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<p>Venetoclax and azacitidine (VEN/AZA), which target BCL-2 and DNA methylation, have demonstrated substantial efficacy, particularly in older or unfit patients with acute myeloid leukemia (AML). <i>Wilms’ Tumor-1</i> (<i>WT1</i>) mRNA, measurable in peripheral blood (PB), is an established biomarker for monitoring treatment response and predicting AML prognosis. We conducted a retrospective analysis of patients with AML treated with VEN/AZA within the Okayama Hematology Study Group (OHSG). Patients who showed a marked reduction in PB <i>WT1</i> mRNA levels from baseline after the first or second treatment cycle had significantly improved overall survival (OS) and progression-free survival (PFS). Moreover, achieving PB <i>WT1</i> mRNA negativity-regardless of whether this occurred early or later in the therapy།was consistently associated with superior OS and PFS. These findings suggest that PB <i>WT1</i> mRNA is a sensitive and reliable biomarker for predicting treatment response and long-term outcomes in patients with AML receiving VEN/AZA.</p>

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WT1 mRNA in peripheral blood enables early prognostic stratification in AML patients receiving venetoclax and azacitidine therapy

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月9日

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  • Hiroyuki Sugiura,
  • Noboru Asada,
  • Takeru Asano,
  • Yasushi Hiramatsu,
  • Tomohiro Urata,
  • Toshi Imai,
  • Makoto Nakamura,
  • Kazuhiko Yamamoto,
  • Tatsunori Ishikawa,
  • Masanori Makita,
  • Takanori Yoshioka,
  • Kazutaka Sunami,
  • Kyosuke Saeki,
  • Yuichiro Nawa,
  • Maiko Kimura,
  • Makoto Takeuchi,
  • Masaya Abe,
  • Shoji Asakura,
  • Tomofumi Yano,
  • Yoshinobu Maeda

摘要

Venetoclax and azacitidine (VEN/AZA), which target BCL-2 and DNA methylation, have demonstrated substantial efficacy, particularly in older or unfit patients with acute myeloid leukemia (AML). Wilms’ Tumor-1 (WT1) mRNA, measurable in peripheral blood (PB), is an established biomarker for monitoring treatment response and predicting AML prognosis. We conducted a retrospective analysis of patients with AML treated with VEN/AZA within the Okayama Hematology Study Group (OHSG). Patients who showed a marked reduction in PB WT1 mRNA levels from baseline after the first or second treatment cycle had significantly improved overall survival (OS) and progression-free survival (PFS). Moreover, achieving PB WT1 mRNA negativity-regardless of whether this occurred early or later in the therapy།was consistently associated with superior OS and PFS. These findings suggest that PB WT1 mRNA is a sensitive and reliable biomarker for predicting treatment response and long-term outcomes in patients with AML receiving VEN/AZA.