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 mutationsApprovalInflammationConsensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes
Zeidan A, Platzbecker U, Bewersdorf J, Stahl M, Adès L, Borate U, Bowen D, Buckstein R, Brunner A, Carraway H, Daver N, Díez-Campelo M, de Witte T, DeZern A, Efficace F, Garcia-Manero G, Garcia J, Germing U, Giagounidis A, Griffiths E, Hasserjian R, Hellström-Lindberg E, Iastrebner M, Komrokji R, Kulasekararaj A, Malcovati L, Miyazaki Y, Odenike O, Santini V, Sanz G, Scheinberg P, Stauder R, van de Loosdrecht A, Wei A, Sekeres M, Fenaux P. Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes. Blood 2023, 141: 2047-2061. PMID: 36724453, DOI: 10.1182/blood.2022018604.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHigh-risk MDSComplete remissionResponse criteriaInternational Working GroupClinical trialsHigh-risk myelodysplastic syndromeEnd pointMarrow complete remissionPartial hematologic recoveryClinical end pointsPatient-centered outcomesNovel investigational drugsVariable clinical presentationEvent end pointsHemoglobin thresholdHematologic recoveryCount recoveryClinical presentationClinical benefitMyelodysplastic syndromeIWG criteriaMyelodysplastic neoplasmsConsensus recommendationsInvestigational drugsNew agents
2020
Following in the footsteps of acute myeloid leukemia: are we witnessing the start of a therapeutic revolution for higher-risk myelodysplastic syndromes?
Bewersdorf JP, Zeidan AM. Following in the footsteps of acute myeloid leukemia: are we witnessing the start of a therapeutic revolution for higher-risk myelodysplastic syndromes? Leukemia & Lymphoma 2020, 61: 2295-2312. PMID: 32421403, PMCID: PMC7670856, DOI: 10.1080/10428194.2020.1761968.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsHigh-risk myelodysplastic syndromeAcute myeloid leukemiaMyelodysplastic syndromeMyeloid leukemiaAllogeneic hematopoietic stem cell transplantationGenetic testingHematopoietic stem cell transplantationImmune checkpoint inhibitorsCurative treatment optionMainstay of therapyRecent therapeutic advancesStem cell transplantationCurrent treatment approachesAgent azacitidineCheckpoint inhibitorsMost patientsMDS patientsAvailable therapiesCell transplantationPatient subgroupsTreatment optionsTherapeutic advancesIDH inhibitorsIndividualized treatmentNew agentsEvolving therapies for lower-risk myelodysplastic syndromes
Bewersdorf JP, Zeidan AM. Evolving therapies for lower-risk myelodysplastic syndromes. Annals Of Hematology 2020, 99: 677-692. PMID: 32078008, DOI: 10.1007/s00277-020-03963-1.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsMeSH KeywordsAnemiaBlood TransfusionChelation TherapyClinical Trials as TopicCri-du-Chat SyndromeDrugs, InvestigationalForecastingHematinicsHematopoietic Stem Cell TransplantationHumansImmunosuppressive AgentsIron OverloadLenalidomideMolecular Targeted TherapyMulticenter Studies as TopicMyelodysplastic SyndromesPrecision MedicinePrognosisRisk AssessmentSeverity of Illness IndexConceptsInternational Prognostic Scoring SystemLow-risk MDS patientsMyelodysplastic syndromeMDS patientsLower-risk myelodysplastic syndromesPrognostic scoring systemRisk stratification toolAdvanced clinical testingSymptomatic anemiaStratification toolHematologic malignanciesCurrent treatmentTherapeutic approachesNew agentsNew drug approvalsTherapeutic conceptsClinical developmentPatientsClinical testingMolecular pathogenesisScoring systemTherapeutic landscapePromising agentDrug approvalGenetic features