2025
Pivotal Results of SELECT-MDS-1 Phase 3 Study of Tamibarotene with Azacitidine in Newly Diagnosed Higher-Risk MDS
DeZern A, Thepot S, de Botton S, Patriarca A, Deeren D, Torregrossa-Diaz J, Marconi G, Bernal T, Burgues J, Xicoy B, Jonášová A, Zeidan A, Dimicoli-Salazar S, Simand C, Valcarcel D, Campelo M, Chai-Ho W, Saini L, Garnier A, Geissler K, Ofran Y, Nagy Z, Krishnamurthy P, Lübbert M, Basak G, Carraway H, Sallman D, Borate U, Santini V, Campbell V, Fenaux P, Braun T, Lanza F, Zaucha J, Roth D, Paul S, Roy P, Kelly M, Volkert A, Chisholm J, Malak T, Klimek V, Cluzeau T, Group T. Pivotal Results of SELECT-MDS-1 Phase 3 Study of Tamibarotene with Azacitidine in Newly Diagnosed Higher-Risk MDS. Blood Advances 2025 PMID: 40334070, DOI: 10.1182/bloodadvances.2025016229.Peer-Reviewed Original ResearchHigher-risk MDSIPSS-RBlast count >5%Higher-risk MDS patientsMDS-EB-1Bone marrow blastsPhase 3 studyHigher-risk featuresBlood-based assayGene overexpressionMarrow blastsCR ratePrimary MDSBaseline characteristicsAzacitidineResponse rateNatural historyPatientsTamibaroteneOverexpressionP-valueTreatment effectsExploration of alternative approachesRARAGroupNCCN Guidelines® Insights: Myelodysplastic Syndromes, Version 2.2025.
Greenberg P, Stone R, Abaza Y, Al-Kali A, Anand S, Ball B, Bennett J, Borate U, Brunner A, Chai-Ho W, Curtin P, DeZern A, Gaensler K, Gahvari Z, Garcia-Manero G, Griffiths E, Haque T, Jacoby M, Jonas B, Keel S, Khanal R, Kishtagari A, Madanat Y, Maness L, McCurdy S, McMahon C, Odenike O, Osman A, Reddy V, Sallman D, Sayar H, Shallis R, Singh A, Tanaka T, Thota S, Kovach E, Nguyen J, Hochstetler C. NCCN Guidelines® Insights: Myelodysplastic Syndromes, Version 2.2025. Journal Of The National Comprehensive Cancer Network 2025, 23: 66-75. PMID: 40073835, DOI: 10.6004/jnccn.2025.0013.Peer-Reviewed Original ResearchConceptsNCCN Guidelines InsightsHigher-risk MDSComprehensive care of patientsPrognostic significanceNCCN guidelinesClinical evidenceCare of patientsTreatment recommendationsMultidisciplinary panelNovel therapeuticsTherapeutic agentsNCCNLowered riskComprehensive careTreatmentBiological factorsPatients
2024
Validation of Recent Response Criteria (ELN-22, IWG-23 and PB-CR) in 1634 MDS/CMML/AML Patients Treated with HMA or HMA-Ven Using CPH Models and a CPH Deep Neural Network - Can or Should Response Criteria be Harmonized for Similarly Treated Patients?
Pleyer L, Vaisband M, Klammer P, Drost M, Angermann H, Keil F, Petzer V, Heibl S, Moritz J, Girschikofsky M, Stampfl-Mattersberger M, Pichler A, Hartmann B, Aschauer G, Schmitt C, Vallet S, Boros S, Pichler P, Hammerl-Steiner A, Renneberg F, Majjiga D, Russ G, Egle A, Leisch M, Melchardt T, Zaborsky N, Faber V, Bewersdorf J, Zeidan A, Hasenauer J, Greil R. Validation of Recent Response Criteria (ELN-22, IWG-23 and PB-CR) in 1634 MDS/CMML/AML Patients Treated with HMA or HMA-Ven Using CPH Models and a CPH Deep Neural Network - Can or Should Response Criteria be Harmonized for Similarly Treated Patients? Blood 2024, 144: 7511-7511. DOI: 10.1182/blood-2024-208073.Peer-Reviewed Original ResearchComposite complete remissionTime to next treatmentCox proportional hazardsHypomethylating agentsOverall survivalCox proportional hazards modelsMedian OSResponse criteriaTreated ptsCycles of HMAHigher-risk MDSKaplan-Meier methodBone marrow evaluationProspective cohort studyStandard of careComplete remissionMarrow evaluationTreated patientsMultivariable adjustmentNext treatmentCohort studyClinical trialsClinical overlapHazard ratioDisease entity
2023
Sex Disparities in Myelodysplastic Syndromes: Genotype, Phenotype, and Outcomes
Tinsley-Vance S, Ali N, Ball S, Aguirre L, Jain A, Hussaini M, Chan O, Kuykendall A, Sweet K, Lancet J, Padron E, Sallman D, Komrokji R. Sex Disparities in Myelodysplastic Syndromes: Genotype, Phenotype, and Outcomes. Clinical Lymphoma Myeloma & Leukemia 2023, 23: 355-359. PMID: 36813626, PMCID: PMC10121764, DOI: 10.1016/j.clml.2023.01.007.Peer-Reviewed Original ResearchConceptsMedian overall survivalLower-risk MDSHigher-risk MDSHigher platelet countMoffitt Cancer CenterOverall survivalMyelodysplastic syndromeRUNX1 mutationsPlatelet countFemale patientsImpact of biological sexLow hemoglobinImpact of sexClinical phenotypeCancer CenterMolecular profilingPatientsSex disparitiesWomenMDS databaseMenPhenotypeOutcomesSexGenotypes
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