Allogeneic hematopoietic stem cell transplantation after azacitidine and venetoclax salvage in relapsed/refractory AML: a multicenter real-world study by the French AURAML group

Fit relapsed/refractory (R/R) acute myeloid leukemia (AML) patients usually undergo intensive chemotherapy (IC)-based salvage to bridge them to allogeneic hematopoietic stem cell transplantation (HSCT), but their prognosis remains poor. Azacitidine and venetoclax (AZA/VEN) are increasingly used as salvage therapy in R/R AML with encouraging results, although data remain limited. In this study, we evaluated the post-HSCT outcomes of 75 R/R AML patients from the VENAURA registry who underwent HSCT after AZA/VEN salvage. After a median follow-up of 16.9 months, the estimated 2-year overall survival (OS) was 61.4% (95% confidence interval [CI]: 49.5-68.1%). The 2-year cumulative incidence of relapse (CIR) was 35.1% (95% CI: 20-50.2%). The estimated 2-year non-relapse mortality (NRM) rate was 10.6% (95% CI: 9.8-23.3%). Cytological response at the end of cycle 1 was independently associated with OS and CIR in multivariate analysis. Comparison with 75 pair-matched patients receiving IC-based salvage prior to HSCT revealed similar OS in both groups. CIR was not significantly higher in AZA/VEN-treated compared to IC-treated patients; however, there was a trend toward a lower 2-year NRM rate in the AZA/VEN group. Our data suggest that AZA/VEN represents a feasible bridge-to-transplant option with a favorable toxicity profile.