Comparison of venetoclax and azacitidine versus a cytarabine-based treatment for patients with acute myeloid leukaemia in first relapse after chemotherapy. A real-world study of the DATAML-IPC-AURAML consortium.

Complete remission (CR) rates for patients with acute myeloid leukaemia (AML) treated with intensive chemotherapy (IC) have increasingly improved. Yet, relapse remains a concern with a dismal prognosis. In this report, results are presented of a retrospective analysis comparing salvage therapy based on intermediate (I) or high (H)-dose cytarabine (DAC) (n = 203) and venetoclax-azacitidine (VEN-AZA) (n = 114) for patients with AML in first relapse. Patients (median age 60 years, interquartile range [IQR] 48-67) had reached CR1 after a standard 7 + 3 (n = 305) or CPX351 schedule (n = 12). I/HDAC-based and VEN-AZA salvage, respectively, resulted in CR2 in 66.0% and 66.7% of the patients (p = 0.96). Allogeneic haematopoietic stem cell transplantation (allo-HSCT) was performed for 115 patients in CR2 and 18 in failure. Relapse-free survival was 13.2 (IQR 7.0-not reached [NR]) and 13.7 (IQR 7.8-NR) months while overall survival (OS) was 14.5 (IQR 6.2-NR) and 12.3 months (IQR 4.8-NR; p = 0.41) for patients who received I/HDAC-based salvage and VEN-AZA respectively. In multivariable analysis, relapsing patients ≥60 years with low/intermediate-risk cytogenetics and VEN-AZA had a better OS (p = 0.002), while I/HDAC was more efficient for those with poor risk cytogenetics and < 60-year-old (p = 0.002). VEN-AZA salvage therapy thus appears promising compared with conventional I/HDAC-based IC; this question warrants evaluation in randomized studies.