2024
A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS
Norman M, Yamartino K, Gerstein R, Shallis R, Mendez L, Podoltsev N, Stahl M, Eighmy W, Zeidan A. A review of the isocitrate dehydrogenase inhibitors in management of adult patients with AML and MDS. Expert Review Of Hematology 2024, ahead-of-print: 1-13. PMID: 39474840, DOI: 10.1080/17474086.2024.2422554.Peer-Reviewed Original ResearchDiagnosed AMLSurvival benefitManagement of acute myeloid leukemiaDevelopment of oral therapiesIsocitrate dehydrogenase inhibitorsNewly diagnosed AMLManagement of adult patientsPost-transplant maintenanceAcute myeloid leukemiaSingle-arm studyExcellent response ratesIDH inhibitorsRelapsed AMLHypomethylating agentsInhibitor therapyMyelodysplastic syndromeOral therapyCombination therapyPost-transplantMyeloid leukemiaImproved survivalSingle-armAdult patientsAzacitidineRandomized studyRisk 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, 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 modelPatientsPre-emptive therapeutic decisions based on measurable residual disease status in acute myeloid leukemia: ready for prime time?
El Chaer F, Perissinotti A, Loghavi S, Zeidan A. Pre-emptive therapeutic decisions based on measurable residual disease status in acute myeloid leukemia: ready for prime time? Leukemia 2024, 1-7. PMID: 39496917, DOI: 10.1038/s41375-024-02458-6.Peer-Reviewed Original ResearchAcute myeloid leukemiaMyeloid leukemiaCore binding factor acute myeloid leukemiaIncreased risk of relapseResidual disease statusPost-allo-HCTHematopoietic cell transplantationRisk of relapseTherapeutic decision-makingInnovative treatment strategiesMRD-positiveIntensive chemotherapyMRD monitoringCell transplantationNPM1 mutationsImprove patient outcomesRisk stratificationTherapeutic decisionsTreatment strategiesIncreased riskRisk factorsMRDNatural historyPreemptive interventionAssess diseaseAnalyzing determinants of premature trial discontinuation in leukemia clinical trials
Rotter L, Alhajahjeh A, Stempel J, Grimshaw A, Bewersdorf J, Blaha O, Kewan T, Podoltsev N, Shallis R, Mendez L, Stahl M, Zeidan A. Analyzing determinants of premature trial discontinuation in leukemia clinical trials. Leukemia & Lymphoma 2024, ahead-of-print: 1-9. PMID: 39440622, DOI: 10.1080/10428194.2024.2416565.Peer-Reviewed Original ResearchClinical trialsSlow accrualLeukemia trialsEarly-phase trialsNon-randomized trialsAcademic-sponsored trialsImprove patient outcomesLeukemia clinical trialsSingle-centerTrial discontinuationSponsored trialsSmall trialsLeukemiaEarly terminationPatient outcomesLate-phaseIndependent reviewersTermination ratesComprehensive searchTrialsTargeted 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 treatmentData-driven, harmonised classification system for myelodysplastic syndromes: a consensus paper from the International Consortium for Myelodysplastic Syndromes
Komrokji R, 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, Delleani M, Campagna A, Borate U, Sanz G, Efficace F, Gore S, Kim T, Daver N, Garcia-Manero G, Rozman M, Orfao A, Wang A, 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, Della Porta M, Syndromes I. Data-driven, harmonised classification system for myelodysplastic syndromes: a consensus paper from the International Consortium for Myelodysplastic Syndromes. The Lancet Haematology 2024, 11: e862-e872. PMID: 39393368, DOI: 10.1016/s2352-3026(24)00251-5.Peer-Reviewed Original ResearchGenomic featuresData-driven approachTP53 inactivationGenomic heterogeneityEntity labelsGenetic featuresDel(7q)/-7Myelodysplastic syndromeGenomic profilingData scientistsMutated SF3B1Cluster assignmentComplex karyotypeRUNX1 mutationsModified Delphi consensus processDel(5qIsolated del(5qAcute myeloid leukemiaData-drivenDelphi consensus processMarrow blastsContemporary Approach to the Diagnosis and Classification of Myelodysplastic Neoplasms/Syndromes—Recommendations From the International Consortium for Myelodysplastic Neoplasms/Syndromes (MDS [icMDS])
Aakash F, Gisriel S, Zeidan A, Bennett J, Bejar R, Bewersdorf J, Borate U, Boultwood J, Brunner A, Buckstein R, Carraway H, Churpek J, Daver N, DeZern A, Efficace F, Fenaux P, Figueroa M, Garcia-Manero G, Gore S, Greenberg P, Griffiths E, Halene S, Hourigan C, Kim T, Kim N, Komrokji R, Kutchroo V, List A, Little R, Majeti R, Nazha A, Nimer S, Odenike O, Padron E, Patnaik M, Platzbecker U, Della Porta M, Roboz G, Sallman D, Santini V, Sanz G, Savona M, Sekeres M, Stahl M, Starczynowski D, Steensma D, Taylor J, Abdel-Wahab O, Wei A, Xie Z, Xu M, Hasserjian R, Loghavi S. Contemporary Approach to the Diagnosis and Classification of Myelodysplastic Neoplasms/Syndromes—Recommendations From the International Consortium for Myelodysplastic Neoplasms/Syndromes (MDS [icMDS]). Modern Pathology 2024, 37: 100615. PMID: 39322118, DOI: 10.1016/j.modpat.2024.100615.Peer-Reviewed Original ResearchIntensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML
Bewersdorf J, Shimony S, Shallis R, Liu Y, Berton G, Schaefer E, Zeidan A, Goldberg A, Stein E, Marcucci G, Bystrom R, Lindsley R, Chen E, Perez J, Stein A, Pullarkat V, Aldoss I, DeAngelo D, Neuberg D, Stone R, Garciaz S, Ball B, Stahl M. Intensive induction chemotherapy vs hypomethylating agents in combination with venetoclax in NPM1-mutant AML. Blood Advances 2024, 8: 4845-4855. PMID: 38941537, PMCID: PMC11416634, DOI: 10.1182/bloodadvances.2024012858.Peer-Reviewed Original ResearchIntensive induction chemotherapyAcute myeloid leukemiaNPM1-Mutant Acute Myeloid LeukemiaInduction chemotherapyHypomethylating agentsMulticenter retrospective cohort study of patientsPatients treated with ICAllogeneic stem cell transplantationRetrospective cohort study of patientsMulticenter retrospective cohort studyCohort study of patientsComposite complete remissionStem cell transplantationYears-oldFLT3-ITD mutationStudy of patientsStandard of careNormal cytogeneticsComplete remissionCell transplantationNPM1 mutationsMyeloid leukemiaFLT3-ITDYounger patientsOlder patientsAplastic 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-156 Efficacy of Imetelstat on Red Blood Cell (RBC)-Transfusion Independence (TI) in the Absence of Platelet Transfusions or Myeloid Growth Factors (MGF) in IMerge
Zeidan A, Santini V, Platzbecker U, Sekeres M, Savona M, Fenaux P, Madanat Y, Raza A, Xia Q, Sun L, Riggs J, Shah S, Navada S, Berry T, Komrokji R. MDS-156 Efficacy of Imetelstat on Red Blood Cell (RBC)-Transfusion Independence (TI) in the Absence of Platelet Transfusions or Myeloid Growth Factors (MGF) in IMerge. Clinical Lymphoma Myeloma & Leukemia 2024, 24: s386. DOI: 10.1016/s2152-2650(24)01344-2.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesMyeloid growth factorsErythropoiesis-stimulating agentsRBC-TIPlatelet transfusionsTransfusion-dependentHb levelsLong-term respondersPercentage of patientsHb riseRBC-TDPlacebo patientsNon-del(5qMyelodysplastic syndromePlacebo groupPrimary endpointSecondary endpointsInvestigator's discretionClinical benefitPlaceboAnalysis cutoffImetelstatDisease progressionTransfusionSupportive careEfficacy of Imetelstat on Red Blood Cell (RBC)-Transfusion Independence (TI) in the Absence of Platelet Transfusions or Myeloid Growth Factors (MGF) in IMerge
Zeidan A, Santini V, Platzbecker U, Sekeres M, Savona M, Fenaux P, Madanat Y, Raza A, Xia Q, Sun L, Riggs J, Shah S, Navada S, Berry T, Komrokji R. Efficacy of Imetelstat on Red Blood Cell (RBC)-Transfusion Independence (TI) in the Absence of Platelet Transfusions or Myeloid Growth Factors (MGF) in IMerge. Clinical Lymphoma Myeloma & Leukemia 2024, 24: s191. DOI: 10.1016/s2152-2650(24)00646-3.Peer-Reviewed Original ResearchMDS-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 rangeTime 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. Time Toxicity for Patients Receiving Oral Versus Parenteral Hypomethylating Agents for Myelodysplastic Syndromes/Neoplasms. Clinical Lymphoma Myeloma & Leukemia 2024, 24: s197. DOI: 10.1016/s2152-2650(24)00684-0.Peer-Reviewed Original ResearchMDS-157 Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Santini V, Komrokji R, Sekeres M, Savona M, Fenaux P, Madanat Y, Oliva E, Buckstein R, Annaášová A, Germing U, Mittelman M, Thepot S, Riggs J, Dougherty S, Berry T, Navada S, Xia Q, Sun L, Zeidan A, Platzbecker U. MDS-157 Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s386-s387. DOI: 10.1016/s2152-2650(24)01345-4.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesNon-del(5q) lower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsRBC-TIOverall survivalRBC-TDNon-del(5qClinical benefitRed blood cellsHemoglobin increaseRBC transfusion dependenceStratified log-rank testMedian follow-upKaplan-Meier methodLog-rank testWithdrawal of consentMedian OSOS ratesHemoglobin riseMyelodysplastic syndromeOS analysisHemoglobin levelsAssess OSPlaceboImetelstatClinical 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 ResearchOverall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Santini V, Komrokji R, Sekeres M, Savona M, Fenaux P, Madanat Y, Oliva E, Buckstein R, Jonášová A, Germing U, Mittelman M, Thepot S, Riggs J, Dougherty S, Berry T, Navada S, Xia Q, Sun L, Zeidan A, Platzbecker U. Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s192. DOI: 10.1016/s2152-2650(24)00647-5.Peer-Reviewed Original ResearchOverall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Santini V, Komrokji R, Sekeres M, Savona M, Fenaux P, Madanat Y, Oliva E, Buckstein R, Jonášová A, Germing U, Mittelman M, Thepot S, Riggs J, Dougherty S, Berry T, Navada S, Xia Q, Sun L, Zeidan A, Platzbecker U. Overall Survival (OS), Clinical Benefit, and Durable Red Blood Cell (RBC) Transfusion Independence (TI) With Imetelstat in the IMerge Phase 3 Trial of RBC-Transfusion Dependent (TD) Lower-Risk Myelodysplastic Syndromes (LR-MDS). Clinical Lymphoma Myeloma & Leukemia 2024, 24: s153. DOI: 10.1016/s2152-2650(24)00388-4.Peer-Reviewed Original ResearchMultilineage 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, Valcárcel D, Fenaux P, Platzbecker U. 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: s192. DOI: 10.1016/s2152-2650(24)00649-9.Peer-Reviewed Original Research