Venetoclax Plus Azacitidine Beneficial for Patients with Untreated AML Ineligible for Intensive Chemotherapy

By Leah Sherwood - Last Updated: December 27, 2022

Long-term follow-up from the phase III VIALE-A trial demonstrated sustained overall survival (OS) benefit with venetoclax in combination with azacitidine in patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy compared to placebo plus azacitidine in all subgroups.

The VIALE-A trial results were presented at the 2022 American Society of Hematology Annual Meeting and Exposition in a poster session by Keith W. Pratz, MD, of the Abramson Cancer Center at the University of Pennsylvania, and colleagues. The results represent an analysis of the VIALE-A data after the occurrence of 100% of the pre-planned survival events.

In the VIALE-A trial, 431 patients with confirmed AML who were previously untreated and ineligible for intensive therapy were randomly assigned 2:1 to azacitidine plus either venetoclax (n=286) or placebo (n=145). All patients received a standard dose of azacitidine 75 mg/m2 subcutaneously or intravenously on days one to seven of every 28-day cycle. Venetoclax 400 mg after a three-day ramp up to reach the target dose in cycle one or a matching placebo was administered orally, once daily, in 28-day cycles.

The median age was 76 years in both groups. Molecular abnormalities of interest included FLT3, observed in 14.1% of patients receiving venetoclax plus azacitidine; IDH1/2, observed in 24.9% of patients; TP53, observed in 23.3% of patients; and NPM1, observed in 16.6% of patients.

With a median follow-up of 43.2 months, the median OS was 14.7 months (95% CI, 12.1-18.7) in the venetoclax plus azacitidine group and 9.6 months (95% CI, 7.4-12.7) in the placebo plus azacitidine group (hazard ratio, 0.58; 95% CI, 0.47-0.72; nominal P<.001).

The median OS for patients with measurable residual disease (<10-3) who had achieved complete remission plus complete remission with incomplete hematologic recovery was 34.2 months, and median OS for patients with IDH1/2 mutations treated with venetoclax plus azacitidine was 19.9 months.

“The VIALE-A [two]-year follow-up analysis confirms the long-term survival benefit for patients treated with [venetoclax plus azacitidine], with no new safety findings,” the authors wrote.


Pratz K, Jonas B, Pullarkat V, et al. Long-term follow-up of the phase 3 VIALE-A clinical trial of venetoclax plus azacitidine for patients with untreated acute myeloid leukemia ineligible for intensive chemotherapy. Abstract #219. Presented at the 64th American Society of Hematology Annual Meeting, December 10-13, 2022; New Orleans, Louisiana.

Editorial Board