2024
Effect of Prior Treatments on the Clinical Activity of Imetelstat in Transfusion-Dependent Patients with Erythropoiesis-Stimulating Agent, Relapsed or Refractory/Ineligible Lower-Risk Myelodysplastic Syndromes
Platzbecker U, Santini V, Zeidan A, Sekeres M, Fenaux P, Raza A, Mittelman M, Thépot S, Buckstein R, Germing U, Madanat Y, Diez-Campelo M, Valcarcel D, Jonasova A, Dougherty S, Shah S, Xia Q, Sun L, Navada S, Savona M, Komrokji R. Effect of Prior Treatments on the Clinical Activity of Imetelstat in Transfusion-Dependent Patients with Erythropoiesis-Stimulating Agent, Relapsed or Refractory/Ineligible Lower-Risk Myelodysplastic Syndromes. Blood 2024, 144: 352-352. DOI: 10.1182/blood-2024-203612.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesHematological improvement-erythroidErythropoiesis-stimulating agentsErythropoiesis-stimulating agent therapyHb riseRBC-TIHypomethylating agentsTransfusion reductionClinical activityMyelodysplastic syndromeQTc studyRed blood cellsPrior treatmentInternational Working GroupRBC transfusion independenceHypomethylating agent treatmentTransfusion-dependent patientsPhase 2/3 trialsLimited treatment optionsUnited States Food and Drug AdministrationStates Food and Drug AdministrationFirst-in-classFood and Drug AdministrationRBC-TDLenalidomide treatment
2020
Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes
Fenaux P, Platzbecker U, Mufti GJ, Garcia-Manero G, Buckstein R, Santini V, Díez-Campelo M, Finelli C, Cazzola M, Ilhan O, Sekeres MA, Falantes JF, Arrizabalaga B, Salvi F, Giai V, Vyas P, Bowen D, Selleslag D, DeZern AE, Jurcic JG, Germing U, Götze KS, Quesnel B, Beyne-Rauzy O, Cluzeau T, Voso MT, Mazure D, Vellenga E, Greenberg PL, Hellström-Lindberg E, Zeidan AM, Adès L, Verma A, Savona MR, Laadem A, Benzohra A, Zhang J, Rampersad A, Dunshee DR, Linde PG, Sherman ML, Komrokji RS, List AF. Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes. New England Journal Of Medicine 2020, 382: 140-151. PMID: 31914241, DOI: 10.1056/nejmoa1908892.Peer-Reviewed Original ResearchConceptsLower-risk myelodysplastic syndromesRed cell transfusionKey secondary end pointRegular red cell transfusionsSecondary end pointsMyelodysplastic syndromeTransfusion independenceAdverse eventsWeek 1Placebo groupEnd pointRing sideroblastsErythropoiesis-stimulating agent therapyIntermediate-risk myelodysplastic syndromesPrimary end pointPhase 2 studyPhase 3 trialErythropoiesis-stimulating agentsSeverity of anemiaGrowth factor βBaseline characteristicsAgent therapyLuspaterceptPatientsSyndrome