Annals of hematology · 2026
WT1 mRNA in peripheral blood enables early prognostic stratification in AML patients receiving venetoclax and azacitidine therapy.
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Sugiura Hiroyuki, Asada Noboru, Asano Takeru, Hiramatsu Yasushi, Urata Tomohiro, Imai Toshi, Nakamura Makoto, Yamamoto Kazuhiko, Ishikawa Tatsunori, Makita Masanori, Yoshioka Takanori, Sunami Kazutaka, Saeki Kyosuke, Nawa Yuichiro, Kimura Maiko, Takeuchi Makoto, Abe Masaya, Asakura Shoji, Yano Tomofumi, Maeda Yoshinobu
Аннотация
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.