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Prognostic value of MRD response in patients with AML treated with Ven-HMA: Validation of ELN-2021 MRD recommendations

By Sheetal Bhurke

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Jun 18, 2025

Learning objective: After reading this article, learners will be able to cite a new clinical development in acute myeloid leukemia.


 

 

Measurable residual disease (MRD) has become an important dynamic biomarker in AML, providing additional prognostic benefit beyond disease genetics and standard response assessment. However, the role of MRD assessment in low-intensity Ven-based regimens remains less clearly defined. A real-world retrospective study evaluating the impact of MRD response according to the ELN 2021 recommendations on survival outcomes in patients with AML treated with Ven-HMA has been published by Jimenez-Vincente et al. in the Blood Cancer Journal.1 

A total of 99 patients diagnosed with AML according to the ICC 2022 and WHO 5th edition classifications and stratified according to ELN 2021 risk classification who achieved an mCR after two cycles of Ven-HMA were analyzed. The study comprised an MRD-negative (n = 52) and an MRD-positive cohort (n = 47). The treatment schedule was Ven in combination with either azacitidine or decitabine. The primary endpoint was to analyze the prognostic value of MRD in OS and LFS.

 

Key learnings

Median OS (23.3 months vs 15.2 months; p = 0.006) and LFS (20.3 months vs 9.2 months; p = 0.01) were higher in the MRD-negative vs MRD-positive cohorts. 

MRD response assessed by RT-qPCR was associated with improved OS (p = 0.006) and LFS (p = 0.04). MRD response assessed by MFC also showed prognostic value for OS and LFS (both p < 0.001).

Multivariate analysis confirmed the prognostic value of MRD for OS (HR, 0.50; 95% CI, 0.23–0.82; p = 0.044) and LFS (HR, 0.44; 95% CI, 0.23–0.82; p = 0.010).

The findings highlight the importance of MRD monitoring in low-intensity AML therapy and identification of those at increased risk of relapse who may benefit from early therapeutic interventions. Further exploration of preemptive interventions based on MRD responses in a well-designed research framework is warranted. 

Abbreviations: AML, acute myeloid leukemia; CI, confidence interval; ELN, European LeukemiaNet; HMA, hypomethylating agent; HR, hazard ratio; ICC, International Consensus Classification of Myeloid Neoplasms; LFS, leukemia-free survival; MFC, multiparametric flow cytometry; MRD, measurable residual disease; mCR, morphologic complete response; OS, overall survival; RT-qPCR, real-time quantitative polymerase chain reaction; Ven, venetoclax; WHO, World Health Organization.

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