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
2021
Clinical effectiveness of DNA methyltransferase inhibitors and lenalidomide in older patients with refractory anemia with ring sideroblasts: a population-based study in the United States
Wang X, Zeidan AM, Wang R, Bewersdorf JP, Zhang C, Podoltsev NA, Huntington SF, Gore SD, Ma X. Clinical effectiveness of DNA methyltransferase inhibitors and lenalidomide in older patients with refractory anemia with ring sideroblasts: a population-based study in the United States. Leukemia & Lymphoma 2021, 62: 2438-2447. PMID: 33899659, DOI: 10.1080/10428194.2021.1913142.Peer-Reviewed Original ResearchConceptsPopulation-based studyDNA methyltransferase inhibitorRARS patientsRefractory anemiaRing sideroblastsRed blood cell transfusion independenceEnd Results-Medicare databaseLower-risk myelodysplastic syndromesPopulation-based cohortMethyltransferase inhibitorRBC-TIMedian durationMedian survivalOlder patientsTransfusion independenceTreatment initiationMyelodysplastic syndromeClinical effectivenessBetter survivalLenalidomideTreatment groupsPatientsRS statusOlder adultsAnemia