2024
Targeted therapies for myelodysplastic syndromes/neoplasms (MDS): current landscape and future directions
Bidikian A, Bewersdorf J, Shallis R, Getz T, Stempel J, Kewan T, Stahl M, Zeidan A. Targeted therapies for myelodysplastic syndromes/neoplasms (MDS): current landscape and future directions. Expert Review Of Anticancer Therapy 2024, 24: 1131-1146. PMID: 39367718, DOI: 10.1080/14737140.2024.2414071.Peer-Reviewed Original ResearchErythropoiesis-stimulating agentsTargeted therapyLR-MDSHR-MDSHypoxia-inducible factorAllogeneic hematopoietic stem cell transplantationLandscape of targeted therapiesHematopoietic stem cell transplantationHeterogeneous group of hematologic malignanciesGroup of hematologic malignanciesMolecular prognostic toolsDuration of responseStem cell transplantationTrial designClinical trial designHypomethylating agentsCell transplantationHematologic malignanciesImprove patient outcomesRNA splicing machineryImmune evasionPrognostic toolTGF-betaTherapyEffective treatmentMDS-167 Multilineage and Safety Results From the COMMANDS Trial in Transfusion-Dependent (TD), Erythropoiesis-Stimulating Agent (ESA)-Naive Patients With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS)
Garcia-Manero G, Della Porta M, Santini V, Zeidan A, Komrokji R, Li J, Pilot R, Kreitz S, Pozharskaya V, Keeperman K, Lai Y, Valcarcel D, Fenaux P, Platzbecker U. MDS-167 Multilineage and Safety Results From the COMMANDS Trial in Transfusion-Dependent (TD), Erythropoiesis-Stimulating Agent (ESA)-Naive Patients With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s388. DOI: 10.1016/s2152-2650(24)01347-8.Peer-Reviewed Original ResearchEA-treated patientsHematological improvement-erythroidAbsolute neutrophil countTransfusion-dependentLR-MDSMyelodysplastic syndromeRBC transfusionBaseline medianPlatelet lineageIntermediate-risk myelodysplastic syndromesESA-naiveBone marrow blastsLowered riskMarrow blastsPlatelet transfusionsRinged sideroblastsPlatelet countNeutrophil countLuspaterceptSafety resultsEpoetin alfaHI-NTreatment periodTransfusionPatientsMDS-166 Clinical Benefit of Luspatercept Treatment in Transfusion-Dependent (TD), Erythropoiesis-Stimulating Agent (ESA)-Naive Patients With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) in the COMMANDS Trial
Zeidan A, Platzbecker U, Della Porta M, Santini V, Garcia-Manero G, Li J, Kreitz S, Pozharskaya V, Rose S, Lai Y, Davidárcel D, Fenaux P, Shortt J, Komrokji R. MDS-166 Clinical Benefit of Luspatercept Treatment in Transfusion-Dependent (TD), Erythropoiesis-Stimulating Agent (ESA)-Naive Patients With Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes (MDS) in the COMMANDS Trial. Clinical Lymphoma Myeloma & Leukemia 2024, 24: s387-s388. DOI: 10.1016/s2152-2650(24)01346-6.Peer-Reviewed Original ResearchEA patientsTransfusion-dependentRBC unitsLuspatercept treatmentRBC-TITransfusion burdenMyelodysplastic syndromeLR-MDSRBC transfusionIntermediate-risk myelodysplastic syndromesEA-treated patientsRBC transfusion independenceBone marrow blastsLowered riskTransfusion independenceMarrow blastsTreatment initiationCumulative medianLuspaterceptEpoetin alfaESA-naivePatients adultsEffective treatmentPatientsInterquartile range
2023
The ELEMENT-MDS Trial: A Phase 3 Randomized Study Evaluating Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Non-Transfusion-Dependent, Lower-Risk Myelodysplastic Syndromes
Zeidan A, Komrokji R, Buckstein R, Santini V, Rose S, Malini P, Lew G, Aggarwal D, Keeperman K, Jiang H, Giuseppi A, Zhang J, Cluzeau T, Shortt J, Platzbecker U. The ELEMENT-MDS Trial: A Phase 3 Randomized Study Evaluating Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Non-Transfusion-Dependent, Lower-Risk Myelodysplastic Syndromes. Blood 2023, 142: 6503. DOI: 10.1182/blood-2023-178635.Peer-Reviewed Original ResearchRBC transfusion dependenceErythropoiesis-stimulating agentsLR-MDSEpoetin alfaMyelodysplastic syndromeTransfusion dependenceSecondary endpointsStarting doseOverall survivalTransfusion independenceWeek 1Baseline serum erythropoietin levelsIntermediate-risk myelodysplastic syndromesIPSS-R risk categoryLower-risk myelodysplastic syndromesRed blood cell transfusionHigh-risk myelodysplastic syndromeInternational Prognostic Scoring SystemAdditional secondary endpointsRBC transfusion independenceTransfusion-free survivalKey secondary endpointBlood cell transfusionPatient Global ImpressionPhase 3 studyImpact of Genomic Landscape and Mutational Burden on Primary Endpoint Responses in the COMMANDS Study
Komrokji R, Guerrero M, Garcia-Manero G, Zeidan A, Platzbecker U, Hayati S, Vodala S. Impact of Genomic Landscape and Mutational Burden on Primary Endpoint Responses in the COMMANDS Study. Blood 2023, 142: 4591. DOI: 10.1182/blood-2023-178689.Peer-Reviewed Original ResearchErythropoiesis-stimulating agentsSuperior clinical benefitLR-MDSClinical benefitSimilar response ratesResponse rateRisk groupsMutational burdenGene mutationsRing sideroblastsRed blood cell transfusionInternational Prognostic Scoring SystemShorter leukemia-free survivalBaseline erythropoietin levelsComparable clinical benefitFavorable clinical benefitBlood cell transfusionLeukemia-free survivalProgression-free survivalSimilar clinical benefitsPrimary endpoint analysisPrognostic scoring systemBone marrow samplesSignificant differencesDriver gene mutations
2019
Clinical Effectiveness of Hypomethylating Agents (HMAs) and Lenalidomide (Len) in Older Patients (pts) with Refractory Anemia with Ring Sideroblasts: A Large Population-Based Study in the United States (US)
Wang X, Wang R, Zhang C, Zeidan A, Podoltsev N, Huntington S, Gore S, Davidoff A, Ma X. Clinical Effectiveness of Hypomethylating Agents (HMAs) and Lenalidomide (Len) in Older Patients (pts) with Refractory Anemia with Ring Sideroblasts: A Large Population-Based Study in the United States (US). Blood 2019, 134: 4748. DOI: 10.1182/blood-2019-128821.Peer-Reviewed Original ResearchPopulation-based studyLR-MDSMedian overall survivalOverall survivalClinical effectivenessHypomethylating agentCelgene CorporationMedian durationRed blood cellsMedian timeRing sideroblastsBoehringer IngelheimAdvisory CommitteeLarge population-based studyDaiichi SankyoOlder adultsAbnormal erythroid precursorsTransfusion independence rateEnd Results-MedicareHigh-risk MDSErythropoiesis-stimulating agentsKaplan-Meier statisticsComparative clinical effectivenessEnd of studyPart D coverageRBC 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 oddsTransfusionPatients
2018
Transfusion Independence in Lower-Risk, Non-del5(q) Myelodysplastic Syndromes (LR-MDS) Among Patients (pts) Initiating Hypomethylating Agents (HMAs) While Receiving Red Blood Cell (RBC) Transfusions
Zeidan A, Zhu W, Wang R, Stahl M, Huntington S, Giri S, Podoltsev N, Gore S, Ma X, Davidoff A. Transfusion Independence in Lower-Risk, Non-del5(q) Myelodysplastic Syndromes (LR-MDS) Among Patients (pts) Initiating Hypomethylating Agents (HMAs) While Receiving Red Blood Cell (RBC) Transfusions. Blood 2018, 132: 838. DOI: 10.1182/blood-2018-99-116497.Peer-Reviewed Original ResearchRBC transfusion independenceHMA initiationTransfusion independenceTransfusion dependenceLR-MDSClinical trial dataHypomethylating agentTransfusion statusMedian timeMedian TI durationCox modelContinuous Medicare Parts ATrial dataRed blood cell transfusionDiagnosis of MDSRBC transfusion dependenceSubset of ptsBlood cell transfusionEnd Results-MedicareErythropoiesis-stimulating agentsHigh-risk diseaseMultivariate Cox modelTreatment of MDSEnd of studyTI duration
2017
Management of lower-risk myelodysplastic syndromes without del5q: current approach and future trends
Stahl M, Zeidan AM. Management of lower-risk myelodysplastic syndromes without del5q: current approach and future trends. Expert Review Of Hematology 2017, 10: 345-364. PMID: 28277851, DOI: 10.1080/17474086.2017.1297704.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsErythropoiesis stimulating agentsMyelodysplastic syndromeLR-MDSImmunosuppressive therapyTreatment modalitiesLower-risk myelodysplastic syndromesTGF-β pathway inhibitorCombination treatment modalitiesLR-MDS patientsPromising investigational agentsSubset of patientsAvailable therapeutic modalitiesGoals of careStandard therapeutic optionPredictors of responseCurrent treatment modalitiesAcute myeloid leukemiaQuality of lifeBone marrow failureRisk assessment toolSymptom controlBlood cytopeniasInvestigational agentsTherapeutic optionsClinical behavior
2015
Comparing the prognostic value of risk stratifying models for patients with lower‐risk myelodysplastic syndromes: Is one model better?
Zeidan AM, Sekeres MA, Wang X, Al Ali N, Garcia‐Manero G, Steensma DP, Roboz G, Barnard J, Padron E, DeZern A, Maciejewski JP, List AF, Komrokji RS, Consortium O. Comparing the prognostic value of risk stratifying models for patients with lower‐risk myelodysplastic syndromes: Is one model better? American Journal Of Hematology 2015, 90: 1036-1040. PMID: 26284571, DOI: 10.1002/ajh.24173.Peer-Reviewed Original ResearchConceptsInternational Prognostic Scoring SystemIPSS-R categoryLR-MDSHarrell's C-indexOverall survivalC-indexLower-risk myelodysplastic syndromesRevised International Prognostic Scoring SystemActual overall survivalMedian overall survivalVaried survival outcomesKaplan-Meier methodPrognostic scoring systemDisease-modifying therapiesCategory 3Eligible patientsPoor OSPrognostic utilitySurvival outcomesMyelodysplastic syndromePrognostic valueLarge cohortPatientsEarly interventionScoring systemComparing the Prognostic Value of Risk stratifying Models for Patients with Lower‐Risk Myelodysplastic Syndromes: Is one model better?
Zeidan A, Sekeres M, Wang X, Al Ali N, Garcia‐Manero G, Steensma D, Roboz G, Barnard J, Padron E, Dezern A, Maciejewski J, List A, Komrokji R. Comparing the Prognostic Value of Risk stratifying Models for Patients with Lower‐Risk Myelodysplastic Syndromes: Is one model better? American Journal Of Hematology 2015 PMCID: PMC4751065, DOI: 10.1002/ajh.24170.Peer-Reviewed Original ResearchInternational Prognostic Scoring SystemIPSS-R categoryLR-MDSHarrell's C-indexOverall survivalC-indexLower-risk myelodysplastic syndromesRevised International Prognostic Scoring SystemActual overall survivalMedian overall survivalVaried survival outcomesKaplan-Meier methodPrognostic scoring systemDisease-modifying therapiesCategory 3Eligible patientsPoor OSPrognostic utilitySurvival outcomesMyelodysplastic syndromePrognostic valueLarge cohortPatientsEarly interventionScoring system