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 studyHealthcare Utilization and Costs Among Patients with Acute Myeloid Leukemia Receiving Oral Azacitidine Maintenance Therapy Versus No Maintenance: A US Claims Database Study
Borate U, Seiter K, Potluri R, Mazumder D, Chevli M, Prebet T, Gaugler L, Strocchia M, Vasconcelos A, Sieluk J. Healthcare Utilization and Costs Among Patients with Acute Myeloid Leukemia Receiving Oral Azacitidine Maintenance Therapy Versus No Maintenance: A US Claims Database Study. Advances In Therapy 2024, 41: 4049-4064. PMID: 39240504, PMCID: PMC11480148, DOI: 10.1007/s12325-024-02947-1.Peer-Reviewed Original ResearchAcute myeloid leukemiaHealthcare resource utilizationOral-AZAPropensity score matchingMyeloid leukemiaReal-world healthcare resource utilizationClinical practiceHematopoietic stem cell transplantationLower healthcare resource utilizationResultsAfter propensity score matchingStem cell transplantationContinuous insurance enrollmentClaims database studyOutpatient visitsOral azacitidinePost-remissionMaintenance therapyProlonged remissionCell transplantationDisease remissionDelayed relapseBaseline characteristicsTreatment patternsPatientsDatabase 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 patientsA post-hoc analysis of outcomes of patients with acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) who received oral azacitidine (Oral-AZA) maintenance therapy in the QUAZAR AML-001 study.
Voso M, De Botton S, Pfeilstöcker M, Figuera Alvarez A, Wang K, L. See W, Ugidos Guerrero M, Lopes de Menezes D, Petrlik E, Prebet T, Roboz G. A post-hoc analysis of outcomes of patients with acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) who received oral azacitidine (Oral-AZA) maintenance therapy in the QUAZAR AML-001 study. Journal Of Clinical Oncology 2024, 42: 6522-6522. DOI: 10.1200/jco.2024.42.16_suppl.6522.Peer-Reviewed Original ResearchRelapse-free survivalHematopoietic stem cell transplantationOral-AZAPoor-risk cytogeneticsAML-MRCOutcomes of patientsMedian OSOverall survivalTreatment armsMedian relapse-free survivalAnalysis of outcomes of patientsProlonged median OSMyelodysplasia-related changesStem cell transplantationPhase 3 studyKaplan-Meier methodAcute myeloid leukemiaEffective treatment optionYears of agePost hoc analysisOral azacitidinePlacebo QDImproved OSMRD negativitySecondary AMLPreliminary 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 endpointDifferentiation syndrome associated with treatment with IDH2 inhibitor enasidenib: pooled analysis from clinical trials
Montesinos P, Fathi A, de Botton S, Stein E, Zeidan A, Zhu Y, Prebet T, Vigil C, Bluemmert I, Yu X, DiNardo C. Differentiation syndrome associated with treatment with IDH2 inhibitor enasidenib: pooled analysis from clinical trials. Blood Advances 2024, 8: 2509-2519. PMID: 38507688, PMCID: PMC11131052, DOI: 10.1182/bloodadvances.2023011914.Peer-Reviewed Original ResearchAcute myeloid leukemiaDevelopment of differentiation syndromeRisk factorsPooled analysisIDH2-mutant acute myeloid leukemiaClinical trialsAcute myeloid leukemia populationMedian time to onsetClinical features of DSBone marrow blastsNon-specific symptomsTime to onsetBaseline risk factorsTreatment of DSSymptoms of DSIdentified risk factorsFeatures of DSMarrow blastsSystemic steroidsPulmonary infiltratesClinical responseMutant IDH2 inhibitorMyeloid leukemiaClinical featuresGrade 3Somatic gene mutation patterns and burden influence outcomes with enasidenib in relapsed/refractory IDH2-mutated AML
Risueño A, See W, Bluemmert I, de Botton S, DiNardo C, Fathi A, Schuh A, Montesinos P, Vyas P, Prebet T, Gandhi A, Hasan M. Somatic gene mutation patterns and burden influence outcomes with enasidenib in relapsed/refractory IDH2-mutated AML. Leukemia Research 2024, 140: 107497. PMID: 38564986, DOI: 10.1016/j.leukres.2024.107497.Peer-Reviewed Original ResearchConceptsConventional care regimensMutational burdenR/R AMLCo-mutationsIDH2-R172Co-mutation patternsAssociated with decreased overall survivalRelapsed/refractory acute myeloid leukemiaTargeted next-generation sequencingAML patient cohortNewly diagnosed AMLLow mutational burdenEvent-free survivalLimited treatment optionsAcute myeloid leukemiaGene mutation patternsIDH2 variantsIDH2 R140Prognostic impactOverall survivalPrognostic relevanceSurvival benefitMyeloid leukemiaIDH2 mutationsPatient cohortCorrigendum to “AML-068 - Survival Differences in Patients With Acute Myeloid Leukemia (AML) Treated With Oral Azacitidine (Oral-AZA) as Maintenance and Those Eligible but Not Treated in a US Electronic Health Record (EHR) Database” [Clinical Lymphoma, Myeloma & Leukemia, 23S1 (2023) S1-S593]
Mims A, Xie Z, Vasconcelos A, Strocchia M, Heydendael W, Chevli M, Rotter D, Potluri R, Prebet T, Sieluk J. Corrigendum to “AML-068 - Survival Differences in Patients With Acute Myeloid Leukemia (AML) Treated With Oral Azacitidine (Oral-AZA) as Maintenance and Those Eligible but Not Treated in a US Electronic Health Record (EHR) Database” [Clinical Lymphoma, Myeloma & Leukemia, 23S1 (2023) S1-S593]. Clinical Lymphoma Myeloma & Leukemia 2024, 24: 203-204. DOI: 10.1016/j.clml.2023.11.010.Peer-Reviewed Original ResearchEFFICACY 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
2023
EE86 Healthcare Resource Utilization (HCRU) and Associated Costs Among Patients with Acute Myeloid Leukemia (AML) Treated with Oral Azacitidine as Maintenance and Those Eligible but Not Treated Using a US Claims Database
Borate U, Seiter K, Potluri R, Mazumder D, Heydendael W, Chevli M, Prebet T, Strocchia M, Vasconcelos A, Sieluk J. EE86 Healthcare Resource Utilization (HCRU) and Associated Costs Among Patients with Acute Myeloid Leukemia (AML) Treated with Oral Azacitidine as Maintenance and Those Eligible but Not Treated Using a US Claims Database. Value In Health 2023, 26: s67. DOI: 10.1016/j.jval.2023.09.358.Peer-Reviewed Original Research
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
2021
Characteristics and Clinical Outcome of Patients with Clonal Cytopenias of Undetermined Significance: A Large Retrospective Multi-Center International Study
Xie Z, Hyun M, Komrokji R, Zeidan A, Madanat Y, Zeidner J, Coombs C, Griffiths E, Lai C, Kishtagari A, Foran J, Badar T, Yi C, Desai P, Ades L, Osman A, Taylor J, Deeg H, Brunner A, Carraway H, Al Ali N, Bewersdorf J, Prebet T, Singh A, Tsai C, Chandhok N, Soong D, Patnaik M, Savona M, Al-Kali A. Characteristics and Clinical Outcome of Patients with Clonal Cytopenias of Undetermined Significance: A Large Retrospective Multi-Center International Study. Blood 2021, 138: 2158. DOI: 10.1182/blood-2021-146254.Peer-Reviewed Original ResearchClinical Trials CommitteeProgression-free survivalIndependent review committeeOverall survivalTrials CommitteeDisease progressionBristol-Myers SquibbMyeloid neoplasmsResponse rateFunctional pathway analysisSpeakers bureauUndetermined significanceMultivariable modelClonal cytopeniaReview CommitteeMedian progression-free survivalBoehringer IngelheimAdvisory CommitteeMulti-centre international studyCG abnormalitiesCox proportional hazards modelGene panelDaiichi SankyoBaseline clinical dataKaplan-Meier methodNCCN Guidelines Insights: Acute Myeloid Leukemia, Version 2.2021.
Pollyea D, Bixby D, Perl A, Bhatt V, Altman J, Appelbaum F, de Lima M, Fathi A, Foran J, Gojo I, Hall A, Jacoby M, Lancet J, Mannis G, Marcucci G, Martin M, Mims A, Neff J, Nejati R, Olin R, Percival M, Prebet T, Przespolewski A, Rao D, Ravandi-Kashani F, Shami P, Stone R, Strickland S, Sweet K, Vachhani P, Wieduwilt M, Gregory K, Ogba N, Tallman M. NCCN Guidelines Insights: Acute Myeloid Leukemia, Version 2.2021. Journal Of The National Comprehensive Cancer Network 2021, 19: 16-27. PMID: 33406488, DOI: 10.6004/jnccn.2021.0002.Peer-Reviewed Original ResearchConceptsAcute myeloid leukemiaNCCN GuidelinesFavorable-risk acute myeloid leukemiaLow-risk acute promyelocytic leukemiaVenetoclax-based therapyNCCN Guidelines InsightsClinical decision-makingTreatment of adultsAcute promyelocytic leukemiaPrognostic importanceBlood transfusionMyeloid leukemiaGenetic alterationsTreatment strategiesClinical trialsPromyelocytic leukemiaNCCNLeukemiaTreatmentGuidelinesDecision-makingPhysiciansPostremissionTransfusionTherapy
2019
Acute Myeloid Leukemia, Version 3.2019, NCCN Clinical Practice Guidelines in Oncology.
Tallman M, Wang E, Altman J, Appelbaum F, Bhatt V, Bixby D, Coutre S, De Lima M, Fathi A, Fiorella M, Foran J, Hall A, Jacoby M, Lancet J, LeBlanc T, Mannis G, Marcucci G, Martin M, Mims A, O'Donnell M, Olin R, Peker D, Perl A, Pollyea D, Pratz K, Prebet T, Ravandi F, Shami P, Stone R, Strickland S, Wieduwilt M, Gregory K, Hammond L, Ogba N. Acute Myeloid Leukemia, Version 3.2019, NCCN Clinical Practice Guidelines in Oncology. Journal Of The National Comprehensive Cancer Network 2019, 17: 721-749. PMID: 31200351, DOI: 10.6004/jnccn.2019.0028.Peer-Reviewed Original ResearchMeSH KeywordsAge FactorsAgedAntineoplastic Combined Chemotherapy ProtocolsBiomarkers, TumorCytogenetic AnalysisDisease-Free SurvivalGraft vs Host DiseaseHematopoietic Stem Cell TransplantationHistocompatibility TestingHLA AntigensHumansLeukemia, Myeloid, AcuteMedical OncologyMiddle AgedRemission InductionRisk AssessmentTransplantation, HomologousUnited StatesConceptsAcute myeloid leukemiaNCCN Clinical Practice GuidelinesClinical practice guidelinesMyeloid leukemiaTreatment optionsManagement of acute myeloid leukemiaLow-intensity regimensExpansion of treatment optionsAnnual deaths due to leukemiasPractice guidelinesDeaths due to leukemiaAcute leukemiaTargeted therapyAdult patientsDiagnostic evaluationLeukemiaNCCNOptionsRegimensUnited StatesTherapyWorkupPatientsGuidelines
2015
French consensus on myelodysplasic syndrome and chronic myelomonocytic leukemia: diagnostic, classification and treatment 2015 update by the Myelodysplasia French Group
Fenaux G, Ades L, Fontenay M, Raynaud S, Eclache V, Rose C, Guerci-Bresler A, Gyan E, Robin M, Prebet T, Itzykson R, Cluzeau T, Natarajan-Amé S, Stamatoullas A, Wattel E, Park S, Beyne Rauzy O, Solary E, Bordessoule D, Isnard F, Quesnel B, Yakoub Agha I, Toma A, Thépot S, Braun T, Gardin C, Chèze S, Delaunay J, Dimicoli S, Kosmider O, Renneville A, Preudhomme C, Chermat F, Vey N, Dreyfus F. French consensus on myelodysplasic syndrome and chronic myelomonocytic leukemia: diagnostic, classification and treatment 2015 update by the Myelodysplasia French Group. Hématologie 2015, 21: 28-45. DOI: 10.1684/hma.2015.0979.Peer-Reviewed Original Research