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

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.