonocytic, ChronicMyelodysplastic SyndromesConceptsHigh-risk myelodysplastic syndromeChronic myelomonocytic leukemiaMyelodysplastic syndromeCMML-2Tim-3Hematopoietic stem cell transplantationT-cell immunoglobulin domainMucin domain 3Risk myelodysplastic syndromesPhase III trialsStem cell transplantationLeukemic stem cellsFavorable tolerabilityIII trialsNovel immunotherapiesPoor outcomeCell transplantationLeukemic blastsClinical trialsNovel therapiesMyelomonocytic leukemiaDurable benefitImmune systemMyeloid malignanciesMeaningful improvements
2022
Prognostic implications of mono-hit and multi-hit TP53 alterations in patients with acute myeloid leukemia and higher risk myelodysplastic syndromes treated with azacitidine-based therapy
Zeidan A, Bewersdorf J, Hasle V, Shallis R, Thompson E, de Menezes D, Rose S, Boss I, Halene S, Haferlach T, Fox B. Prognostic implications of mono-hit and multi-hit TP53 alterations in patients with acute myeloid leukemia and higher risk myelodysplastic syndromes treated with azacitidine-based therapy. Leukemia 2022, 37: 240-243. PMID: 36437356, DOI: 10.1038/s41375-022-01766-z.Peer-Reviewed Original ResearchMeSH KeywordsAntimetabolites, AntineoplasticAzacitidineHumansLeukemia, Myeloid, AcuteMyelodysplastic SyndromesPrognosisTumor Suppressor Protein p53Prospective comparison of outcomes with azacitidine and decitabine in patients with AML ineligible for intensive chemotherapy
Zeidan AM, Fenaux P, Gobbi M, Mayer J, Roboz GJ, Krauter J, Robak T, Kantarjian HM, Novák J, Jedrzejczak WW, Thomas X, Ojeda-Uribe M, Miyazaki Y, Min YH, Yeh SP, Brandwein JM, Gercheva L, Demeter J, Griffiths EA, Yee KWL, Issa JJ, Bewersdorf JP, Keer H, Hao Y, Azab M, Döhner H. Prospective comparison of outcomes with azacitidine and decitabine in patients with AML ineligible for intensive chemotherapy. Blood 2022, 140: 285-289. PMID: 35507690, PMCID: PMC9305088, DOI: 10.1182/blood.2022015832.Peer-Reviewed Original ResearchMeSH KeywordsAntimetabolites, AntineoplasticAntineoplastic Combined Chemotherapy ProtocolsAzacitidineDecitabineHumansLeukemia, Myeloid, AcuteTreatment OutcomeImpact of Hypomethylating Agent Use on Hospital and Emergency Room Visits, and Predictors of Early Discontinuation in Patients With Higher-Risk Myelodysplastic Syndromes
Zeidan AM, Joshi N, Kale H, Wang WJ, Corman S, Salimi T, Epstein RS. Impact of Hypomethylating Agent Use on Hospital and Emergency Room Visits, and Predictors of Early Discontinuation in Patients With Higher-Risk Myelodysplastic Syndromes. Clinical Lymphoma Myeloma & Leukemia 2022, 22: 670-679. PMID: 35614009, DOI: 10.1016/j.clml.2022.04.016.Peer-Reviewed Original ResearchMeSH KeywordsAgedAntimetabolites, AntineoplasticAzacitidineDecitabineDNA MethylationEmergency Service, HospitalHospitalsHumansMedicareMyelodysplastic SyndromesTreatment OutcomeUnited StatesConceptsHigh-risk myelodysplastic syndromeHMA therapyPoor performance statusRate of hospitalizationSEER-Medicare databaseMyelodysplastic syndromeER visitsEarly discontinuationPerformance statusEmergency roomOlder agePredictors of discontinuationEmergency room visitsHigh-risk groupHigh economic burdenTreatment discontinuationExcess blastsMore hospitalizationsNew hospitalizationRefractory anemiaRoom visitsHigh riskDiscontinuationMD diagnosisAgent use
2021
Enasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial
DiNardo CD, Schuh AC, Stein EM, Montesinos P, Wei AH, de Botton S, Zeidan AM, Fathi AT, Kantarjian HM, Bennett JM, Frattini MG, Martin-Regueira P, Lersch F, Gong J, Hasan M, Vyas P, Döhner H. Enasidenib plus azacitidine versus azacitidine alone in patients with newly diagnosed, mutant-IDH2 acute myeloid leukaemia (AG221-AML-005): a single-arm, phase 1b and randomised, phase 2 trial. The Lancet Oncology 2021, 22: 1597-1608. PMID: 34672961, DOI: 10.1016/s1470-2045(21)00494-0.Peer-Reviewed Original ResearchMeSH KeywordsAgedAminopyridinesAntimetabolites, AntineoplasticAntineoplastic Combined Chemotherapy ProtocolsAzacitidineDrug Administration ScheduleDrug-Related Side Effects and Adverse ReactionsFemaleHumansIsocitrate DehydrogenaseLeukemia, Myeloid, AcuteMaleMutationProgression-Free SurvivalRandom AllocationTreatment OutcomeTriazinesConceptsAcute myeloid leukemiaSerious treatment-related adverse eventsTreatment-related adverse eventsDose-finding portionOverall response rateMyeloid leukemiaAdverse eventsFebrile neutropeniaCombination groupInterim analysisEastern Cooperative Oncology Group performance statusCommon treatment-related grade 3Response rateInteractive web response systemTreatment-related grade 3Phase 1b/2 trialPrespecified interim analysisTreatment-related deathsPhase 2 trialWeb response systemPhase 2Acute myeloid leukemia subtypesPhase 2 portionBristol-Myers SquibbAzacitidine monotherapyInjectable Hypomethylating Agents for Management of Myelodysplastic Syndromes: Patients’ Perspectives on Treatment
Zeidan AM, Jayade S, Schmier J, Botteman M, Hassan A, Ruiters D, Hill K, Joshi N. Injectable Hypomethylating Agents for Management of Myelodysplastic Syndromes: Patients’ Perspectives on Treatment. Clinical Lymphoma Myeloma & Leukemia 2021, 22: e185-e198. PMID: 34674983, DOI: 10.1016/j.clml.2021.09.009.Peer-Reviewed Original ResearchMeSH KeywordsAdultAntimetabolites, AntineoplasticAzacitidineCross-Sectional StudiesFemaleHumansMiddle AgedMyelodysplastic SyndromesRisk FactorsTreatment OutcomeConceptsMDS patientsAdult MDS patientsMedian patient agePain/discomfortMost MDS patientsOnline cross-sectional surveyDaily activitiesCross-sectional surveyPatient advocacy groupsPatient ageMyelodysplastic syndromeSC administrationSubcutaneous administrationTreatment administrationSC therapyTreatment challengesPatient's perspectiveHypomethylating agentRisk scoreHMA treatmentMD treatmentPatientsAdministrationAzacitidinePain
2019
Association of provider experience and clinical outcomes in patients with myelodysplastic syndromes receiving hypomethylating agents
Zeidan AM, Hu X, Zhu W, Stahl M, Wang R, Huntington SF, Giri S, Bewersdorf JP, Podoltsev NA, Gore SD, Ma X, Davidoff AJ. Association of provider experience and clinical outcomes in patients with myelodysplastic syndromes receiving hypomethylating agents. Leukemia & Lymphoma 2019, 61: 397-408. PMID: 31570040, PMCID: PMC7732188, DOI: 10.1080/10428194.2019.1663423.Peer-Reviewed Original ResearchMeSH KeywordsAgedAntimetabolites, AntineoplasticHumansMedicareMyelodysplastic SyndromesProportional Hazards ModelsUnited StatesConceptsMyelodysplastic syndromeOverall survivalHMA cyclesHMA therapyProvider experienceCox proportional hazards modelOlder MDS patientsMedian overall survivalPercent of patientsSEER-Medicare dataMultivariate logistic regressionProportional hazards modelHMA initiationClinical outcomesMDS patientsClinical trialsMedian numberHMA treatmentHazards modelPatientsSignificant associationLogistic regressionPopulation-level survivalSurvivalSyndromeRBC transfusion independence among lower risk MDS patients receiving hypomethylating agents: a population-level analysis
Zeidan AM, Zhu W, Stahl M, Wang R, Huntington SF, Giri S, Podoltsev NA, Gore SD, Ma X, Davidoff AJ. RBC transfusion independence among lower risk MDS patients receiving hypomethylating agents: a population-level analysis. Leukemia & Lymphoma 2019, 60: 3181-3187. PMID: 31170846, DOI: 10.1080/10428194.2019.1622700.Peer-Reviewed Original ResearchConceptsRBC transfusion independenceLR-MDS patientsTransfusion independenceHMA initiationRBC transfusionClinical effectivenessReal-life clinical effectivenessRed blood cell transfusionLower-risk myelodysplastic syndromesLow-risk MDS patientsRisk MDS patientsBlood cell transfusionRisk myelodysplastic syndromesHMA therapyLR-MDSCell transfusionMost patientsDisease courseMDS patientsMedicare databaseMyelodysplastic syndromePopulation-level estimatesLower oddsTransfusionPatientsTreatment sequence of lenalidomide and hypomethylating agents and the impact on clinical outcomes for patients with myelodysplastic syndromes
Zeidan AM, Klink AJ, McGuire M, Feinberg B. Treatment sequence of lenalidomide and hypomethylating agents and the impact on clinical outcomes for patients with myelodysplastic syndromes. Leukemia & Lymphoma 2019, 60: 2050-2055. PMID: 30636526, DOI: 10.1080/10428194.2018.1551538.Peer-Reviewed Original ResearchConceptsMyelodysplastic syndromeFirst-line useLonger treatment durationAgent azacitidineTreatment discontinuationClinical outcomesMedian timeInsurance disenrollmentUS payersTreatment durationLenalidomidePatientsTreatment sequenceOptimal sequencingSyndromeSurvivalOutcomesDiscontinuationAzacitidineDecitabineDisenrollment
2018
Conviction in the face of affliction: a case series of Jehovah’s Witnesses with myeloid malignancies
Shallis RM, Xu ML, Curtis SA, Medoff E, Mixon R, Folkers A, Zeidan AM. Conviction in the face of affliction: a case series of Jehovah’s Witnesses with myeloid malignancies. Annals Of Hematology 2018, 97: 2245-2248. PMID: 30088044, DOI: 10.1007/s00277-018-3459-6.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsHypomethylating agents in relapsed and refractory AML: outcomes and their predictors in a large international patient cohort
Stahl M, DeVeaux M, Montesinos P, Itzykson R, Ritchie EK, Sekeres MA, Barnard JD, Podoltsev NA, Brunner AM, Komrokji RS, Bhatt VR, Al-Kali A, Cluzeau T, Santini V, Fathi AT, Roboz GJ, Fenaux P, Litzow MR, Perreault S, Kim TK, Prebet T, Vey N, Verma V, Germing U, Bergua JM, Serrano J, Gore SD, Zeidan AM. Hypomethylating agents in relapsed and refractory AML: outcomes and their predictors in a large international patient cohort. Blood Advances 2018, 2: 923-932. PMID: 29685952, PMCID: PMC5916007, DOI: 10.1182/bloodadvances.2018016121.Peer-Reviewed Original ResearchConceptsOverall survivalRR-AMLMedian OSOlder acute myeloid leukemia patientsAcute myeloid leukemia patientsCR/CRiIncomplete count recoveryMedian overall survivalDecreased overall survivalBone marrow blastsReasonable therapeutic optionMyeloid leukemia patientsPredictors of responseImproved response ratesLarge international patient cohortInternational patient cohortGood responseComplete remissionHematologic improvementRefractory AMLMarrow blastsMedian ageCount recoveryFrontline treatmentTherapeutic optionsAllogeneic Hematopoietic Stem Cell Transplantation Following the Use of Hypomethylating Agents among Patients with Relapsed or Refractory AML: Findings from an International Retrospective Study
Stahl M, DeVeaux M, Montesinos P, Itzykson R, Ritchie EK, Sekeres MA, Majhail N, Barnard J, Podoltsev NA, Brunner AM, Komrokji RS, Bhatt VR, Al-Kali A, Cluzeau T, Santini V, Roboz GJ, Fenaux P, Litzow M, Fathi AT, Perreault S, Kim TK, Prebet T, Vey N, Verma V, Kobbe G, Bergua J, Serrano J, Gore SD, Zeidan AM. Allogeneic Hematopoietic Stem Cell Transplantation Following the Use of Hypomethylating Agents among Patients with Relapsed or Refractory AML: Findings from an International Retrospective Study. Transplantation And Cellular Therapy 2018, 24: 1754-1758. PMID: 29649620, DOI: 10.1016/j.bbmt.2018.03.025.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationHMA therapyStem cell transplantationComplete remissionCell transplantationUnrelated hematopoietic stem cell transplantationInternational retrospective studyLimited treatment optionsAcute myeloid leukemiaChronic GVHDMedian OSPrimary refractoryRR-AMLConditioning regimenRefractory AMLPatients patientsUnrelated donorsEntire cohortPoor prognosisRetrospective studyTreatment optionsMyeloid leukemiaInternational cohortHypomethylating agent
2017
Hypomethylating agents in myelodysplastic syndromes and population-level outcomes: a changing landscape or a small dent?
Zeidan AM. Hypomethylating agents in myelodysplastic syndromes and population-level outcomes: a changing landscape or a small dent? Leukemia & Lymphoma 2017, 59: 1030-1032. PMID: 28952833, DOI: 10.1080/10428194.2017.1382700.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAntimetabolites, AntineoplasticAzacitidineDNA MethylationHumansMyelodysplastic SyndromesA call for action: Increasing enrollment of untreated patients with higher‐risk myelodysplastic syndromes in first‐line clinical trials
Zeidan AM, Stahl M, Sekeres MA, Steensma DP, Komrokji RS, Gore SD. A call for action: Increasing enrollment of untreated patients with higher‐risk myelodysplastic syndromes in first‐line clinical trials. Cancer 2017, 123: 3662-3672. PMID: 28759108, DOI: 10.1002/cncr.30903.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAntimetabolites, AntineoplasticAzacitidineClinical Trials as TopicDecitabineHumansMyelodysplastic SyndromesPatient SelectionReproducibility of ResultsSurvival AnalysisTreatment FailureConceptsHigh-risk myelodysplastic syndromeAZA-001 trialClinical trialsMinimal survival gainsLarge randomized trialsManagement of patientsPopulation-based studyClinical trial enrollmentQuality of lifeMonotherapy armMedian survivalRandomized trialsSurvival impactTrial enrollmentSurvival gainMyelodysplastic syndromeRegistry analysisAggressive malignancyPatientsNatural historyRoutine useTrialsSurvivalEnrollmentHMAsHypomethylating agent therapy use and survival in older patients with chronic myelomonocytic leukemia in the United States: A large population‐based study
Zeidan AM, Hu X, Long JB, Wang R, Ma X, Podoltsev NA, Huntington SF, Gore SD, Davidoff AJ. Hypomethylating agent therapy use and survival in older patients with chronic myelomonocytic leukemia in the United States: A large population‐based study. Cancer 2017, 123: 3754-3762. PMID: 28621841, PMCID: PMC6540984, DOI: 10.1002/cncr.30814.Peer-Reviewed Original ResearchConceptsChronic myelomonocytic leukemiaRisk of deathSupportive careEnd Results-Medicare databaseOlder adultsMedian OS timeOverall survival benefitCohort of patientsProportional hazards modelUse of HMAsMedian OSAgent therapySurvival benefitOS timeCMML patientsMyelomonocytic leukemiaHazards modelHMA treatmentPatientsTemporal improvementSecondary analysisPropensity scoreLimited evidenceSurvival changesApprovalLenalidomide use in myelodysplastic syndromes: Insights into the biologic mechanisms and clinical applications
Stahl M, Zeidan AM. Lenalidomide use in myelodysplastic syndromes: Insights into the biologic mechanisms and clinical applications. Cancer 2017, 123: 1703-1713. PMID: 28192601, DOI: 10.1002/cncr.30585.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAnemiaAntimetabolites, AntineoplasticAzacitidineChromosomes, Human, Pair 5Disease ProgressionHumansImmunologic FactorsLenalidomideLeukemia, Myeloid, AcuteMyelodysplastic SyndromesThalidomideTreatment OutcomeConceptsAcute myeloid leukemiaProlong survivalLow-risk MDSLR-MDS patientsEfficacy of lenalidomideImpressive clinical activityRecent clinical dataClonal myeloid neoplasmsMechanism of actionBlood cytopeniasConventional careTransfusion independenceCytogenetic responseClinical activityMyeloid leukemiaClinical dataIneffective hematopoiesisMyeloid neoplasmsDelay progressionHigh riskLenalidomidePatientsVariable riskHeterogeneous groupProgression
2016
Differential response to hypomethylating agents based on sex: a report on behalf of the MDS Clinical Research Consortium (MDS CRC)*
DeZern AE, Zeidan AM, Barnard J, Hand W, Al Ali N, Brown F, Zimmerman C, Roboz GJ, Garcia-Manero G, Steensma DP, Komrokji RS, Sekeres MA. Differential response to hypomethylating agents based on sex: a report on behalf of the MDS Clinical Research Consortium (MDS CRC)*. Leukemia & Lymphoma 2016, 58: 1325-1331. PMID: 27774847, PMCID: PMC5394924, DOI: 10.1080/10428194.2016.1246726.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overAntimetabolites, AntineoplasticAzacitidineDecitabineDisease ProgressionDNA MethylationFemaleHumansMaleMiddle AgedMyelodysplastic SyndromesSex FactorsTreatment OutcomeConceptsHigh-risk myelodysplastic syndromeMyelodysplastic syndromeOverall survivalFemale patientsMDS Clinical Research ConsortiumLonger median OSFirst-line therapyClinical Research ConsortiumMedian OSBetter OSPatient sexCytidine deaminase activityPatientsResponse rateDifferential response ratesSignificant differencesSexResearch ConsortiumDeaminase activityDifferential responseBiological reasonsSyndromeTherapyDecitabineDifferencesComparative clinical effectiveness of azacitidine versus decitabine in older patients with myelodysplastic syndromes
Zeidan AM, Davidoff AJ, Long JB, Hu X, Wang R, Ma X, Gross CP, Abel GA, Huntington SF, Podoltsev NA, Hajime U, Prebet T, Gore SD. Comparative clinical effectiveness of azacitidine versus decitabine in older patients with myelodysplastic syndromes. British Journal Of Haematology 2016, 175: 829-840. PMID: 27650975, DOI: 10.1111/bjh.14305.Peer-Reviewed Original ResearchMeSH KeywordsAgedAged, 80 and overAnemia, Refractory, with Excess of BlastsAntimetabolites, AntineoplasticAzacitidineDecitabineFemaleHumansKaplan-Meier EstimateMaleMyelodysplastic SyndromesSurvival RateTreatment OutcomeConceptsMyelodysplastic syndromeRAEB patientsMedian survivalClinical trialsMultivariate Cox proportional hazards modelCox proportional hazards modelKaplan-Meier methodPopulation-based survivalSignificant survival differenceComparative clinical effectivenessProportional hazards modelAgent azacitidineHMA initiationExcess blastsOlder patientsRandomized trialsHistological subtypesRefractory anemiaClinical effectivenessSurvival differencesSubset analysisSurvival advantageHazards modelPatientsDecitabine
2015
Azacitidine with or without Entinostat for the treatment of therapy‐related myeloid neoplasm: further results of the E1905 North American Leukemia Intergroup study
Prebet T, Sun Z, Ketterling RP, Zeidan A, Greenberg P, Herman J, Juckett M, Smith MR, Malick L, Paietta E, Czader M, Figueroa M, Gabrilove J, Erba HP, Tallman MS, Litzow M, Gore SD, intergroup T. Azacitidine with or without Entinostat for the treatment of therapy‐related myeloid neoplasm: further results of the E1905 North American Leukemia Intergroup study. British Journal Of Haematology 2015, 172: 384-391. PMID: 26577691, PMCID: PMC4794257, DOI: 10.1111/bjh.13832.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overAntimetabolites, AntineoplasticAntineoplastic Combined Chemotherapy ProtocolsAzacitidineBenzamidesDrug Administration ScheduleFemaleHumansKaplan-Meier EstimateLeukemia, Myeloid, AcuteMaleMiddle AgedMyelodysplastic SyndromesNeoplasms, Second PrimaryPyridinesTreatment OutcomeConceptsTherapy-related myeloid neoplasmsMyeloid neoplasmsDe novo MDS/AMLRandomized phase 2 studyCombination of azacitidineLeukemia intergroup studyMedian overall survivalPhase 2 studyMDS/AMLSerious late effectsHistone deacetylase inhibitor entinostatAZA monotherapyCombination armNormalization rateOverall survivalIntergroup studyTreatment of cancerPoor responseLate effectsMedian numberAzacitidinePatientsSingle agentResponse rateConventional treatmentThe evolving field of prognostication and risk stratification in MDS: Recent developments and future directions
Lee EJ, Podoltsev N, Gore SD, Zeidan AM. The evolving field of prognostication and risk stratification in MDS: Recent developments and future directions. Blood Reviews 2015, 30: 1-10. PMID: 26119927, DOI: 10.1016/j.blre.2015.06.004.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromeRisk stratificationRecurrent molecular mutationsRisk stratification toolRelevant clinical outcomesPrediction of outcomeRisk/benefit calculationsClinical courseClinical outcomesStratification toolMDS patientsPrognostication toolsIndividual patientsClinical practiceMolecular testingTherapeutic interventionsPatient careTherapeutic selectionPatientsMolecular mutationsIndependent impactPrognosticationBiological heterogeneityOutcomesReliable assay