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Positive LSC status is associated with inferior survival outcomes in patients with acute myeloid leukemiaLeukemia Stem Cell Counts Help Doctors Predict Patient Survival Outcomes

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Key Takeaway
Note that positive LSC status is associated with significantly worse survival outcomes in acute myeloid leukemia.

This systematic review and reconstructed individual patient data meta-analysis evaluated the prognostic impact of LSC frequency in patients with acute myeloid leukemia. The study analyzed outcomes for patients at diagnosis (n=7781 for OS; n=5032 for EFS) and during remission (n=2006 for OS; n=1342 for EFS).

The meta-analysis found that positive LSC status at diagnosis was associated with inferior overall survival (HR 1.96; 95% CI, 1.85-2.08) and inferior event-free survival (HR 2.32; 95% CI, 2.15-2.49). Similarly, positive LSC status in remission was associated with significantly worse overall survival (HR 2.59; 95% CI, 2.23-3.03) and worse event-free survival (HR 2.53; 95% CI, 2.18-2.94).

The authors suggest that LSC frequency is a key prognostic factor that warrants implementation into clinical practice. However, the data indicates an association rather than a confirmed causal link. Clinical application should consider these findings as part of the broader risk stratification landscape for acute myeloid leukemia.

How this fits prior evidence

This meta-analysis addresses a gap in identifying specific prognostic biomarkers for acute myeloid leukemia. While previous evidence has identified risk factors such as MRG mutations in NPM1-mutated AML and the impact of KIT mutations on outcomes with low-dose chemotherapy, this study specifically identifies LSC frequency as a significant prognostic factor for both survival and event-free survival at diagnosis and in remission.

Doctors are looking for better ways to predict how patients with acute myeloid leukemia will respond to treatment. One way they do this is by checking for the presence of leukemia stem cells. These cells are the core cells that allow the cancer to grow and persist.

This large study looked at thousands of patients to see if these stem cells affected survival. The results showed that patients with a positive count of these stem cells had a much lower chance of survival. This was true whether the test was done at the start of treatment or after the patient was in remission.

Additionally, patients with these stem cells were more likely to experience complications or have their disease return. Because these results were so consistent, the study suggests that checking for these cells is a useful tool for doctors. It helps them understand the risks and plan better care for their patients.

What this means for you:
Patients with detectable leukemia stem cells have a much lower chance of survival and better outcomes.

Common questions

What is the link between LSC and leukemia survival?

The study found that a positive LSC status is associated with inferior overall survival and event-free survival. This link was observed in patients at the time of diagnosis and also for those in remission. The data suggests that LSC frequency is a significant factor in predicting how the disease progresses.

How many patients were included in this study?

The analysis included a large number of patients to reach its conclusions. Specifically, 7,781 patients were included for overall survival at diagnosis, and 5,032 were included for event-free survival at diagnosis. Smaller groups were also analyzed for patients in remission.

Does this mean LSC measurement replaces current treatments?

No, the study does not suggest that LSC measurement replaces current risk stratification methods. It identifies LSC frequency as a key prognostic factor. Patients should talk to their doctors about how these findings fit into their specific clinical care.

Study Details

Study typeMeta analysis
Sample sizen = 7,781
EvidenceLevel 1
PublishedOct 2026
View Original Abstract ↓
Leukemia stem cells (LSC) are thought to be responsible for relapse in patient with acute myeloid leukemia (AML). LSC can be quantified at diagnosis to improve risk stratification and during follow-up to monitor residual disease. To systematically evaluate the prognostic relevance of measuring LSC at diagnosis and in remission in patients with AML, we conducted a systematic and reconstructed individual patient data meta-analysis. We performed a comprehensive search for studies that reported overall survival (OS) and/or event-free survival (EFS) in relation to LSC measurements at diagnosis or in remission. We reconstructed individual patient data based on Kaplan-Meier curves. Fifty-six studies were included, including a total of 44 cohorts (n = 7781) for OS and 37 cohorts (n = 5032) for EFS at diagnosis; and 19 cohorts (n = 2006) for OS and 22 cohorts (n = 1342) for EFS in remission. Positive LSC status was significantly associated with inferior OS and EFS at diagnosis (OS: hazard ratio [HR], 1.96; 95% confidence interval [CI], 1.85-2.08; EFS: HR, 2.32; 95% CI, 2.15-2.49) and in remission (OS: HR, 2.59; 95% CI, 2.23-3.03; EFS: HR, 2.53; 95% CI, 2.18-2.94). To conclude, we found that measuring LSC has prognostic relevance both at diagnosis and in remission. This supports LSC frequency as a key prognostic factor that warrants implementation into clinical practice.
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