2024
Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them
Bidikian A, Bewersdorf J, Kewan T, Stahl M, Zeidan A. Acute Promyelocytic Leukemia in the Real World: Understanding Outcome Differences and How We Can Improve Them. Cancers 2024, 16: 4092. PMID: 39682277, PMCID: PMC11640703, DOI: 10.3390/cancers16234092.Peer-Reviewed Original ResearchAcute promyelocytic leukemiaAdvent of all-trans retinoic acidEarly mortalityLong-term treatment toxicitiesArsenic trioxidePromyelocytic leukemiaClinical practiceIncidence of acute promyelocytic leukemiaRates of remissionLong-term outcomesTreatment of acute promyelocytic leukemiaLong-term survivalComprehensive patient evaluationResource-limited settingsTreatment toxicityAll-trans retinoic acidDelayed diagnosisPatient demographicsSignificant comorbiditiesTreatment initiationOlder patientsExpert centersClinical trialsTreatment outcomesReal-world settingsCost-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 effectSurvivalTreatmentSustained 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 studyPre-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 diseasePhase I Study of Ruxolitinib in Combination with Abemaciclib for Patients with Primary or Post-Polycythemia Vera/Essential Thrombocythemia Myelofibrosis
Bewersdorf J, Derkach A, Zeidan A, Stein E, Mauro M, Podoltsev N, Rampal R. Phase I Study of Ruxolitinib in Combination with Abemaciclib for Patients with Primary or Post-Polycythemia Vera/Essential Thrombocythemia Myelofibrosis. Blood 2024, 144: 6659-6659. DOI: 10.1182/blood-2024-194918.Peer-Reviewed Original ResearchDose-limiting toxicityCancer Institute Common Terminology Criteria for Adverse EventsTreatment-related adverse eventsAdverse eventsDose levelsCombination therapySpleen volumeInhibitor abemaciclibGrade 3Patients discontinued treatment due to adverse eventsNational Cancer Institute Common Terminology Criteria for Adverse EventsPhase I dose-escalation trialTreatment due to adverse eventsCommon Terminology Criteria for Adverse EventsDisease progressionRecommended phase II doseMulticenter Phase IPlanned dose levelsGrade 3 thrombocytopeniaMedian overall survivalPhase II doseBone marrow blastsBone marrow fibrosisClinically significant bleedingData cut-offOral Decitabine/Cedazuridine in Patients with MDS and TP53 Mutations: A Propensity Score Matching Analysis from the Phase II and III Trials
Urrutia S, Sasaki K, Bataller A, Kantarjian H, Montalban-Bravo G, McCloskey J, Griffiths E, Yee K, Zeidan A, Savona M, Oganesian A, Sano Y, Keer H, Garcia-Manero G. Oral Decitabine/Cedazuridine in Patients with MDS and TP53 Mutations: A Propensity Score Matching Analysis from the Phase II and III Trials. Blood 2024, 144: 661-661. DOI: 10.1182/blood-2024-193274.Peer-Reviewed Original ResearchMedian overall survivalHematopoietic stem cell transplantationHypomethylating agentsTP53 mutationsOverall survivalImproved survivalMedian follow-up timeIPSS-R scoreComplete response rateStem cell transplantationECOG performance statusPropensity score matching analysisFollow-up timeScore matching analysisIPSS-RComplex cytogeneticsCell transplantationPerformance statusDecitabine/cedazuridineTP53wtCo-mutationsLandmark analysisPoor outcomeTP53mutMedian numberClinical Benefits of Achieving Hemoglobin (Hb) Levels ≥ 10 g/dL in Transfusion-Dependent (TD) Erythropoiesis-Stimulating Agent (ESA)-Naive Patients (Pts) with Lower-Risk (LR) Myelodysplastic Syndromes (MDS) Treated with Luspatercept in the COMMANDS Trial
Santini V, Zeidan A, Platzbecker U, Komrokji R, Garcia-Manero G, Miteva D, Yucel A, Pozharskaya V, Rose S, Lai Y, Giuseppi A, Valcárcel Ferreiras D, Fenaux P, Shortt J, Della Porta M. Clinical Benefits of Achieving Hemoglobin (Hb) Levels ≥ 10 g/dL in Transfusion-Dependent (TD) Erythropoiesis-Stimulating Agent (ESA)-Naive Patients (Pts) with Lower-Risk (LR) Myelodysplastic Syndromes (MDS) Treated with Luspatercept in the COMMANDS Trial. Blood 2024, 144: 1818-1818. DOI: 10.1182/blood-2024-194239.Peer-Reviewed Original ResearchDuration of responseMedian duration of responseDose escalationRBC-TILR-MDSHb levelsTransfusion-dependentClinical benefitRed blood cellsHigh dosesTransfusion independenceMedian durationCutoff dateShort duration of responseExposure-adjusted incidence ratesData cutoff dateIncidence of gradeTarget Hb levelBaseline Hb levelsClinically meaningful ratesIncreased Hb levelsClinically significant increaseLowered riskLuspatercept groupClinically significant improvementLong-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 indicesZiftomenib Combined with Intensive Induction (7+3) in Newly Diagnosed NPM1- m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007
Zeidan A, Wang E, Issa G, Erba H, Altman J, Balasubramanian S, Strickland S, Roboz G, Schiller G, McMahon C, Palmisiano N, Madanat Y, Rotta M, Nadiminti K, Wei H, Riches M, Corum D, Leoni M, Dale S, Fathi A. Ziftomenib Combined with Intensive Induction (7+3) in Newly Diagnosed NPM1- m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007. Blood 2024, 144: 214-214. DOI: 10.1182/blood-2024-198218.Peer-Reviewed Original ResearchDose-limiting toxicityMinimal residual diseaseAcute myeloid leukemiaMedian time to neutrophil recoveryDays to platelet recoveryMinimal residual disease negativityDecreased neutrophil countKMT2A-rDecreased platelet countNPM1 mutationsAdverse eventsCRC ratesDose levelsNeutrophil recoveryData cutoffQTc prolongationPlatelet recoveryPlatelet countMyeloid leukemiaNeutrophil countDifferentiation syndromeClinical activityCycle 1 day 8Persistent acute myeloid leukemiaStandard dose of cytarabineChanges 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 assessmentDrug Development for Hematological Cancer Targets Using Asclepius
Crucitti D, Pérez Míguez C, Piñeiro Fiel M, Diaz Arias J, Gómez Fernández J, Mosquera Orgueira A, Zeidan A. Drug Development for Hematological Cancer Targets Using Asclepius. Blood 2024, 144: 7464-7464. DOI: 10.1182/blood-2024-198610.Peer-Reviewed Original ResearchStructure-based drug design approachBinding energy evaluationComputational chemistry calculationsBiological targetsDrug design approachMolecular dynamics simulationsFree energy techniquesSynthetic stepsChemistry calculationsSynthetic accessibilityADMET profileSynthetic costArray of moleculesMolecular dockingDynamics simulationsDrug designNovel inhibitorsDrug discoveryADMETMoleculesStructural conformationIdentified inhibitorsProtein targetsHematological cancersIntegration of artificial intelligenceZiftomenib Combined with Venetoclax/Azacitidine in Relapsed/Refractory NPM1 -m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007
Fathi A, Issa G, Wang E, Erba H, Altman J, Balasubramanian S, Roboz G, Schiller G, McMahon C, Palmisiano N, Juckett M, Madanat Y, Rotta M, Pratz K, Yaghmour G, Nadiminti K, Wei H, Riches M, Corum D, Leoni M, Dale S, Zeidan A. Ziftomenib Combined with Venetoclax/Azacitidine in Relapsed/Refractory NPM1 -m or KMT2A -r Acute Myeloid Leukemia: Interim Phase 1a Results from KOMET-007. Blood 2024, 144: 2880-2880. DOI: 10.1182/blood-2024-199170.Peer-Reviewed Original ResearchAcute myeloid leukemiaDecreased neutrophil countDecreased platelet countNPM1 mutationsKMT2A-rAdverse eventsCRC ratesClinical activityR/R patientsData cutoffQTc prolongationPlatelet countMyeloid leukemiaNucleophosmin 1Neutrophil countDifferentiation syndromeComposite complete remission rateCycle 1 day 8R/R acute myeloid leukemiaTreatment-emergent adverse eventsDose of venetoclaxInhibitor-naive patientsDose-escalation cohortsDose-expansion phaseComplete remission rateCorrelation 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 microenvironmentInequalities in Treatment Utilization Among Older Medicare Beneficiaries with Newly Diagnosed Acute Myeloid Leukemia Who Are Ineligible for Induction Therapy
Zeidan A, Xu Y, Kapustyan T, Miu K, Chen C, Kamalakar R, Lin C, Ma E, Montez M, Wu Z, Yee T, Sun H, Rava A, Kim S, Huntington S. Inequalities in Treatment Utilization Among Older Medicare Beneficiaries with Newly Diagnosed Acute Myeloid Leukemia Who Are Ineligible for Induction Therapy. Blood 2024, 144: 3795-3795. DOI: 10.1182/blood-2024-200213.Peer-Reviewed Original ResearchTime to treatment initiationND-AMLAssociated with lower oddsAcute myeloid leukemiaAML treatmentVEN-HMACharlson Comorbidity IndexTargeted therapyInduction therapyFemale patientsMyeloid leukemiaTreatment initiationShorter time to treatment initiationTherapy uptakeMedicare beneficiariesNewly diagnosed acute myeloid leukemiaLower oddsOdds ratioHematopoietic stem cell transplantationTime-to-treatment initiationDiagnosed acute myeloid leukemiaIn-hospital survival rateLow-dose chemotherapyClinical performance statusStem cell transplantationTrial in Progress: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 2 Study of AK117/Placebo in Combination with Azacitidine in Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes (AK117-205)
Zeidan A, Tong H, Xiao Z, Baratam P, Abboud R, Benton C, Zeidner J, Borate U, Chai-Ho W, Lu Y, Yang J, Hu M, Li B, Xia M, Sallman D. Trial in Progress: A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 2 Study of AK117/Placebo in Combination with Azacitidine in Patients with Newly Diagnosed Higher-Risk Myelodysplastic Syndromes (AK117-205). Blood 2024, 144: 6705-6705. DOI: 10.1182/blood-2024-200541.Peer-Reviewed Original ResearchAllogeneic hematopoietic stem cell transplantationHematopoietic stem cell transplantationEastern Cooperative Oncology GroupIPSS-R scoreEvent-free survivalStem cell transplantationAcute myeloid leukemiaOverall survivalIPSS-RTransfusion independenceComplete responseCR rateDouble-blindCell transplantationHR-MDSMyeloproliferative neoplasmsAdverse eventsChimeric antigen receptor T cellsTransformation to acute myeloid leukemiaAnemia ratesMulticenter phase 2 studyTreated with hypomethylating agentsHigher-risk myelodysplastic syndromesSeverity of adverse eventsAdequate organ functionRenew Trial in Progress: A Phase 3, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Neoplasms (MDS)
Komrokji R, Diez-Campelo M, Chee L, Cluzeau T, DeZern A, Fenaux P, Garcia-Manero G, Giagounidis A, Platzbecker U, Della Porta M, Santini V, Sekeres M, Zeidan A, Buckstein R, Ross M, Jiang Y, Bobba S, Hankin M, Materna C, Graham C, Thamake S, Rovaldi C, Grayson D, Salstrom J. Renew Trial in Progress: A Phase 3, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elritercept (KER-050) for the Treatment of Transfusion-Dependent Anemia in Adult Participants with Very Low-, Low-, or Intermediate-Risk Myelodysplastic Neoplasms (MDS). Blood 2024, 144: 3228.1-3228.1. DOI: 10.1182/blood-2024-200797.Peer-Reviewed Original ResearchDurability of responseMyelodysplastic neoplasmsFollow-up periodTransfusion burdenProportion of participantsAdverse eventsTransfusion independenceIneffective hematopoiesisDouble-blindPlacebo-controlled phase 3 studyAchievement of transfusion independenceMultiple stages of hematopoiesisSafety follow-up periodDouble-blind treatment periodLong-term follow-up periodSeverity of adverse eventsLong-term follow-upErythroid maturation agentLow transfusion burdenSustained hematologic improvementTreatment of transfusion-dependent anemiaPlacebo-controlled studyTransfusion-dependent anemiaPhase 3 studyProgression to AML