Labrador, JorgeSaiz-Rodríguez, Miriamde Miguel, Duniade Laiglesia, AlmudenaRodríguez-Medina, CarlosVidriales, María BelénPérez-Encinas, ManuelSánchez-Sánchez, María JoséCuello, RebecaRoldán-Pérez, AliciaVives, SusanaBenzo-Callejo, GonzaloColorado, MercedesGarcía-Fortes, MaríaSayas, María JoséOlivier, CarmenRecio, IsabelConde-Royo, DiegoBienert-García, ÁlvaroVahi, MaríaMuñoz-García, CarmenSeri-Merino, CristinaTormo, MarVall-Llovera, FerranFoncillas, María-ÁngelesMartínez-Cuadrón, DavidSanz, Miguel ÁngelMontesinos, Pau2023-05-032023-05-032022-03-292072-6694http://hdl.handle.net/10668/20882The effectiveness of venetoclax (VEN) in relapsed or refractory acute myeloid leukemia (RR-AML) has not been well established. This retrospective, multicenter, observational database studied the effectiveness of VEN in a cohort of 51 RR-AML patients and evaluated for predictors of response and overall survival (OS). The median age was 68 years, most were at high risk, 61% received ≥2 therapies for AML, 49% had received hypomethylating agents, and ECOG was ≥2 in 52%. Complete remission (CR) rate, including CR with incomplete hematological recovery (CRi), was 12.4%. Additionally, 10.4% experienced partial response (PR). The CR/CRi was higher in combination with azacitidine (AZA; 17.9%) than with decitabine (DEC; 6.7%) and low-dose cytarabine (LDAC; 0%). Mutated NPM1 was associated with increased CR/CRi. Median OS was 104 days (95% CI: 56-151). For the combination with AZA, DEC, and LDAC, median OS was 120 days, 104 days, and 69 days, respectively; p = 0.875. Treatment response and ECOG 0 influenced OS in a multivariate model. A total of 28% of patients required interruption of VEN because of toxicity. Our real-life series describes a marginal probability of CR/CRi and poor OS after VEN-based salvage. Patients included had very poor-risk features and were heavily pretreated. The small percentage of responders did not reach the median OS.enAttribution 4.0 Internationalhttp://creativecommons.org/licenses/by/4.0/acute myeloid leukemiarefractoryrelapsedvenetoclaxresearch article35406512open access10.3390/cancers14071734PMC8997036https://www.mdpi.com/2072-6694/14/7/1734/pdf?version=1648551807https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8997036/pdf