2024
Lenalidomide in Transfusion-Dependent IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes and Isolated Del(5q): Results of a European Post-Authorization Safety Surveillance Study
Poloni A, Raaschou-Jensen K, Mohedo F, Paolini S, Oliva E, Buccisano F, Vasconcelos A, Kim I, Makwana A, Bernasconi D, Rosettani B, Prebet T, Santini V. Lenalidomide in Transfusion-Dependent IPSS Low- or Intermediate-1-Risk Myelodysplastic Syndromes and Isolated Del(5q): Results of a European Post-Authorization Safety Surveillance Study. Clinical Lymphoma Myeloma & Leukemia 2024 DOI: 10.1016/j.clml.2024.10.007.Peer-Reviewed Original ResearchInternational Prognostic Scoring SystemDose of lenalidomideMyelodysplastic syndromeIsolated del(5qAcute myeloid leukemiaBenefit-risk profileSafety populationTransfusion-dependentOverall survivalCumulative incidenceCumulative incidence of acute myeloid leukemiaGrade 3/4 treatment-emergent adverse eventsNon-interventional post-authorization safety studyInternational Prognostic Scoring System low-Intermediate-1-risk myelodysplastic syndromeIncidence of acute myeloid leukemiaLower-risk myelodysplastic syndromesTreatment-emergent adverse eventsEffects of lenalidomidePrognostic Scoring SystemKaplan-Meier analysisPost-authorization safety studyRoutine careClinical trial dataSafety surveillance studyLuspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): primary analysis of a phase 3, open-label, randomised, controlled trial
Della Porta M, Garcia-Manero G, Santini V, Zeidan A, Komrokji R, Shortt J, Valcárcel D, Jonasova A, Dimicoli-Salazar S, Tiong I, Lin C, Li J, Zhang J, Pilot R, Kreitz S, Pozharskaya V, Keeperman K, Rose S, Prebet T, Lai Y, Degulys A, Paolini S, Cluzeau T, Fenaux P, Platzbecker U. Luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): primary analysis of a phase 3, open-label, randomised, controlled trial. The Lancet Haematology 2024, 11: e646-e658. PMID: 39038479, DOI: 10.1016/s2352-3026(24)00203-5.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesRed blood cell transfusion independenceTreatment-emergent adverse eventsMedian follow-upEpoetin alfa groupMyelodysplastic syndromeLuspatercept groupTransfusion-dependentSerum erythropoietin concentrationPrimary endpointEpoetin alfaTransfusion independenceOpen-labelAlfa groupAdverse eventsFollow-upRed blood cell transfusion burdenErythropoietin concentrationIntention-to-treat populationControlled trialsCommon grade 3Epoetin alfa recipientsMean haemoglobin increasePrimary analysisProportion of patientsPreliminary safety and efficacy of oral azacitidine (Oral-AZA) in patients (pts) with low-/Intermediate (Int)-risk myelodysplastic syndromes (MDS): Phase 2 results from the ASTREON trial.
Garcia-Manero G, Yee K, Hernandez F, Della Porta M, Paolini S, Ahn S, Santini V, Fenaux P, Suzuki T, Sekeres M, He J, Li J, Barkalifa R, Vigil C, Prebet T, Lopes de Menezes D, Burnett J, Komrokji R, Giagounidis A. Preliminary safety and efficacy of oral azacitidine (Oral-AZA) in patients (pts) with low-/Intermediate (Int)-risk myelodysplastic syndromes (MDS): Phase 2 results from the ASTREON trial. Journal Of Clinical Oncology 2024, 42: 6509-6509. DOI: 10.1200/jco.2024.42.16_suppl.6509.Peer-Reviewed Original ResearchLower-risk MDSOral-AZAMyelodysplastic syndromeAdverse eventsPreliminary efficacy dataComplete remissionEfficacy dataOverall responseLower-risk myelodysplastic syndromesHigher-risk myelodysplastic syndromesIPSS-R scoreRBC transfusion burdenRate of adverse eventsDose-optimization studyErythropoiesis-stimulating agentsSerious adverse eventsAcute myeloid leukemiaMITT populationOral azacitidineTransfusion burdenIPSS-ROpen-labelEligible ptsPhase 2/3Primary endpointEFFICACY AND SAFETY OF LUSPATERCEPT VERSUS EPOETIN ALFA IN ERYTHROPOIESIS-STIMULATING AGENT (ESA)-NAIVE PATIENTS WITH TRANSFUSION-DEPENDENT LOWER-RISK MYELODYSPLASTIC SYNDROMES (LR-MDS): FULL ANALYSIS OF THE COMMANDS TRIAL
Garcia-Manero G, Platzbecker U, Santini V, Zeidan A, Fenaux P, Komrokji R, Shortt J, Valcarcel D, Jonasova A, Dimicoli-Salazar S, Tiong I, Lin C, Li J, Zhang J, Giuseppi A, Kreitz S, Pozharskaya V, Keeperman K, Rose S, Prebet T, Degulys A, Paolini S, Cluzeau T, Della Porta M. EFFICACY AND SAFETY OF LUSPATERCEPT VERSUS EPOETIN ALFA IN ERYTHROPOIESIS-STIMULATING AGENT (ESA)-NAIVE PATIENTS WITH TRANSFUSION-DEPENDENT LOWER-RISK MYELODYSPLASTIC SYNDROMES (LR-MDS): FULL ANALYSIS OF THE COMMANDS TRIAL. Leukemia Research Reports 2024, 21: 100447. DOI: 10.1016/j.lrr.2024.100447.Peer-Reviewed Original ResearchLower-risk myelodysplastic syndromesTreatment-emergent adverse eventsEA-treated patientsRBC-TIPrimary endpointHI-ERed blood cell transfusion independenceHematological improvement-erythroidTransfusion independenceErythroid responseMyelodysplastic syndromeSecondary endpointsAdverse eventsFull analysisLuspaterceptAssessed efficacySafety resultsEpoetin alfaTreatment durationPatientsEndpointEfficacyDurationPost-treatmentAML
2022
Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial
Watts J, Baer M, Yang J, Prebet T, Lee S, Schiller G, Dinner S, Pigneux A, Montesinos P, Wang E, Seiter K, Wei A, De Botton S, Arnan M, Donnellan W, Schwarer A, Récher C, Jonas B, Ferrell P, Marzac C, Kelly P, Sweeney J, Forsyth S, Guichard S, Brevard J, Henrick P, Mohamed H, Cortes J. Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial. The Lancet Haematology 2022, 10: e46-e58. PMID: 36370742, DOI: 10.1016/s2352-3026(22)00292-7.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaInhibitor of mutant isocitrate dehydrogenase 1Recommended phase 2 doseIDH1-mutated acute myeloid leukemiaPhase 2 doseDose-limiting toxicityCombination therapy groupMyeloid leukemiaCombination therapyMyelodysplastic syndromeClinical activityFebrile neutropeniaMonotherapy groupSmall-molecule inhibitor of mutant isocitrate dehydrogenase 1Eastern Cooperative Oncology Group performance status 0Treatment-emergent adverse eventsHigh-risk myelodysplastic syndromeRefractory acute myeloid leukemiaOpen-label clinical trialTherapy groupDose-escalation cohortsPerformance status 0Phase 1/2 studyTreatment-naive patientsPhase 1/2 trial