# Real-world outcomes of venetoclax and azacitidine in Japanese patients with newly diagnosed acute myeloid leukemia (VENUS study)

## TL;DR

This study examines the real-world effectiveness of venetoclax and azacitidine in Japanese patients with newly diagnosed acute myeloid leukemia.

## Contribution

It provides real-world evidence for venetoclax and azacitidine outcomes in patients with prior MDS or AZA treatment, who are often excluded from clinical trials.

## Key findings

- The complete remission rate was 56.7% in the overall cohort.
- Median overall survival was 14.8 months for the overall cohort.
- Outcomes were comparable to clinical trial results despite prior MDS or AZA treatment.

## Abstract

Venetoclax (VEN) with azacitidine (AZA) is the standard treatment for patients with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy. However, real-world evidence on dosing, scheduling, and outcomes is lacking, particularly for patients with prior myelodysplastic syndrome (MDS) or AZA treatment, who have been excluded from clinical trials. This was a multicenter retrospective study of VEN + AZA in 120 patients newly diagnosed with AML between June 2021 and September 2022. The cohort had a median age of 77 years, 52% had secondary AML, 74% had previously been diagnosed with MDS, and 39% had previously received AZA. During cycle 1, half of the patients received 400 mg of VEN for a median of 27 days, with a median holding period of 12 days. With a median follow-up of 13.6 months, the rate of complete remission (CR) or CR with incomplete blood count recovery was 56.7% in VEN + AZA-treated patients in the overall cohort and 56.5% in patients with prior MDS. Median overall survival was 14.8 months for the overall cohort and 15.4 months for those with prior MDS. The real-world outcomes were comparable to those of clinical trials.

The online version contains supplementary material available at 10.1007/s12185-025-04093-y.