2024
Cost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML
Alhajahjeh A, Patel K, Shallis R, Podoltsev N, Kewan T, Stempel J, Mendez L, Huntington S, Stahl M, Goshua G, Bewersdorf J, Zeidan A. Cost-effectiveness of Enasidenib versus conventional care for older patients with IDH2-mutant refractory/relapsed AML. Leukemia & Lymphoma 2024, ahead-of-print: 1-9. PMID: 39560957, DOI: 10.1080/10428194.2024.2426073.Peer-Reviewed Original ResearchConventional care regimensOlder patientsTreatment of older patientsIDH2 inhibitor enasidenibIncremental cost-effectiveness ratioCost-effectiveness ratioProbabilistic sensitivity analysesR/R AMLRefractory/relapsed AMLEvent-freeCost-effective treatmentEnasidenibCare regimensAMLPatientsCost-effectiveIncremental costLife yearsConventional careR/RIncremental effectSurvivalTreatmentAplastic anemia in association with multiple myeloma: clinical and pathophysiological insights
Muradashvili T, Liu Y, VanOudenhove J, Gu S, Krause D, Montanari F, Carlino M, Mancuso R, Stempel J, Halene S, Zeidan A, Podoltsev N, Neparidze N. Aplastic anemia in association with multiple myeloma: clinical and pathophysiological insights. Leukemia & Lymphoma 2024, ahead-of-print: 1-8. PMID: 39225418, DOI: 10.1080/10428194.2024.2393260.Peer-Reviewed Original ResearchAplastic anemiaMultiple myelomaImmunosuppressive therapyTransfusion requirementsProgenitor cellsPlasma cell-directed therapyT-cell destructionCell-directed therapiesInhibition of erythroid colony formationErythroid colony formationLevels of IL8Severe AAImmune cytopeniasPartial responseMM patientsHematopoietic stemSerum testsPartial improvementPathophysiological insightsPatientsImmune systemPlatelet apoptosisCytopeniasColony formationMyelomaMDS-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-772 Time Toxicity for Patients Receiving Oral Versus Parenteral Hypomethylating Agents for Myelodysplastic Syndromes/Neoplasms
Zeidan A, Olopoenia A, Costantino H, Modi K, Salimi T, Washington T, Krenitsky J, Epstein R. MDS-772 Time Toxicity for Patients Receiving Oral Versus Parenteral Hypomethylating Agents for Myelodysplastic Syndromes/Neoplasms. Clinical Lymphoma Myeloma & Leukemia 2024, 24: s406. DOI: 10.1016/s2152-2650(24)01382-x.Peer-Reviewed Original ResearchHMA therapyRetrospective analysis of adult patientsAnalysis of adult patientsRoute of administrationMedian life expectancyPropensity score matchingHypomethylating agentsMDS treatmentBurden of treatmentAdult patientsPatient cohortInfusion dayParenteral treatmentMatched cohortResults PatientsRetrospective analysisOlder patientsEmergency room visitsCancer therapyHMA treatmentParenteral administrationTherapyPatientsOutpatient settingScore matchingMDS-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 rangeClinical 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, Valcárcel D, Fenaux P, Shortt J, Komrokji R. 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: s192. DOI: 10.1016/s2152-2650(24)00648-7.Peer-Reviewed Original ResearchRisk prediction for clonal cytopenia: multicenter real-world evidence
Xie Z, Komrokji R, Al Ali N, Regelson A, Geyer S, Patel A, Saygin C, Zeidan A, Bewersdorf J, Mendez L, Kishtagari A, Zeidner J, Coombs C, Madanat Y, Chung S, Badar T, Foran J, Desai P, Tsai C, Griffiths E, Al Malki M, Amanam I, Lai C, Deeg H, Ades L, Arana Yi C, Osman A, Dinner S, Abaza Y, Taylor J, Chandhok N, Soong D, Brunner A, Carraway H, Singh A, Elena C, Ferrari J, Gallì A, Pozzi S, Padron E, Patnaik M, Malcovati L, Savona M, Al-Kali A. Risk prediction for clonal cytopenia: multicenter real-world evidence. Blood 2024, 144: 2033-2044. PMID: 38996210, PMCID: PMC11561536, DOI: 10.1182/blood.2024024756.Peer-Reviewed Original ResearchMyeloid neoplasmsIncidence of MNClonal cytopeniaCumulative incidencePlatelet count <High-risk mutationsCox proportional hazards modelsVariant allele fractionProportional hazards modelClinical trial designCCUS patientsStratify patientsGray's testC-indexDisease entityRisk groupsCytopeniasAllele fractionSomatic mutationsRisk factorsHigh riskNatural historyRisk scoreHazards modelPatientsPrognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases
Badar T, Nanaa A, Atallah E, Shallis R, Craver E, Li Z, Goldberg A, Saliba A, Patel A, Bewersdorf J, Duvall A, Burkart M, Bradshaw D, Abaza Y, Stahl M, Palmisiano N, Murthy S, Zeidan A, Kota V, Patnaik M, Litzow M. Prognostic impact of ‘multi-hit’ versus ‘single-hit’ TP53 alteration in patients with acute myeloid leukemia: results from the Consortium on Myeloid Malignancies and Neoplastic Diseases. Haematologica 2024, 109: 3533-3542. PMID: 38813716, PMCID: PMC11532685, DOI: 10.3324/haematol.2024.285000.Peer-Reviewed Original ResearchAcute myeloid leukemiaMyelodysplastic syndromeComplex cytogeneticsMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationLower-risk myelodysplastic syndromesHematopoietic stem cell transplantationHigher-risk myelodysplastic syndromesOutcomes of SHStem cell transplantationAllo-HCTTP53 alterationsPrognostic impactMyeloid malignanciesTP53 mutationsCell transplantationFLT3-ITDIDH1 mutationMultivariate analysisSupportive careUS academic institutionsNeoplastic diseasePatientsSuperior EFSPredicting outcomeTime toxicity for patients receiving oral versus parenteral hypomethylating agents for myelodysplastic syndromes/neoplasms (MDS).
Epstein R, Zeidan A, Olopoenia A, Costantino H, Modi K, Salimi T, Washington T, Krenitsky J. Time toxicity for patients receiving oral versus parenteral hypomethylating agents for myelodysplastic syndromes/neoplasms (MDS). Journal Of Clinical Oncology 2024, 42: 6568-6568. DOI: 10.1200/jco.2024.42.16_suppl.6568.Peer-Reviewed Original ResearchHMA therapyHypomethylating agentsRetrospective analysis of adult patientsAnalysis of adult patientsEmergency roomRoute of administrationTime burdenMedian life expectancyPropensity score matchingMDS treatmentBurden of treatmentAdult patientsParenteral treatmentPatient cohortInfusion dayRetrospective analysisCancer therapyPatientsTherapyOutpatient settingScore matchingTreatment groupsCohortHealthcare daysOutpatient visitsToward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS)
Efficace F, Buckstein R, Abel G, Giesinger J, Fenaux P, Bewersdorf J, Brunner A, Bejar R, Borate U, DeZern A, Greenberg P, Roboz G, Savona M, Sparano F, Boultwood J, Komrokji R, Sallman D, Xie Z, Sanz G, Carraway H, Taylor J, Nimer S, Della Porta M, Santini V, Stahl M, Platzbecker U, Sekeres M, Zeidan A. Toward a more patient‐centered drug development process in clinical trials for patients with myelodysplastic syndromes/neoplasms (MDS): Practical considerations from the International Consortium for MDS (icMDS). HemaSphere 2024, 8: e69. PMID: 38774655, PMCID: PMC11106800, DOI: 10.1002/hem3.69.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsPatient-reported outcomesPatient-reported outcome dataHealth-related qualityPatient-reported outcome endpointsHealth-related quality of lifeClinical trialsImprove patients' health-related qualityPatients' health-related qualityPatient-reported outcome itemsQuality of lifeTime-to-event analysisRandomized controlled trialsTreatment decision-makingPatient carePatient populationTreatment advancesControlled trialsPatientsIncurable diseaseAssessment strategiesHRQoLBenefit/risk assessmentDrug development processTrialsTherapyClinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem-Cell Transplantation in Patients With Myelodysplastic Syndromes
Tentori C, Gregorio C, Robin M, Gagelmann N, Gurnari C, Ball S, Caballero Berrocal J, Lanino L, D'Amico S, Spreafico M, Maggioni G, Travaglino E, Sauta E, Meggendorfer M, Zhao L, Campagna A, Savevski V, Santoro A, Al Ali N, Sallman D, Sole F, Garcia-Manero G, Germing U, Kroger N, Kordasti S, Santini V, Sanz G, Kern W, Platzbecker U, Diez-Campelo M, Maciejewski J, Ades L, Fenaux P, Haferlach T, Zeidan A, Castellani G, Komrokji R, Ieva F, Della Porta M, Bernardi M, Di Grazia C, Vago L, Rivoli G, Borin L, Chiusolo P, Giaccone L, Voso M, Bewersdorf J, Nibourel O, Beyá M, Jerez A, Hernández F, Kennedy K, Xicoy B, Ubezio M, Russo A, Todisco G, Mannina D, Bramanti S, Zampini M, Riva E, Bicchieri M, Asti G, Viviani F, Buizza A, Tinterri B, Kubasch A, Bacigalupo A, Raiola A, Rambaldi A, Passamonti F, Ciceri F. Clinical and Genomic-Based Decision Support System to Define the Optimal Timing of Allogeneic Hematopoietic Stem-Cell Transplantation in Patients With Myelodysplastic Syndromes. Journal Of Clinical Oncology 2024, 42: 2873-2886. PMID: 38723212, PMCID: PMC11328926, DOI: 10.1200/jco.23.02175.Peer-Reviewed Original ResearchHematopoietic stem-cell transplantationAllogeneic hematopoietic stem-cell transplantationStem-cell transplantationMyelodysplastic syndromeIPSS-MMolecular International Prognostic Scoring SystemInternational Prognostic Scoring SystemPrognostic Scoring SystemTime of transplantationProportion of patientsHigh-risk categoryOptimal timingProlonged life expectancyRevised IPSSIPSS-RRetrospective populationValidation cohortCurative treatmentClinical relevanceTransplantationPatientsModerately high-Scoring systemAverage survivalLife expectancyEFFICACY AND SAFETY OF LUSPATERCEPT VERSUS EPOETIN ALFA IN ERYTHROPOIESIS-STIMULATING AGENT (ESA)-NAIVE PATIENTS WITH TRANSFUSION-DEPENDENT LOWER-RISK MYELODYSPLASTIC SYNDROMES (LR-MDS): FULL ANALYSIS OF THE COMMANDS TRIAL
Garcia-Manero G, Platzbecker U, Santini V, Zeidan A, Fenaux P, Komrokji R, Shortt J, Valcarcel D, Jonasova A, Dimicoli-Salazar S, Tiong I, Lin C, Li J, Zhang J, Giuseppi A, Kreitz S, Pozharskaya V, Keeperman K, Rose S, Prebet T, Degulys A, Paolini S, Cluzeau T, Della Porta M. EFFICACY AND SAFETY OF LUSPATERCEPT VERSUS EPOETIN ALFA IN ERYTHROPOIESIS-STIMULATING AGENT (ESA)-NAIVE PATIENTS WITH TRANSFUSION-DEPENDENT LOWER-RISK MYELODYSPLASTIC SYNDROMES (LR-MDS): FULL ANALYSIS OF THE COMMANDS TRIAL. Leukemia Research Reports 2024, 21: 100447. DOI: 10.1016/j.lrr.2024.100447.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesTreatment-emergent adverse eventsEA-treated patientsRBC-TIPrimary endpointHI-ERed blood cell transfusion independenceHematological improvement-erythroidTransfusion independenceErythroid responseMyelodysplastic syndromeSecondary endpointsAdverse eventsFull analysisLuspaterceptAssessed efficacySafety resultsEpoetin alfaTreatment durationPatientsEndpointEfficacyDurationPost-treatmentAMLPatients’ perspectives on oral decitabine/cedazuridine for the treatment of myelodysplastic syndromes/neoplasms
Zeidan A, Perepezko K, Salimi T, Washington T, Epstein R. Patients’ perspectives on oral decitabine/cedazuridine for the treatment of myelodysplastic syndromes/neoplasms. Therapeutic Advances In Hematology 2024, 15: 20406207241257313. PMID: 39091323, PMCID: PMC11292726, DOI: 10.1177/20406207241257313.Peer-Reviewed Original ResearchHypomethylating agentsDEC-CQuality of lifeHypomethylating agent therapyGuideline-recommended treatmentHMA therapyOral therapySurvey of patientsTreatment side effectsAdult patientsImprove quality of lifeUS patientsSide effectsPatientsTreatment administrationTherapyDecitabine/cedazuridineTreatmentDaily activitiesMonthsLittle/no impact
2023
Traditional Medicare or Medicare Advantage? The Leukemia and Cancer Perspective
Kantarjian H, Zeidan A, Fathi A, Stein E, Rajkumar V, Tefferi A. Traditional Medicare or Medicare Advantage? The Leukemia and Cancer Perspective. Mayo Clinic Proceedings 2023, 99: 15-21. PMID: 38108685, DOI: 10.1016/j.mayocp.2023.11.004.Peer-Reviewed Original ResearchAre We Ready For “Triplet” Therapy in Higher-Risk MDS?
Brunner A, Platzbecker U, DeZern A, Zeidan A. Are We Ready For “Triplet” Therapy in Higher-Risk MDS? Clinical Hematology International 2023, 5: 23-32. PMID: 37933301, PMCID: PMC10625655, DOI: 10.46989/001c.88301.Peer-Reviewed Original ResearchAcute myeloid leukemiaCombination chemotherapyPatient populationOnly disease-modifying therapyNovel combination chemotherapyOngoing therapeutic challengeOlder patient populationUnique patient populationDisease-modifying therapiesCurrent treatment paradigmsTherapeutic challengeTreatment paradigmEffective therapyMyeloid leukemiaNew therapiesAppropriate endpointsTherapyChemotherapyComorbiditiesPatientsTransplantNeoplasmsLeukemiaPopulationDecitabineImpact of Mutational Status on Clinical Response to Imetelstat in Patients with Lower-Risk Myelodysplastic Syndromes in the IMerge Phase 3 Study
Santini V, Zeidan A, Fenaux P, Madanat Y, Berry T, Feller F, Sun L, Xia Q, Wan Y, Huang F, Savona M, Platzbecker U. Impact of Mutational Status on Clinical Response to Imetelstat in Patients with Lower-Risk Myelodysplastic Syndromes in the IMerge Phase 3 Study. Blood 2023, 142: 4603. DOI: 10.1182/blood-2023-179378.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesPlacebo groupTransfusion independenceTI ratesHot spot mutationsPoor prognosisMyelodysplastic syndromeRed blood cell transfusion independenceASXL1 mutationsErythropoiesis-stimulating agentsPhase 3 studyStudy of patientsTI responsesPresence of mutationsSpecific mutationsClinical responseStudy entryClinical efficacyClinical benefitPeripheral bloodMutation subgroupsDNMT3A mutationsEpigenetic modifiersPatientsRUNX1 mutationsData-Driven Harmonization of 2022 Who and ICC Classifications of Myelodysplastic Syndromes/Neoplasms (MDS): A Study By the International Consortium for MDS (icMDS)
Lanino L, Ball S, Bewersdorf J, Marchetti M, Maggioni G, Travaglino E, Al Ali N, Fenaux P, Platzbecker U, Santini V, Diez-Campelo M, Singh A, Jain A, Aguirre L, Tinsley-Vance S, Schwabkey Z, Chan O, Xie Z, Brunner A, Kuykendall A, Bennett J, Buckstein R, Bejar R, Carraway H, DeZern A, Griffiths E, Halene S, Hasserjian R, Lancet J, List A, Loghavi S, Odenike O, Padron E, Patnaik M, Roboz G, Stahl M, Sekeres M, Steensma D, Savona M, Taylor J, Xu M, Sweet K, Sallman D, Nimer S, Hourigan C, Wei A, Sauta E, D'Amico S, Asti G, Castellani G, Borate U, Sanz G, Efficace F, Gore S, Kim T, Daver N, Garcia-Manero G, Rozman M, Orfao A, Wang S, Foucar M, Germing U, Haferlach T, Scheinberg P, Miyazaki Y, Iastrebner M, Kulasekararaj A, Cluzeau T, Kordasti S, van de Loosdrecht A, Ades L, Zeidan A, Komrokji R, Della Porta M. Data-Driven Harmonization of 2022 Who and ICC Classifications of Myelodysplastic Syndromes/Neoplasms (MDS): A Study By the International Consortium for MDS (icMDS). Blood 2023, 142: 998. DOI: 10.1182/blood-2023-186580.Peer-Reviewed Original ResearchBlast countMost patientsTP53 mutationsTET2 mutationsChromosomal abnormalitiesMore TP53 mutationsBone marrow blastsGene mutationsSF3B1 mutationsClinical decision-making processHigh-risk mutationsMarrow blastsMultilineage dysplasiaPatient characteristicsAML patientsClinical entityInternational cohortSHAP analysisMDS casesPatientsClinical relevanceCytogenetic abnormalitiesClinical settingComplex karyotypeU2AF1 mutationsClinical 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 differencesWhen 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 revisionUpdates on risk stratification and management of lower-risk myelodysplastic syndromes/neoplasms
Badar T, Madanat Y, Zeidan A. Updates on risk stratification and management of lower-risk myelodysplastic syndromes/neoplasms. Future Oncology 2023, 19: 1877-1889. PMID: 37750305, DOI: 10.2217/fon-2023-0454.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsErythropoiesis-stimulating agentsTreatment-naive patientsHigh-risk patientsMeaningful efficacyIndolent courseDismal prognosisRandomized trialsRisk stratificationNeoplasm patientsSerial monitoringClinical trialsNovel therapiesPatientsAppropriate managementModest responseGood responseNeoplasmsTrialsNovel compoundsLuspaterceptLenalidomidePrognosisAnemiaTherapyMore needs