Venetoclax + high-dose cytarabine + mitoxantrone salvage in R/R AML: A real-world analysis

Results from a multicenter, retrospective, real-world analysis evaluating the addition of venetoclax to high-dose cytarabine + mitoxantrone (HAM) in 128 adults with relapsed/refractory (R/R) acute myeloid leukemia (AML) were published in Haematologica by Berdel et al. The co-primary endpoints were the composite complete remission (CR) / CR with incomplete hematologic recovery (CRi) rate, relapse-free survival (RFS), and overall survival (OS). 

Key data: Overall, 69% of patients achieved CR/CRi (CR, 30%; CRi, 39%). Patients treated with 1 prior line of therapy had a CR/CRi rate of 78%, compared with 48% in those who had received ≥2 lines. At a median follow-up of 11.5 months, the median RFS among patients who achieved CR/CRi was not reached; the 1-year RFS rate was 59%. The estimated 1-year OS rate was 58%. Infectious complications were the most common adverse event (AE), occurring in 70% of patients. Mortality rates at Day 30 and Day 60 were 6% and 12%, respectively. Overall, 68% of patients proceeded to allogeneic hematopoietic cell transplantation (allo-HSCT).

Key learning: Venetoclax + HAM demonstrated encouraging real-world efficacy in patients with R/R AML, supporting its potential both as salvage therapy and as a bridge-to-transplant.