2024
Somatic 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
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