2024
Sustained benefits of imetelstat on patient-reported fatigue in patients with lower-risk myelodysplastic syndromes ineligible for, or relapsed/refractory to, erythropoiesis-stimulating agents and high transfusion burden in the phase 3 IMerge study
Sekeres M, Santini V, Díez-Campelo M, Komrokji R, Fenaux P, Savona M, Madanat Y, Valcárcel-Ferreiras D, Oliva E, Regnault A, Creel K, Sengupta N, Dougherty S, Shah S, Sun L, Wan Y, Navada S, Zeidan A, Platzbecker U. Sustained benefits of imetelstat on patient-reported fatigue in patients with lower-risk myelodysplastic syndromes ineligible for, or relapsed/refractory to, erythropoiesis-stimulating agents and high transfusion burden in the phase 3 IMerge study. Leukemia & Lymphoma 2024, ahead-of-print: 1-6. PMID: 39535901, DOI: 10.1080/10428194.2024.2426057.Peer-Reviewed Original ResearchA 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, 17: 755-767. 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 studyImmune Landscape and Outcomes of Patients with RNA Splicing Factor-Mutant Acute Myeloid Leukemia and Myelodysplastic Syndromes Treated with Azacitidine +/- the Anti-PD-L1 Antibody Durvalumab
Bewersdorf J, Hasle V, Shallis R, Thompson E, De Menezes D, Rose S, Boss I, Mendez L, Podoltsev N, Stahl M, Kewan T, Halene S, Haferlach T, Fox B, Zeidan A. Immune Landscape and Outcomes of Patients with RNA Splicing Factor-Mutant Acute Myeloid Leukemia and Myelodysplastic Syndromes Treated with Azacitidine +/- the Anti-PD-L1 Antibody Durvalumab. Blood 2024, 144: 4585. DOI: 10.1182/blood-2024-194929.Peer-Reviewed Original ResearchAcute myeloid leukemiaAnti-PD-L1 antibody durvalumabOverall response rateMyelodysplastic syndromeComplete responseBM aspiratesMyeloid leukemiaInternational Working GroupBone marrowMyelodysplastic syndromes treated with azacitidineAcute myeloid leukemia ptsWild-type acute myeloid leukemiaSecondary acute myeloid leukemiaResponse criteriaAnti-PD-L1Immune checkpoint inhibitorsTreated with azacitidineOutcomes of patientsAny-cause deathGeneration of neoantigensVariant allele frequencySusceptible to treatmentMarrow CRAdverse cytogeneticsCheckpoint inhibitorsThe Prognostic and Predictive Value of RUNX1 Mutations in Newly Diagnosed Acute Myeloid Leukemia - an International Multicenter Cohort Study
Bystrom R, Bewersdorf J, Liu Y, Schaefer E, Shallis R, Boussi L, Zucenka A, Garciaz S, Aguirre L, DeAngelo D, Stone R, Luskin M, Garcia J, Winer E, Chen E, Wadleigh M, Ling K, Stein E, Goldberg A, Zeidan A, Shimony S, Stahl M. The Prognostic and Predictive Value of RUNX1 Mutations in Newly Diagnosed Acute Myeloid Leukemia - an International Multicenter Cohort Study. Blood 2024, 144: 2947-2947. DOI: 10.1182/blood-2024-205581.Peer-Reviewed Original ResearchAcute myeloid leukemiaComposite complete responseMedian OSRUNX1 mutationsComplete responseIntensive chemotherapyOverall survivalMyelodysplastic syndromeAllo-SCTIntermediate riskPredictive valueMyeloid leukemiaInternational multicenter retrospective cohort studyTreatment strategiesCohort studyNewly diagnosed acute myeloid leukemiaAllogeneic stem cell transplantationMulticenter retrospective cohort studyNext-generation sequencingAntecedent MDSConcomitant TP53 mutationIncomplete count recoveryTreated with ICStem cell transplantationAdverse risk featuresLong-Term Response Analysis of Transfusion Independence in Erythropoiesis Stimulating Agent-Naive Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes Treated with Luspatercept Vs Epoetin Alfa in the COMMANDS Trial
Garcia-Manero G, Santini V, Zeidan A, Komrokji R, Pozharskaya V, Keeperman K, Lai Y, Aggarwal B, Miteva D, Ferreiras D, Fenaux P, Shortt J, Della Porta M, Platzbecker U. Long-Term Response Analysis of Transfusion Independence in Erythropoiesis Stimulating Agent-Naive Patients with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Syndromes Treated with Luspatercept Vs Epoetin Alfa in the COMMANDS Trial. Blood 2024, 144: 350. DOI: 10.1182/blood-2024-194240.Peer-Reviewed Original ResearchHigh-risk myelodysplastic syndromeAcute myeloid leukemiaLower-risk myelodysplastic syndromesRBC-TIDuration of responseMyelodysplastic syndromeRed blood cellsEpoetin alfaClinical benefitSerum erythropoietinIntermediate-risk myelodysplastic syndromesExposure-adjusted incidence ratesRed blood cell transfusionCumulative durationWithdrawal of consentClinically relevant subgroupsLong-term clinical valueVery low-Transfusion independenceData cutoffEligible ptsMyeloid leukemiaClinical efficacyTreatment armsLuspaterceptEvaluating Molecular and Clinical Predictors in Myelodysplastic Syndromes through Machine Learning Integration
Mosquera Orgueira A, Perez Encinas M, Pérez Míguez C, Crucitti D, Piñeiro Fiel M, Díaz Varela N, Mora E, Díaz-Beyá M, Montoro M, Pomares H, Ramos Ortega F, Tormo M, Jerez A, Nomdedeu J, de Miguel Sanchez C, Arenillas L, Carcel Corella P, Cedena Romero M, Xicoy B, Rivero M, Del Orbe Barreto R, Bewersdorf J, Stahl M, Stempel J, Kewan T, Zeidan A, Diez-Campelo M, Valcarcel D. Evaluating Molecular and Clinical Predictors in Myelodysplastic Syndromes through Machine Learning Integration. Blood 2024, 144: 6683-6683. DOI: 10.1182/blood-2024-198114.Peer-Reviewed Original ResearchLeukemia-free survivalPredictors of OSMyelodysplastic syndromeOverall survivalIPSS-MC-indexFLT3-ITDPredictors of leukemia-free survivalRevised International Prognostic Scoring SystemPrognostication of myelodysplastic syndromesInternational Prognostic Scoring SystemPrognostic Scoring SystemCox regression analysisMolecular dataClinical trial designIPSS-RGenetic variabilityMLL-PTDRandom survival forestPrognostic landscapePrognostic significanceLaboratory parametersAffected prognosisClinical predictorsClinical indicesChanges in Red Blood Cell Transfusion Burden with Luspatercept Versus Epoetin Alfa in Patients with Lower-Risk Myelodysplastic Syndromes in the Phase 3, Open-Label, Randomized, Controlled COMMANDS Trial
Garcia-Manero G, Della Porta M, Santini V, Zeidan A, Komrokji R, Fenaux P, Valcárcel D, Shortt J, Glassberg M, Yucel A, Lai Y, Miteva D, Rose S, Hnoosh A, Platzbecker U. Changes in Red Blood Cell Transfusion Burden with Luspatercept Versus Epoetin Alfa in Patients with Lower-Risk Myelodysplastic Syndromes in the Phase 3, Open-Label, Randomized, Controlled COMMANDS Trial. Blood 2024, 144: 1832-1832. DOI: 10.1182/blood-2024-198304.Peer-Reviewed Original ResearchRegular red blood cell transfusionsLower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsTreated with epoetin alfaEpoetin alfaRBC unitsLuspatercept treatmentTransfusion burdenMyelodysplastic syndromeOpen-labelFirst-in-class erythroid maturation agentRed blood cell transfusion burdenEfficacy of erythropoiesis-stimulating agentsRed blood cell transfusionErythroid maturation agentBlood cell transfusionBaseline Hb levelsArm of treatmentRBC-TICell transfusionMedian ageResponse durabilityChronic anemiaHb levelsLuspaterceptHealth-Related Quality of Life of Luspatercept Versus Epoetin Alfa in Red Blood Cell Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes: Results from the Final Datacut of the Phase 3 COMMANDS Study
Oliva E, Platzbecker U, Della Porta M, Garcia-Manero G, Santini V, Fenaux P, Shortt J, Komrokji R, Pelligra C, Guo S, Yucel A, Glassberg M, Eliason L, Hnoosh A, Miteva D, Rose S, Kreitz S, Sekeres M, Zeidan A. Health-Related Quality of Life of Luspatercept Versus Epoetin Alfa in Red Blood Cell Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes: Results from the Final Datacut of the Phase 3 COMMANDS Study. Blood 2024, 144: 3216. DOI: 10.1182/blood-2024-198374.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesReduction of red blood cellsFunctional Assessment of Cancer Therapy-AnemiaClinically important differenceEpoetin alfaMyelodysplastic syndromeRevised International Prognostic Scoring SystemInternational Prognostic Scoring SystemHealth-related qualityClinically important difference thresholdImprovement of cytopeniasMean haemoglobin increaseRBC transfusion independencePrognostic Scoring SystemPhase 3 studyStratified Cox proportional hazard regressionsFront-line treatmentOnset of improvementQLQ-C30Cox proportional hazards regressionEORTC QLQ-C30Treatment of anemiaProportional hazards regressionSuperior treatment effectsPost-baseline assessmentCorrelation of Patient-Reported Outcomes with Red Blood Cell Transfusion Reduction and Rise in Hemoglobin in Patients with Lower-Risk Myelodysplastic Syndromes in the IMerge Trial
Sekeres M, Santini V, Zeidan A, Platzbecker U, Komrokji R, Diez-Campelo M, Fenaux P, Savona M, Madanat Y, Valcarcel D, Regnault A, Creel K, Sengupta N, Dougherty S, Shah S, Sun L, Wan Y, Navada S, Oliva E. Correlation of Patient-Reported Outcomes with Red Blood Cell Transfusion Reduction and Rise in Hemoglobin in Patients with Lower-Risk Myelodysplastic Syndromes in the IMerge Trial. Blood 2024, 144: 3210. DOI: 10.1182/blood-2024-199636.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsPatient-reported outcomesFunctional Assessment of Cancer Therapy-AnemiaRed blood cellsAnemia symptomsQuality of lifeRBC-TDRBC-TITransfusion burdenPlacebo recipientsTransfusion independenceMyelodysplastic syndromeTransfusion reductionRed blood cell transfusion independenceFunctional Assessment of Chronic Illness Therapy (FACIT)-FatigueBaseline to cycleUnited States Food and Drug AdministrationStates Food and Drug AdministrationCorrelations of patient-reported outcomesFirst-in-classFunctional assessmentFood and Drug AdministrationMaintenance of quality of lifePost hoc analysisLandscape of Immune Cell States and Ecosystems in Patients with Myelodysplastic Syndrome to Refine Prognostic Assessment and Predict Treatment Response. a Study By i4MDS Consortium
Riva E, Calvi M, Zampini M, Dall'Olio L, Merlotti A, Russo A, Maggioni G, Orlandi L, Frigo A, Ficara F, Crisafulli L, Sauta E, D'Amico S, Lugli E, Campagna A, Ubezio M, Tentori C, Todisco G, Lanino L, Buizza A, Ventura D, Pinocchio N, Saba E, Santoro A, Santini V, van de Loosdrecht A, Komrokji R, Garcia-Manero G, Fenaux P, Ades L, Platzbecker U, Haferlach T, Almeida A, Zeidan A, Kordasti S, Remondini D, Castellani G, Di Vito C, Mavilio D, Della Porta M. Landscape of Immune Cell States and Ecosystems in Patients with Myelodysplastic Syndrome to Refine Prognostic Assessment and Predict Treatment Response. a Study By i4MDS Consortium. Blood 2024, 144: 665-665. DOI: 10.1182/blood-2024-200184.Peer-Reviewed Original ResearchMyelodysplastic syndromeImmune dysfunctionClinical work-upIPSS-MHypomethylating agentsBone marrowImmune ecosystemNatural killerNK cellsImmune monitoringPeripheral bloodT cellsAntibody panelClinical heterogeneity of myelodysplastic syndromesPatients treated with hypomethylating agentsCohort of MDS patientsLevel of immune dysfunctionRisk of leukemic transformationResponse to hypomethylating agentsHeterogeneity of myelodysplastic syndromesMulti-color flow cytometryWork-up of patientsClinical work-up of patientsImmune monitoring approachesMDS microenvironmentInitial Results from the QTc Substudy of the IMerge Phase 3 Trial Demonstrate Clinically Meaningful Efficacy, Manageable Safety, and Absence of Proarrhythmic Risk in Patients with Lower-Risk Myelodysplastic Syndromes Who Received Prior Therapies Beyond Erythropoiesis Stimulating Agents
Komrokji R, Santini V, Platzbecker U, Van Eygen K, Diez-Campelo M, De Paz R, Sanz G, Thépot S, Kaźmierczak M, Oliva E, Sekeres M, Fenaux P, Madanat Y, Savona M, Riggs J, Dougherty S, Lennox A, Xia Q, Sun L, Berry T, Zeidan A. Initial Results from the QTc Substudy of the IMerge Phase 3 Trial Demonstrate Clinically Meaningful Efficacy, Manageable Safety, and Absence of Proarrhythmic Risk in Patients with Lower-Risk Myelodysplastic Syndromes Who Received Prior Therapies Beyond Erythropoiesis Stimulating Agents. Blood 2024, 144: 4590-4590. DOI: 10.1182/blood-2024-200885.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsHematological improvement-erythroidFood and Drug AdministrationRBC-TIPhase 3 trialPlacebo recipientsHypomethylating agentsInternational Working GroupData cutoffMyelodysplastic syndromeTransfusion reductionRed blood cellsProarrhythmic riskProgression to acute myeloid leukemiaErythropoiesis-stimulating agent useConcentration-QT relationshipEffects of imetelstatRBC transfusion independenceGrade 3/4 neutropeniaMedian treatment durationKaplan-Meier methodologyClinically meaningful efficacyUnited States Food and Drug AdministrationAcute myeloid leukemiaA Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes
Zeidan A, Sekeres M, Al-Samkari H, Carraway H, DeZern A, Mittelman M, Little M, Beynon V, Dai X, Sommakia S, Despotovic J, Patel P, Dibacco M, Fattizzo B, Stein E, Sallman D, Kulasekararaj A, Platzbecker U, Fenaux P. A Phase 2B, Open-Label Multicenter Study of Tebapivat (AG-946), a Potent Pyruvate Kinase Activator, in Patients with Anemia Due to Lower-Risk Myelodysplastic Syndromes. Blood 2024, 144: 6708-6708. DOI: 10.1182/blood-2024-203073.Peer-Reviewed Original ResearchLower-risk MDSErythropoiesis-stimulating agentsLower-risk myelodysplastic syndromesRed blood cell unitsMyelodysplastic syndromeProportion of patientsDose levelsRed blood cellsIPSS-RTransfusion burdenOpen-labelFirst doseRevised International Prognostic Scoring SystemHistory of acute myeloid leukemiaMDS mouse modelsMedian duration of responsePhase 2bInternational Prognostic Scoring SystemOpen-label multicenter studyAssociated with increased morbidityBone marrow neoplasmsHigh transfusion burdenIPSS-R scoreLow transfusion burdenDose level 1Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN)
Garcia-Manero G, McCloskey J, Scott B, Griffiths E, Kiner-Strachan B, Brunner A, Zeidan A, Traer E, Madanat Y, Meyer J, Erba H, Baratam P, Borate U, Sano Y, Oganesian A, Zhu L, Keer H, Savona M. Results from a Phase 1 Open-Label Dose Escalation and Expansion Trial of Oral Azacitidine + Cedazuridine (ASTX030) in Patients with Myelodysplastic Syndromes (MDS) and MDS/Myeloproliferative Neoplasms (MPN). Blood 2024, 144: 662. DOI: 10.1182/blood-2024-203569.Peer-Reviewed Original ResearchDose-limiting toxicityPhase 1 trialDose-expansion partMDS/MPN overlap syndromesMyelodysplastic syndromeAdverse eventsDose combinationMDS/myeloproliferative neoplasmImmediate-releaseOverlap syndromeAUC exposureOpen-label phase 1 trialDelayed-releaseCytidine deaminaseProlonged grade 4 neutropeniaIncreased absolute bioavailabilityMarrow complete responsePhase 2 doseGrade 4 neutropeniaDose-escalation partErythropoiesis-stimulating agentsAcute myeloid leukemiaTreatment of patientsClinical efficacy assessmentDNA methyltransferase inhibitorEffect of Prior Treatments on the Clinical Activity of Imetelstat in Transfusion-Dependent Patients with Erythropoiesis-Stimulating Agent, Relapsed or Refractory/Ineligible Lower-Risk Myelodysplastic Syndromes
Platzbecker U, Santini V, Zeidan A, Sekeres M, Fenaux P, Raza A, Mittelman M, Thépot S, Buckstein R, Germing U, Madanat Y, Diez-Campelo M, Valcarcel D, Jonasova A, Dougherty S, Shah S, Xia Q, Sun L, Navada S, Savona M, Komrokji R. Effect of Prior Treatments on the Clinical Activity of Imetelstat in Transfusion-Dependent Patients with Erythropoiesis-Stimulating Agent, Relapsed or Refractory/Ineligible Lower-Risk Myelodysplastic Syndromes. Blood 2024, 144: 352-352. DOI: 10.1182/blood-2024-203612.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesHematological improvement-erythroidErythropoiesis-stimulating agentsErythropoiesis-stimulating agent therapyHb riseRBC-TIHypomethylating agentsTransfusion reductionClinical activityMyelodysplastic syndromeQTc studyRed blood cellsPrior treatmentInternational Working GroupRBC transfusion independenceHypomethylating agent treatmentTransfusion-dependent patientsPhase 2/3 trialsLimited treatment optionsUnited States Food and Drug AdministrationStates Food and Drug AdministrationFirst-in-classFood and Drug AdministrationRBC-TDLenalidomide treatmentEnhancing Personalized Prognostic Assessment of Myelodysplastic Syndromes through a Multimodal and Explainable Deep Data Fusion Approach (MAGAERA)
Sauta E, Sartori F, Lanino L, Asti G, D'Amico S, Delleani M, Riva E, Zampini M, Zazzetti E, Bicchieri M, Maggioni G, Campagna A, Todisco G, Tentori C, Ubezio M, Russo A, Buizza A, Ficara F, Crisafulli L, Brindisi M, Ventura D, Pinocchio N, Rahal D, Lancellotti C, Bonometti A, Di Tommaso L, Savevski V, Santoro A, Derus N, Dall'Olio D, Santini V, Sole F, Platzbecker U, Fenaux P, Diez-Campelo M, Komrokji R, Garcia-Manero G, Haferlach T, Kordasti S, Zeidan A, Castellani G, Sanavia T, Fariselli P, Della Porta M. Enhancing Personalized Prognostic Assessment of Myelodysplastic Syndromes through a Multimodal and Explainable Deep Data Fusion Approach (MAGAERA). Blood 2024, 144: 105-105. DOI: 10.1182/blood-2024-205413.Peer-Reviewed Original ResearchPersonalized medicine programsMyelodysplastic syndrome patientsMyelodysplastic syndromeOverall survivalConcordance indexClinical outcomesMay-Grunwald-GiemsaHypomethylating agentsBone marrowAnalysis of hematological malignanciesSomatic mutation screeningEvaluation of T lymphocytesResponse to hypomethylating agentsCD34+ bone marrowStudies of myelodysplastic syndromesGenomic featuresMDS populationRNA-seqPrediction of patient outcomeGenomic characterizationHarrell's concordance indexPredicting clinical outcomesHematoxylin and eosin (H&EMorphological dataMulti-omicsThe Implication of TP53 Allelic Status for Outcome and Erythropoiesis in MDS
Li B, Zeng Y, Li C, Liu J, Song G, Yao Y, Zeidan A, Xiao Z. The Implication of TP53 Allelic Status for Outcome and Erythropoiesis in MDS. Blood 2024, 144: 6707-6707. DOI: 10.1182/blood-2024-205429.Peer-Reviewed Original ResearchNext-generation sequencingMultiplex ligation-dependent probe amplificationComplex karyotypeMyelodysplastic syndromeCD34+ cellsPrognostic impactClinical characteristicsBone marrowPercentage of erythroid progenitorsCD34+ BM cellsAllele statusSubtype of myelodysplastic syndromeLigation-dependent probe amplificationTP53-mutant patientsPercentage of blastsSingle-cell mutational analysisCopy number assayCopy number alterationsClone sizeLineage dysplasiaMutant patientsTP53 mutationsBM cellsMDS-UWHO classificationReal-World Use Patterns and Clinical Outcomes for Myelodysplastic Syndrome Patients Initiating Oral Decitabine and Cedazuridine or Intravenous/Subcutaneous Hypomethylating Agents
Zeidan A, Zhao R, Pierre-Victor D, Wang Y, Tepsick J, Lan Z, Salimi T. Real-World Use Patterns and Clinical Outcomes for Myelodysplastic Syndrome Patients Initiating Oral Decitabine and Cedazuridine or Intravenous/Subcutaneous Hypomethylating Agents. Blood 2024, 144: 5189-5189. DOI: 10.1182/blood-2024-205495.Peer-Reviewed Original ResearchAML-free survivalTreated with hypomethylating agentsOral hypomethylating agentsHypomethylating agentsMyelodysplastic syndrome patientsMyelodysplastic syndromeDEC-CClinical characteristicsMDS diagnosisNext treatmentMonths median follow-upRisk of AML transformationHigh-risk myelodysplastic syndromeReal-world treatment outcomesTreatment of adult patientsHypomethylating agent treatmentKaplan-Meier survival analysis methodMedian follow-upCox regression analysisReal-world studyAML transformationHMA therapyOral decitabineECOG 0Electronic health record databaseEncouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study
Kontro M, Stein A, Pyörälä M, Rimpiläinen J, Siitonen T, Rannikko J, Mickos J, Turpin R, Hakoniemi M, Berns B, Aakko S, Pawlitzky I, Hollmén M, Zeidan A, Daver N. Encouraging Efficacy of Bexmarilimab with Azacitidine in Relapsed or Refractory MDS in Bexmab Ph1/2 Study. Blood 2024, 144: 4265. DOI: 10.1182/blood-2024-206804.Peer-Reviewed Original ResearchMedian overall survival estimatesMyelodysplastic syndrome patientsTreatment-emergent adverse eventsMyelodysplastic syndromeBone marrowMarrow CRStable diseaseHematologic improvementPartial responseClever-1HLA-DROutcome of high-risk myelodysplastic syndromeHuman leukocyte antigen-DR isotypePhase 1 dose-escalationResponse of stable diseaseAllogeneic stem cell transplantationHigh-risk myelodysplastic syndromeHigher-risk myelodysplastic syndromesSimon 2-stage designTreatment of myelodysplastic syndromesResponse rateBayesian optimal intervalMedian overall survivalPrimary refractory diseaseRefractory myelodysplastic syndromeData-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 blastsMDS-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 care