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 HSCTValidation 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 therapyComparison of the Revised 4 Th (2016) and 5 Th (2022) Editions of the World Health Organization (WHO) Classification in a Cohort of Patients with Lower-Risk Myelodysplastic Syndromes/Neoplasms (MDS) - a Glam Registry (REGLAM) Analysis
Iastrebner M, Zeidan A, Arbelbide J, Velloso E, Pereira T, Boada M, Crisp R, Pereyra P, Reyes J, Zappa M, perez-Jacobo F, Dela Peña Celaya J, Moreno E, Abello V, Solano M, Cuervo D, Espinosa D, Casas C, Montoya L, Enrico A, Prates V, Chavez E, Ontiveros-Austria J, Kornblihtt L, Leon A, Toledo V, Negri L, Serrano J, Sánchez A, Rodriguez-Zuñiga A, Stevenazzi M, Goldschmidt V, Grille S. Comparison of the Revised 4 Th (2016) and 5 Th (2022) Editions of the World Health Organization (WHO) Classification in a Cohort of Patients with Lower-Risk Myelodysplastic Syndromes/Neoplasms (MDS) - a Glam Registry (REGLAM) Analysis. Blood 2023, 142: 5179. DOI: 10.1182/blood-2023-185122.Peer-Reviewed Original ResearchProgression-free survivalTime of diagnosisAcute myeloid leukemiaMDS-MLDOverall survivalMDS patientsMDS-RSInternational Prognostic Scoring SystemLow-risk MDS patientsWorld Health Organization classificationLR-MDS patientsMedian blast countRisk MDS patientsCohort of patientsKaplan-Meier methodPrognostic scoring systemClassification of patientsNon-Hispanic whitesMDS-EB2PFS probabilityWHO 2016Baseline characteristicsBlast countMedian ageMeier methodEfficacy of Imetelstat in Achieving Red Blood Cell Transfusion Independence (RBC-TI) across Different Risk Subgroups in Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis-Stimulating Agents (ESAs) in IMerge Phase 3 Study
Komrokji R, Santini V, Fenaux P, Savona M, Madanat Y, Berry T, Sherman L, Navada S, Feller F, Sun L, Xia Q, Wan Y, Huang F, Zeidan A, Platzbecker U. Efficacy of Imetelstat in Achieving Red Blood Cell Transfusion Independence (RBC-TI) across Different Risk Subgroups in Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis-Stimulating Agents (ESAs) in IMerge Phase 3 Study. Blood 2023, 142: 194. DOI: 10.1182/blood-2023-181237.Peer-Reviewed Original ResearchInternational Prognostic Scoring SystemLower-risk myelodysplastic syndromesDifferent risk subgroupsErythropoiesis stimulating agentsLow-risk subgroupsRisk groupsRisk subgroupsResponse rateTI ratesRBC-TIClinical efficacyRisk categoriesHigh riskLower riskRed blood cell transfusion independenceIPSS risk groupPhase 3 portionIntermediate-risk groupCytogenetic risk groupHigh-risk patientsPhase 3 studyPrognostic scoring systemIntermediate-risk subgroupsHigh-risk subgroupsHigh-risk groupMolecular 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 valueImpact 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 mutationsCombining Gene Mutation with Transcriptomic Data Improves Outcome Prediction in Myelodysplastic Syndromes
Sauta E, Zampini M, Dall'Olio D, Sala C, Todisco G, Travaglino E, Lanino L, Tentori C, Maggioni G, D'Amico S, Asti G, Dall'Olio L, Mosca E, Ubezio M, Campagna A, Riva E, Bicchieri M, Savevski V, Santoro A, Kordasti S, Santini V, Diez-Campelo M, Kubasch A, Platzbecker U, Fenaux P, Zhao L, Zeidan A, Haferlach T, Castellani G, Della Porta M. Combining Gene Mutation with Transcriptomic Data Improves Outcome Prediction in Myelodysplastic Syndromes. Blood 2023, 142: 1863. DOI: 10.1182/blood-2023-186222.Peer-Reviewed Original ResearchAcute myeloid leukemiaProportional hazards modelOverall survivalClinical outcomesMyelodysplastic syndromeClinical featuresMDS patientsPrognostic informationConcordance indexCox proportional hazards modelConventional prognostic scoresIPSS-R scorePrimary end pointBone marrow blastsHarrell's concordance indexPeripheral blood cytopeniasPrognostic scoring systemRisk of progressionAdditional prognostic informationCytogenetic alterationsGene mutationsIndividual patient levelSame clinical phenotypeBlood cytopeniasMarrow blastsWhen to use which molecular prognostic scoring system in the management of patients with MDS?
Kewan T, Bewersdorf J, Gurnari C, Xie Z, Stahl M, Zeidan A. When to use which molecular prognostic scoring system in the management of patients with MDS? Best Practice & Research Clinical Haematology 2023, 36: 101517. PMID: 38092484, DOI: 10.1016/j.beha.2023.101517.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsInternational Prognostic Scoring SystemPrognostic scoring systemAcute myeloid leukemiaScoring systemRisk stratificationRecurrent molecular alterationsHigh-risk patientsAppropriate risk stratificationManagement of patientsRecurrent genetic mutationsIntensive therapyMyeloid leukemiaTreatment strategiesPrognostic toolDisease pathogenesisMolecular alterationsHematopoietic cancersClinical decisionHeterogeneous groupGenetic mutationsNext-generation sequencingPrognostic systemPatientsVariable propensitySubsequent revisionNovel Approaches and Future Directions in Myelodysplastic Syndrome Treatment
Bewersdorf J, Xie Z, Zeidan A. Novel Approaches and Future Directions in Myelodysplastic Syndrome Treatment. The Cancer Journal 2023, 29: 195-202. PMID: 37195776, DOI: 10.1097/ppo.0000000000000658.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHigh-risk MDS patientsInternational Prognostic Scoring SystemPhase III clinical trialsErythropoiesis-stimulating agentsPrognostic scoring systemRisk stratification toolAdvanced clinical testingStandard of careAcute myeloid leukemiaTelomerase inhibitor imetelstatEncouraging early resultsMyelodysplastic Syndromes TreatmentAnemic patientsAgent monotherapyMDS patientsStratification toolSyndrome treatmentCombination therapyDysplastic changesClinical trialsMyeloid leukemiaTreatment decisionsClonal disorderTreatment selectionClinical testing
2022
How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms
Gurnari C, Xie Z, Zeidan A. How I Manage Transplant Ineligible Patients with Myelodysplastic Neoplasms. Clinical Hematology International 2022, 5: 8-20. PMID: 36574201, PMCID: PMC10063738, DOI: 10.1007/s44228-022-00024-4.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMyelodysplastic syndromeMyelodysplastic neoplasmsInternational Prognostic Scoring SystemHematopoietic stem cell transplantManagement of cytopeniasNon-transplant treatmentTransplant-ineligible patientsBone marrow dysplasiaAvailable therapeutic optionsMinority of patientsNew investigational agentsPrognostic scoring systemStem cell transplantMain clinical problemLow-risk groupHigh-risk casesAcute myeloid leukemiaIneligible patientsMarrow dysplasiaCell transplantInvestigational agentsTherapeutic optionsClinical managementCurative approachMyeloid leukemiaDisease Characteristics and International Prognostic Scoring Systems (IPSS, IPSS-R, IPSS-M) in Adult Patients with Higher-Risk Myelodysplastic Syndromes (MDS) Participating in Two Randomized, Double-Blind, Placebo-Controlled Studies with Intravenous Sabatolimab Added to Hypomethylating Agents (HMA) (STIMULUS-MDS1 and MDS2)
Santini V, Platzbecker U, Fenaux P, Giagounidis A, Miyazaki Y, Sekeres M, Xiao Z, Sanz G, Van Hoef M, Ma F, Hertle S, Ramos P, Zeidan A. Disease Characteristics and International Prognostic Scoring Systems (IPSS, IPSS-R, IPSS-M) in Adult Patients with Higher-Risk Myelodysplastic Syndromes (MDS) Participating in Two Randomized, Double-Blind, Placebo-Controlled Studies with Intravenous Sabatolimab Added to Hypomethylating Agents (HMA) (STIMULUS-MDS1 and MDS2). Blood 2022, 140: 1340-1342. DOI: 10.1182/blood-2022-160282.Peer-Reviewed Original ResearchPrognostication in myelodysplastic syndromes (neoplasms): Molecular risk stratification finally coming of age
Xie Z, Chen E, Stahl M, Zeidan A. Prognostication in myelodysplastic syndromes (neoplasms): Molecular risk stratification finally coming of age. Blood Reviews 2022, 59: 101033. PMID: 36357283, DOI: 10.1016/j.blre.2022.101033.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromePrognostic modelManagement of MDSInternational Prognostic Scoring SystemMolecular prognostic modelPrognostic scoring systemMolecular risk stratificationHeterogeneous clinical outcomesBone marrow failure disordersMarrow failure disordersRevised IPSSClinical outcomesRisk prognosticationRisk stratificationTraditional risk modelsScoring systemMDS pathobiology
2021
IMerge: A phase 3 study to evaluate imetelstat in transfusion-dependent subjects with IPSS low or intermediate-1 risk myelodysplastic syndromes that are relapsed/refractory to erythropoiesis-stimulating agent treatment.
Platzbecker U, Komrokji R, Fenaux P, Zeidan A, Sekeres M, Savona M, Madanat Y, Sherman L, Dougherty S, Sun L, Huang F, Wan Y, Rizo A, Berry T, Feller F, Santini V. IMerge: A phase 3 study to evaluate imetelstat in transfusion-dependent subjects with IPSS low or intermediate-1 risk myelodysplastic syndromes that are relapsed/refractory to erythropoiesis-stimulating agent treatment. Journal Of Clinical Oncology 2021, 39: tps7056-tps7056. DOI: 10.1200/jco.2021.39.15_suppl.tps7056.Peer-Reviewed Original ResearchInternational Prognostic Scoring SystemErythropoiesis-stimulating agentsPhase 3 partRisk myelodysplastic syndromesMyelodysplastic syndromeRBC-TIRed blood cell (RBC) transfusion-dependent patientsErythropoiesis-stimulating agent treatmentLower-risk myelodysplastic syndromesPhase 2 partProgression of MDSRBC transfusion independenceTransfusion dependent subjectsPlacebo-controlled trialPhase 2/3 studyPhase 3 studyPrognostic scoring systemCurrent treatment optionsTransfusion-dependent patientsQuality of lifeMedian TI durationRate of CRVariant allele frequencyMechanism of actionAdult pts
2020
Prognostic Models in Myelodysplastic Syndromes
Bewersdorf J, Zeidan A. Prognostic Models in Myelodysplastic Syndromes. 2020, 109-127. DOI: 10.1007/978-3-030-51878-3_7.Peer-Reviewed Original ResearchInternational Prognostic Scoring SystemRisk stratification toolMyelodysplastic syndromeScoring systemStratification toolPrognostic scoring systemClinical trial enrollmentTrial enrollmentPrognostic relevanceDisease characteristicsPatient counselingBody of evidenceTreatment recommendationsPatient subpopulationsIndividual patientsTreatment selectionClinical scenariosPrognostic modelGenetic testingSomatic mutationsSyndromeScoresPatientsPrognostication
2018
Wide Variation in Use and Interpretation of Gene Mutation Profiling Panels Among Health Care Providers of Patients with Myelodysplastic Syndromes (MDS): Results of a Large Web-Based Survey
Pine A, Chokr N, Stahl M, Steensma D, Sekeres M, Litzow M, Luger S, Stone R, Greenberg P, Bejar R, Gore S, Zeidan A. Wide Variation in Use and Interpretation of Gene Mutation Profiling Panels Among Health Care Providers of Patients with Myelodysplastic Syndromes (MDS): Results of a Large Web-Based Survey. Blood 2018, 132: 1825. DOI: 10.1182/blood-2018-99-113888.Peer-Reviewed Original ResearchGene mutation profilingSouthwest Oncology GroupMyelodysplastic syndromeRisk stratificationHealth care providersMutation profilingOncology GroupMDS patientsClinical trialsCare providersWeb-based surveyConsensus evidence-based guidelinesInternational Prognostic Scoring SystemEastern Cooperative Oncology GroupGene panelAdvisory CommitteeConventional prognostic modelsThird of respondersHigh-risk patientsCooperative Oncology GroupPrognostic scoring systemRisk stratification toolManagement of patientsStem cell transplantEvidence-based guidelines
2017
Comparison of clinical outcomes and prognostic utility of risk stratification tools in patients with therapy-related vs de novo myelodysplastic syndromes: a report on behalf of the MDS Clinical Research Consortium
Zeidan AM, Al Ali N, Barnard J, Padron E, Lancet JE, Sekeres MA, Steensma DP, DeZern A, Roboz G, Jabbour E, Garcia-Manero G, List A, Komrokji R. Comparison of clinical outcomes and prognostic utility of risk stratification tools in patients with therapy-related vs de novo myelodysplastic syndromes: a report on behalf of the MDS Clinical Research Consortium. Leukemia 2017, 31: 1391-1397. PMID: 28111463, DOI: 10.1038/leu.2017.33.Peer-Reviewed Original ResearchConceptsInternational Prognostic Scoring SystemT-MDS patientsDe novo myelodysplastic syndromePrognostic scoring systemNovo myelodysplastic syndromeMyelodysplastic syndromeT-MDSClinical outcomesScoring systemMDS Clinical Research ConsortiumClinical Research ConsortiumRisk stratification toolRisk group categoriesRisk stratification modelConventional risk stratification modelPrognostic systemPrior therapyMedian survivalIndolent courseInferior survivalMDS patientsPrognostic utilityStratification toolWorse outcomesPatients
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 systemComparison of risk stratification tools in predicting outcomes of patients with higher-risk myelodysplastic syndromes treated with azanucleosides
Zeidan AM, Sekeres MA, Garcia-Manero G, Steensma DP, Zell K, Barnard J, Ali NA, Zimmerman C, Roboz G, DeZern A, Nazha A, Jabbour E, Kantarjian H, Gore SD, Maciejewski JP, List A, Komrokji R. Comparison of risk stratification tools in predicting outcomes of patients with higher-risk myelodysplastic syndromes treated with azanucleosides. Leukemia 2015, 30: 649-657. PMID: 26464171, PMCID: PMC4775363, DOI: 10.1038/leu.2015.283.Peer-Reviewed Original ResearchConceptsInternational Prognostic Scoring SystemPrognostic scoring systemMD Anderson Prognostic Scoring SystemMyelodysplastic syndromePrognostic toolScoring systemDifferent prognostic scoring systemsHigh-risk myelodysplastic syndromeRelative prognostic performanceOutcomes of patientsFirst-line therapyRisk stratification toolHigh-risk groupWorld Health OrganizationHR-MDSMedian OSObjective responseOverall survivalStandard therapyPrognostic utilityStratification toolPatient cohortPrognostic performancePatientsHealth OrganizationComparing 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
2014
The Prognostic Utility of the Current Risk Models in Predicting Outcomes of Patients (pts) with Higher-Risk Myelodysplastic Syndromes (HR-MDS) Treated with Hypomethylating Agents (HMA)
Zeidan A, Sekeres M, Garcia-Manero G, Barnard J, Al Ali N, Zimmerman C, Roboz G, Steensma D, DeZern A, Jabbour E, Kantarjian H, Zell K, Wang Q, Gore S, Nazha A, Maciejewski J, List A, Komrokji R. The Prognostic Utility of the Current Risk Models in Predicting Outcomes of Patients (pts) with Higher-Risk Myelodysplastic Syndromes (HR-MDS) Treated with Hypomethylating Agents (HMA). Blood 2014, 124: 1935. DOI: 10.1182/blood.v124.21.1935.1935.Peer-Reviewed Original ResearchMD Anderson Prognostic Scoring SystemHigh-risk myelodysplastic syndromeInternational Prognostic Scoring SystemMedian overall survivalHematopoietic cell transplantationPrognostic scoring systemOverall response rateOverall survivalHMA therapyHypomethylating agentComplete responseScoring systemStable diseaseHematologic improvementPartial responseProgressive diseaseConfidence intervalsInternational Working Group 2006 criteriaMDS Clinical Research ConsortiumPrognostic modelRevised International Prognostic Scoring SystemRisk categoriesBoehringer Ingelheim CorpCycles of therapyMarrow complete response