Résumé
Abstract Objectives To compare the efficacy of venetoclax‐azacitidine (VEN–AZA) with AZA in the real‐life for patients with first relapsed or refractory acute myeloid leukaemia (R/R AML). Methods We retrospectively analysed R/R AML patients treated with VEN–AZA at the Institut Paoli Calmettes between September 2020 and February 2022. We compared them to a historical cohort of patients treated with AZA between 2010 and 2021. Results Thirty‐five patients treated with VEN–AZA were compared with 140 patients treated with AZA. There were more favourable cytogenetics (25.7% vs. 8.6%; p = 0.01) and less FLT3‐ITD mutated AML (8.8% vs. 25.5%; p = .049) in the VEN–AZA group. The overall 30‐day mortality rate was 7.4% and the overall 90‐day mortality was 20%, with no difference between the groups. The complete remission rate was 48.6% in the VEN–AZA group versus 15% ( p < .0001). The composite complete response rate was 65.7% in the VEN–AZA group versus 23.6% ( p < .0001). OS was 12.8 months in the VEN–AZA group versus 7.3 months ( p = 0.059). Patients with primary refractory AML, poor‐risk cytogenetics, prior hematopoietic stem‐cell transplantation (HSCT) and FLT3‐ITD mutated AML had lower response and survival rates. Conclusion VEN–AZA was associated with a better response rate and a longer survival than AZA monotherapy in AML patients who relapsed after or were refractory to intensive chemotherapy.