Azacitidine plus venetoclax for maintenance treatment in intermediate‑to‑low‑risk acute myeloid leukemia: A comparative analysis of clinical outcomes in an observational cohort

Abstract

The combination of azacitidine (AZA) and venetoclax (VEN) for maintenance treatment in patients with intermediate-to-low-risk acute myeloid leukemia (AML) is a contentious issue. The aim of the present study was to investigate the relationship between the use of VEN plus AZA (VEN-AZA) and the relapse rate of intermediate-to-low-risk AML among adult Chinese participants. The primary endpoint was AML relapse, analyzed using time-to-event methods. A multivariate Cox proportional hazards model was used to compare outcomes between patients who continued VEN-AZA for maintenance treatment and those who discontinued such therapy. Among the 43 patients, 22 (51.1%) received VEN-AZA with treatment cycles every 2-3 months, while the remaining 21 patients discontinued maintenance therapy. The median ages for the two groups were 59 and 49 years, respectively, with an age range of 21-81 years. With a median follow-up of 29.6 months (range 7-74), the median progression-free survival (PFS) was not reached in the maintenance therapy group and was 47.3 months in the group that discontinued treatment. The number of grade 3-4 adverse events was low in the maintenance group, with neutropenia and thrombocytopenia as the primary hematological adverse events, and respiratory infection as the main non-hematological adverse event. Univariate analysis indicated that age, white blood cell count and maintenance therapy were associated with AML relapse. After adjusting for confounding factors, multivariate Cox proportional hazards model showed that maintenance treatment was associated with a reduced risk of relapse and a longer PFS time, compared with discontinued treatment (hazard ratio, 0.06; 95% confidence interval, 0-0.77). Therefore, patients treated with VEN-AZA exhibited a longer PFS time, suggesting that further clinical trials are warranted.

Keywords: acute myeloid leukemia; azacitidine; maintenance treatment; progression-free survival; venetoclax.

Figure 1.

(A) Relationship between PFS and WBC count. (B) PFS in age groups above and below 60 years. (C) PFS in the low-risk and intermediate risk groups. (D) PFS according to VEN-AZA maintenance therapy. PFS, progression-free survival; WBC, white blood cell; VEN, venetoclax; AZA, azacitidine.