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 ResearchLower-risk myelodysplastic syndromesPatient-reported fatigueHigh transfusion burdenErythropoiesis-stimulating agentsTransfusion burdenMyelodysplastic syndromeChanges 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 levelsLuspaterceptCorrelation 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 analysisInitial 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 treatmentTargeted 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 treatmentMDS-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 careMDS-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 OSPlaceboImetelstatTreatment of Myelodysplastic Syndromes for Older Patients: Current State of Science, Challenges, and Opportunities
Kewan T, Stahl M, Bewersdorf J, Zeidan A. Treatment of Myelodysplastic Syndromes for Older Patients: Current State of Science, Challenges, and Opportunities. Current Hematologic Malignancy Reports 2024, 19: 138-150. PMID: 38632155, DOI: 10.1007/s11899-024-00733-y.Peer-Reviewed Original ResearchHematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationLower-risk MDSErythropoiesis-stimulating agentsHypomethylating agentsIPSS-MHR-MDSRisk stratificationMolecular International Prognostic Scoring SystemRisk of leukemia progressionTreated with hypomethylating agentsInternational Prognostic Scoring SystemTreatment of myelodysplastic syndromesOral hypomethylating agentsHigh-risk MDSPrognostic Scoring SystemStem cell transplantationEnhanced risk stratificationRecent FindingsRecent advancesRefine treatment strategiesQuality-of-life improvementAssociated with treatmentTreatment decision-makingIntensive chemotherapyMDS patients
2023
Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial
Platzbecker U, Santini V, Fenaux P, Sekeres M, Savona M, Madanat Y, Díez-Campelo M, Valcárcel D, Illmer T, Jonášová A, Bělohlávková P, Sherman L, Berry T, Dougherty S, Shah S, Xia Q, Sun L, Wan Y, Huang F, Ikin A, Navada S, Feller F, Komrokji R, Zeidan A. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet 2023, 403: 249-260. PMID: 38048786, DOI: 10.1016/s0140-6736(23)01724-5.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsPlacebo groupAdverse eventsMyelodysplastic syndromeGrade 3Subsequent anti-cancer therapyTreatment-emergent adverse eventsTreatment-emergent grade 3Days of randomisationIPSS risk groupRBC transfusion burdenTransfusion independence rateTreatment-related deathsUnacceptable toxic effectsPlacebo-controlled trialDisease-modifying activityPhase 2 trialPhase 3 trialPrimary efficacy analysisProportion of patientsWithdrawal of consentUnmet medical needComputer-generated scheduleAnti-cancer therapyThe ELEMENT-MDS Trial: A Phase 3 Randomized Study Evaluating Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Non-Transfusion-Dependent, Lower-Risk Myelodysplastic Syndromes
Zeidan A, Komrokji R, Buckstein R, Santini V, Rose S, Malini P, Lew G, Aggarwal D, Keeperman K, Jiang H, Giuseppi A, Zhang J, Cluzeau T, Shortt J, Platzbecker U. The ELEMENT-MDS Trial: A Phase 3 Randomized Study Evaluating Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Non-Transfusion-Dependent, Lower-Risk Myelodysplastic Syndromes. Blood 2023, 142: 6503. DOI: 10.1182/blood-2023-178635.Peer-Reviewed Original ResearchRBC transfusion dependenceErythropoiesis-stimulating agentsLR-MDSEpoetin alfaMyelodysplastic syndromeTransfusion dependenceSecondary endpointsStarting doseOverall survivalTransfusion independenceWeek 1Baseline serum erythropoietin levelsIntermediate-risk myelodysplastic syndromesIPSS-R risk categoryLower-risk myelodysplastic syndromesRed blood cell transfusionHigh-risk myelodysplastic syndromeInternational Prognostic Scoring SystemAdditional secondary endpointsRBC transfusion independenceTransfusion-free survivalKey secondary endpointBlood cell transfusionPatient Global ImpressionPhase 3 studyCharacterization and Management of Cytopenias after Imetelstat Treatment in the IMerge Phase 3 Trial of Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS)
Zeidan A, Savona M, Madanat Y, Fenaux P, Komrokji R, Jonášová A, Illmer T, Sun L, Berry T, Feller F, Navada S, Santini V, Platzbecker U. Characterization and Management of Cytopenias after Imetelstat Treatment in the IMerge Phase 3 Trial of Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS). Blood 2023, 142: 6478. DOI: 10.1182/blood-2023-180962.Peer-Reviewed Original ResearchTreatment-emergent adverse eventsLower-risk myelodysplastic syndromesPhase 3 trialManagement of cytopeniasGrade 3Placebo groupDose adjustmentMedian timeTreatment delayDose reductionInternational Prognostic Scoring System risk groupsHematologic treatment-emergent adverse eventsRed blood cell transfusion dependencyImetelstat treatmentExperienced grade 3RBC transfusion burdenRBC transfusion independenceTreatment cycles 1Grade 4 neutropeniaGrade 4 thrombocytopeniaPrimary end pointGrowth factor supportErythropoiesis-stimulating agentsCycle 1High telomerase activityImpact 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 mutationsImpact 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 mutationsUpdates 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 needsEfficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial
Platzbecker U, Della Porta M, Santini V, Zeidan A, 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, Shetty J, Hayati S, Vodala S, Prebet T, Degulys A, Paolini S, Cluzeau T, Fenaux P, Garcia-Manero G. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. The Lancet 2023, 402: 373-385. PMID: 37311468, DOI: 10.1016/s0140-6736(23)00874-7.Peer-Reviewed Original ResearchConceptsLower-risk myelodysplastic syndromesRed blood cell transfusion independenceEpoetin alfa groupErythropoiesis-stimulating agentsEpoetin alfaMyelodysplastic syndromeInterim analysisPrimary endpointAdverse eventsAlfa groupTransfusion independenceLower riskBody weightTreatment-emergent adverse eventsTreatment-related adverse eventsRed blood cell transfusionDurable clinical efficacyMean hemoglobin increaseMedian treatment exposureBlood cell transfusionCOVID-19 pneumoniaSubgroup of patientsWeeks of treatmentTreatment of anemiaAcute myeloid leukemiaNovel 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 testingWhy do we not have more drugs approved for MDS? A critical viewpoint on novel drug development in MDS
Frumm S, Shimony S, Stone R, DeAngelo D, Bewersdorf J, Zeidan A, Stahl M. Why do we not have more drugs approved for MDS? A critical viewpoint on novel drug development in MDS. Blood Reviews 2023, 60: 101056. PMID: 36805300, DOI: 10.1016/j.blre.2023.101056.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromeDNA methyltransferase inhibitorLow risk (LR) MDSHigh-risk myelodysplastic syndromeHigh transfusion needsRisk myelodysplastic syndromesCurrent treatment landscapeErythropoiesis-stimulating agentsNovel drug developmentHR-MDSTreatment landscapeTransfusion needsTargetable mutationsClinical trialsMDS pathogenesisNew agentsRing sideroblastsMore drugsUnmet needTherapy developmentDrug developmentMethyltransferase inhibitorFactor mutationsApprovalInflammation