Background: The recently issued ELN 2024 Less-Intensive risk stratification aims to refine prognosis for adults with acute myeloid leukemia (AML) treated with lower-intensity regimens such as venetoclax plus azacitidine (VA). Real-world validation in Asian populations is limited. We assessed the prognostic performance of ELN 2024 versus ELN 2022 in a Chinese cohort.

Methods: We retrospectively reviewed 53 newly diagnosed AML patients deemed unfit for intensive chemotherapy who received continuous VA (venetoclax 400 mg qd on days 1-28; azacitidine 75 mg/m² qd on days 1-7, every 28 days) from August 2020 to June 2024. Patients were stratified by both ELN 2024 Less-Intensive and ELN 2022 criteria. Primary endpoints were overall survival (OS) and relapse-free survival (RFS). Secondary analyses included composite complete remission (cCR), measurable residual disease (MRD) status, and mutation impact.

Results: Median age was 65 years (range, 20-78); median follow-up, 27.6 months. Under ELN 2024, 15 (28.3 %) patients were favorable, 27 (50.9 %) intermediate, and 11 (20.8 %) adverse. Median OS differed significantly: 39.6, 25.0, and 6.6 months, respectively (log-rank P = 0.025). ELN 2022 failed to discriminate OS (25.4, 33.9, and 25.4 months; P = 0.857). MRD negativity after 1–2 induction cycles (41/53, 77.3 %) predicted longer RFS (P = 0.009). Five of six patients (83 %) harboring PTPN11 mutations relapsed within six months, suggesting adverse prognostic significance.

Conclusions: In this real-world Chinese cohort, ELN 2024 Less-Intensive stratification clearly separated survival outcomes and outperformed ELN 2022 in VA-treated AML. MRD negativity remained a robust predictor of favorable RFS, and PTPN11 mutations were associated with early relapse. Larger multicenter studies are warranted.

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2025
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