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
2020
Management of higher risk myelodysplastic syndromes after hypomethylating agents failure: are we about to exit the black hole?
Bewersdorf JP, Zeidan AM. Management of higher risk myelodysplastic syndromes after hypomethylating agents failure: are we about to exit the black hole? Expert Review Of Hematology 2020, 13: 1131-1142. PMID: 32876498, DOI: 10.1080/17474086.2020.1819233.Peer-Reviewed Original ResearchMeSH KeywordsAntineoplastic AgentsBiomarkersCombined Modality TherapyDisease ManagementDisease SusceptibilityDNA MethylationDrug DevelopmentDrug Resistance, NeoplasmHumansMolecular Targeted TherapyMutationMyelodysplastic SyndromesPrognosisRemission InductionRetreatmentTreatment FailureTreatment OutcomeConceptsHigh-risk myelodysplastic syndromeHMA failureMyelodysplastic syndromeHR-MDS patientsRisk myelodysplastic syndromesMainstay of treatmentImmune pathogenesisMost patientsComplete responseImmune therapyAbysmal prognosisNovel agentsTherapeutic approachesTherapeutic conceptsImmune evasionTreatment approachesPatientsGenetic testingSyndromePathogenesisTreatmentMolecular mechanismsRecent studiesFailureAnnual MeetingFedratinib hydrochloride to treat intermediate-2 or high-risk primary or secondary myelofibrosis.
Schiffer M, Kowalski A, Zhao J, Bewersdorf JP, Lewis RS, Zeidan AM. Fedratinib hydrochloride to treat intermediate-2 or high-risk primary or secondary myelofibrosis. Drugs Of Today 2020, 56: 755-768. PMID: 33332482, DOI: 10.1358/dot.2020.56.12.3230206.Peer-Reviewed Original ResearchMeSH KeywordsDrug DevelopmentHumansPrimary MyelofibrosisProtein Kinase InhibitorsPyrrolidinesSulfonamidesConceptsSecondary myelofibrosisClinical holdSelective Janus kinase 2 inhibitorNovel oral agentsCommon adverse effectsClinical trial dataFurther drug developmentGastrointestinal symptomsLiver transaminasesOral agentsSymptom burdenJanus kinase 2 inhibitorDrug AdministrationMyeloproliferative neoplasmsTrial dataU.S. FoodAdverse effectsKinase 2 inhibitorPhase IIDrug developmentMyelofibrosisSignificant reductionFurther investigationFedratinibSplenomegaly