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 therapyImprovement of Patient-Reported Outcomes Among Heavily Pretreated Patients with Lower-Risk Myelodysplastic Syndromes and High Transfusion Burden Treated with Imetelstat on the IMerge Phase 3 Trial
Sekeres M, Diez-Campelo M, Zeidan A, Platzbecker U, Regnault A, Creel K, Sengupta N, Wan Y, Sun L, Xia Q, Berry T, Dougherty S, Shah S, Navada S, Santini V, Valcárcel D. Improvement of Patient-Reported Outcomes Among Heavily Pretreated Patients with Lower-Risk Myelodysplastic Syndromes and High Transfusion Burden Treated with Imetelstat on the IMerge Phase 3 Trial. Blood 2023, 142: 6479. DOI: 10.1182/blood-2023-181103.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesPatient-reported outcomesHigh transfusion burdenTransfusion burdenPlacebo groupMyelodysplastic syndromeTreatment groupsFunctional assessmentCancer Therapy-AnemiaPatient-reported fatigueTransfusion independence rateChronic Illness TherapyPhase 3 trialInferior overall survivalTransfusion-dependent anemiaLeast square meansImprovement of patientsPRO end pointsComposite scorePositive mean changesQuality of lifeFACT-AnTreat populationDyspnea scoreOverall survival
2019
Clinical Effectiveness of Hypomethylating Agents (HMAs) and Lenalidomide (Len) in Older Patients (pts) with Refractory Anemia with Ring Sideroblasts: A Large Population-Based Study in the United States (US)
Wang X, Wang R, Zhang C, Zeidan A, Podoltsev N, Huntington S, Gore S, Davidoff A, Ma X. Clinical Effectiveness of Hypomethylating Agents (HMAs) and Lenalidomide (Len) in Older Patients (pts) with Refractory Anemia with Ring Sideroblasts: A Large Population-Based Study in the United States (US). Blood 2019, 134: 4748. DOI: 10.1182/blood-2019-128821.Peer-Reviewed Original ResearchPopulation-based studyLR-MDSMedian overall survivalOverall survivalClinical effectivenessHypomethylating agentCelgene CorporationMedian durationRed blood cellsMedian timeRing sideroblastsBoehringer IngelheimAdvisory CommitteeLarge population-based studyDaiichi SankyoOlder adultsAbnormal erythroid precursorsTransfusion independence rateEnd Results-MedicareHigh-risk MDSErythropoiesis-stimulating agentsKaplan-Meier statisticsComparative clinical effectivenessEnd of studyPart D coverage