2023
Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial
Platzbecker U, Santini V, Fenaux P, Sekeres M, Savona M, Madanat Y, Díez-Campelo M, Valcárcel D, Illmer T, Jonášová A, Bělohlávková P, Sherman L, Berry T, Dougherty S, Shah S, Xia Q, Sun L, Wan Y, Huang F, Ikin A, Navada S, Feller F, Komrokji R, Zeidan A. Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet 2023, 403: 249-260. PMID: 38048786, DOI: 10.1016/s0140-6736(23)01724-5.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesErythropoiesis-stimulating agentsPlacebo groupAdverse eventsMyelodysplastic syndromeGrade 3Subsequent anti-cancer therapyTreatment-emergent adverse eventsTreatment-emergent grade 3Days of randomisationIPSS risk groupRBC transfusion burdenTransfusion independence rateTreatment-related deathsUnacceptable toxic effectsPlacebo-controlled trialDisease-modifying activityPhase 2 trialPhase 3 trialPrimary efficacy analysisProportion of patientsWithdrawal of consentUnmet medical needComputer-generated scheduleAnti-cancer therapy
2021
IMerge: A phase 3 study to evaluate imetelstat in transfusion-dependent subjects with IPSS low or intermediate-1 risk myelodysplastic syndromes that are relapsed/refractory to erythropoiesis-stimulating agent treatment.
Platzbecker U, Komrokji R, Fenaux P, Zeidan A, Sekeres M, Savona M, Madanat Y, Sherman L, Dougherty S, Sun L, Huang F, Wan Y, Rizo A, Berry T, Feller F, Santini V. IMerge: A phase 3 study to evaluate imetelstat in transfusion-dependent subjects with IPSS low or intermediate-1 risk myelodysplastic syndromes that are relapsed/refractory to erythropoiesis-stimulating agent treatment. Journal Of Clinical Oncology 2021, 39: tps7056-tps7056. DOI: 10.1200/jco.2021.39.15_suppl.tps7056.Peer-Reviewed Original ResearchInternational Prognostic Scoring SystemErythropoiesis-stimulating agentsPhase 3 partRisk myelodysplastic syndromesMyelodysplastic syndromeRBC-TIRed blood cell (RBC) transfusion-dependent patientsErythropoiesis-stimulating agent treatmentLower-risk myelodysplastic syndromesPhase 2 partProgression of MDSRBC transfusion independenceTransfusion dependent subjectsPlacebo-controlled trialPhase 2/3 studyPhase 3 studyPrognostic scoring systemCurrent treatment optionsTransfusion-dependent patientsQuality of lifeMedian TI durationRate of CRVariant allele frequencyMechanism of actionAdult pts