<p>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&#xa0;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&#xa0;mg of VEN for a median of 27&#xa0;days, with a median holding period of 12&#xa0;days. With a median follow-up of 13.6&#xa0;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&#xa0;months for the overall cohort and 15.4&#xa0;months for those with prior MDS. The real-world outcomes were comparable to those of clinical trials.</p>

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

  • Ryota Imanaka,
  • Hiroki Numata,
  • Yuna Katsuoka,
  • Nobuhiko Uoshima,
  • Satoru Hara,
  • Jun Ando,
  • Shuichi Ota,
  • Goichi Yoshimoto,
  • Akihito Matsuoka,
  • Tetsuo Morita,
  • Atsuko Tsutsui,
  • Mizuha Kosugi-Kanaya,
  • Tatsunori Goto

摘要

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.