2023
The ELEMENT-MDS Trial: A Phase 3 Randomized Study Evaluating Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Non-Transfusion-Dependent, Lower-Risk Myelodysplastic Syndromes
Zeidan A, Komrokji R, Buckstein R, Santini V, Rose S, Malini P, Lew G, Aggarwal D, Keeperman K, Jiang H, Giuseppi A, Zhang J, Cluzeau T, Shortt J, Platzbecker U. The ELEMENT-MDS Trial: A Phase 3 Randomized Study Evaluating Luspatercept Versus Epoetin Alfa in Erythropoiesis-Stimulating Agent-Naive, Non-Transfusion-Dependent, Lower-Risk Myelodysplastic Syndromes. Blood 2023, 142: 6503. DOI: 10.1182/blood-2023-178635.Peer-Reviewed Original ResearchRBC transfusion dependenceErythropoiesis-stimulating agentsLR-MDSEpoetin alfaMyelodysplastic syndromeTransfusion dependenceSecondary endpointsStarting doseOverall survivalTransfusion independenceWeek 1Baseline serum erythropoietin levelsIntermediate-risk myelodysplastic syndromesIPSS-R risk categoryLower-risk myelodysplastic syndromesRed blood cell transfusionHigh-risk myelodysplastic syndromeInternational Prognostic Scoring SystemAdditional secondary endpointsRBC transfusion independenceTransfusion-free survivalKey secondary endpointBlood cell transfusionPatient Global ImpressionPhase 3 study
2019
A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of MBG453 Added to Hypomethylating Agents (HMAs) in Patients (pts) with Intermediate, High, or Very High Risk Myelodysplastic Syndrome (MDS): Stimulus-MDS1
Zeidan A, Miyazaki Y, Platzbecker U, Malek K, Niolat J, Kiertsman F, Fenaux P. A Randomized, Double-Blind, Placebo-Controlled, Phase II Study of MBG453 Added to Hypomethylating Agents (HMAs) in Patients (pts) with Intermediate, High, or Very High Risk Myelodysplastic Syndrome (MDS): Stimulus-MDS1. Blood 2019, 134: 4259. DOI: 10.1182/blood-2019-127041.Peer-Reviewed Original ResearchLeukemia-free survivalProgression-free survivalAcute myeloid leukemiaHR-MDSMyelodysplastic syndromeOverall survivalHypomethylating agentTim-3Leukemic stem cellsClinical trialsHigh riskGood safety/tolerability profileInternational Working Group 2006 criteriaSolid tumorsDiagnosis of AMLAllogeneic hematopoietic stem cell transplantationMulticenter phase II clinical trialPrior treatmentAnti-Tim-3 antibodyIPSS-R risk categorySafety/tolerability profileCelgene CorporationTherapy-related myelodysplastic syndromeHigh-risk myelodysplastic syndromeInternational Prognostic Scoring System