2023
Why 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
2019
Transforming growth factor (TGF)-β pathway as a therapeutic target in lower risk myelodysplastic syndromes
Bewersdorf JP, Zeidan AM. Transforming growth factor (TGF)-β pathway as a therapeutic target in lower risk myelodysplastic syndromes. Leukemia 2019, 33: 1303-1312. PMID: 30962581, DOI: 10.1038/s41375-019-0448-2.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsErythropoiesis stimulating agentsMyelodysplastic syndromeGrowth factorIneffective erythropoiesisLower-risk myelodysplastic syndromesTGF-β pathway inhibitorLR-MDS patientsFurther therapeutic optionsRisk myelodysplastic syndromesLimited therapy optionsTGF-β receptor activationPromising therapeutic approachPhysiologic conditionsTGF-β pathwayAberrant TGFImmunomodulator lenalidomideLR-MDSMyelosuppressive effectsTherapeutic optionsTherapy optionsLife impairmentAdditional potential targetsClinical dataMDS pathogenesisTherapeutic approachesGetting personal with myelodysplastic syndromes: is now the right time?
Chokr N, Pine AB, Bewersdorf JP, Shallis RM, Stahl M, Zeidan AM. Getting personal with myelodysplastic syndromes: is now the right time? Expert Review Of Hematology 2019, 12: 215-224. PMID: 30977414, PMCID: PMC6540985, DOI: 10.1080/17474086.2019.1592673.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsMyelodysplastic syndromeNext-generation sequencingTherapy selectionPrognosis of MDSRole of NGSPrognosis of patientsRoutine clinical practiceMinimal residual diseaseRecurrent genetic abnormalitiesResidual diseaseBlood countDisease stagePeripheral bloodHematologic malignanciesPrognostic evaluationMDS pathogenesisRoutine managementTherapy decisionsHealthy individualsBone marrowClinical practiceCytological examinationPatientsScoring systemDiagnostic accuracy