Molecular monitoring in AML: Not just prognostic, but predictive?
June 23, 2025
New Horizons in Leukemia Research: Focus on Selected Papers
A recently published study in the journal Lancet Haematology presented the results of two randomized, controlled, phase 3 trials: "UK NCRI AML17" and "AML19." Both studies examined how molecular monitoring affects the survival of adults with acute myeloid leukemia (AML). The studies aimed to determine if adjusting therapy based on measurable residual disease (MRD) results could improve patient survival.
According to Potter et al., this is the first randomized study to compare molecular minimal residual disease (MRD) monitoring and MRD-guided treatment with standard clinical treatment without monitoring. Both studies examined patients aged 16 to 60 years with newly diagnosed acute myeloid leukemia (AML) for molecular markers suitable for disease monitoring, including NPM1 mutations and fusion genes. A total of 637 patients were subsequently randomized:
- Group 1: Sequential molecular MRD monitoring during treatment and for three years afterward
- Group 2: Standard clinical care without molecular monitoring
Study results in detail
The results showed that MRD monitoring did not improve the overall survival rate of the study population. At a median follow-up of 4.9 years, the 3-year overall survival rate was 70% (95% confidence interval [CI]: 66-75) in the monitoring group and 73% (95% CI: 68-80) in the non-monitoring group (Figure 1).
However, a survival
advantage was observed for patients with NPM1 and FLT3-ITD
mutations who were assigned to MRD monitoring (Figure 2). The 3-year survival
rate was 69% (95% CI: 60-79) in the MRD-monitored group, compared to 58% (95%
CI: 45-74) in the non-monitored group (HR: 0.53; 95% CI: 0.31-0.91; p=0.021).
For patients with NPM1 mutations without FLT3-ITD or with fusion
gene transcripts, MRD monitoring did not show any survival benefit.
MRD is already well-established as a prognostic
biomarker in AML. Data from this study indicate that MRD can act as both a
prognostic and a predictive biomarker. For certain molecular subgroups,
therapeutic measures based on MRD results can improve overall patient survival.
This could lead to a more targeted, personalized therapeutic approach,
particularly for patients with specific genetic markers.
For further information and access to the full
publication, please visit the website of the Lancet Haematology.
Source
Potter N et al Molecular monitoring versus standard clinical care in younger adults with acute myeloid leukaemia: results from the UK NCRI AML17 and AML19 randomised, controlled, phase 3 trials. Lancet Haematol. 2025;12(5):e346-e356. https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(25)00037-7/fulltext
The author
»If you have any further questions about this study, please contact us!«
Dr. rer. nat. Katharina Hörst