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 studyA 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 AMLSomatic 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 cohort
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 method