Stahl, MaximilianDeVeaux, MichelleMontesinos, PauItzykson, RaphaelRitchie, Ellen KSekeres, Mikkael ABarnard, John DPodoltsev, Nikolai ABrunner, Andrew MKomrokji, Rami SBhatt, Vijaya RAl-Kali, ArefCluzeau, ThomasSantini, ValeriaFathi, Amir TRoboz, Gail JFenaux, PierreLitzow, Mark RPerreault, SarahKim, Tae KonPrebet, ThomasVey, NorbertVerma, VivekGerming, UlrichBergua, Juan MiguelSerrano, JosefinaGore, Steven DZeidan, Amer M2023-01-252023-01-252018-03-13Stahl M, DeVeaux M, Montesinos P, Itzykson R, Ritchie EK, Sekeres MA, et al. Hypomethylating agents in relapsed and refractory AML: outcomes and their predictors in a large international patient cohort. Blood Adv. 2018 Apr 24;2(8):923-932.http://hdl.handle.net/10668/12380Although hypomethylating agents (HMAs) are frequently used in the frontline treatment of older acute myeloid leukemia (AML) patients, little is known about their effectiveness in relapsed or primary treatment-refractory (RR)-AML. Using an international multicenter retrospective database, we studied the effectiveness of HMAs in RR-AML and evaluated for predictors of response and overall survival (OS). A total of 655 patients from 12 centers received azacitidine (57%) or decitabine (43%), including 290 refractory (44%) and 365 relapsed (56%) patients. Median age at diagnosis was 65 years. Best response to HMAs was complete remission (CR; 11%) or CR with incomplete count recovery (CRi; 5.3%). Additionally, 8.5% experienced hematologic improvement. Median OS was 6.7 months (95% confidence interval, 6.1-7.3). As expected, OS differed significantly by best response, with patients achieving CR and CRi having a median OS of 25.3 and 14.6 months, respectively. In multivariate analysis, the presence of ≤5% circulating blasts and a 10-day schedule of decitabine were associated with improved response rates, whereas the presence of >5% circulating blasts and >20% bone marrow blasts were associated with decreased OS. A significant subset of RR-AML patients (16%) achieved CR/CRi with HMAs and experienced a median OS of 21 months. Outside of a clinical trial, HMAs represent a reasonable therapeutic option for some patients with RR-AML.enMyeloid NeoplasiaClinical Trials and ObservationsAntimetabolitos antineoplásicosAdolescentAdultAgedAged, 80 and overAntimetabolites, AntineoplasticCohort StudiesDNA MethylationDatabases, FactualDecitabineHumansLeukemia, Myeloid, AcuteMiddle AgedPrognosisRemission InductionRetrospective StudiesSalvage TherapySurvival AnalysisTreatment OutcomeYoung Adultresearch article29685952open accessAdolescenteAnálisis de supervivenciaBases de datos factualesDecitabinaEstudios retrospectivosEstudios de cohortesInducción de remisiónLeucemia mieloide agudaMetilación de ADNPronósticoResultado del tratamientoTerapia recuperativa10.1182/bloodadvances.20180161212473-9537PMC5916007https://ashpublications.org/bloodadvances/article-pdf/2/8/923/880924/advances016121.pdfhttps://www.ncbi.nlm.nih.gov/pmc/articles/PMC5916007/pdf