A large new study has confirmed the clinical relevance of a pharmacogenomic score that could help tailor leukaemia treatment and address persistent racial disparities in outcomes.
Researchers have validated the ara-C pharmacogenomics score (ACS10), a 10-variant genetic marker originally developed for children, as a predictor of treatment response in both pediatric and young adult patients with acute myeloid leukaemia (AML). The study, which analysed data from 1,086 patients across multiple clinical trials, found that a low ACS10 score was linked to significantly worse event-free survival (EFS) following standard induction chemotherapy.
Crucially, the findings also shed light on racial disparities in AML outcomes. Black patients were far more likely to have low ACS10 scores than White patients, 84% vs. 22% in one cohort, which correlated with poorer survival rates. However, when standard chemotherapy was augmented with the drug bortezomib, survival outcomes between Black and White patients were no longer significantly different, suggesting that therapy adjustments could help mitigate these disparities. The ACS10 score remained a strong predictor of outcomes even after adjusting for factors like age, race, and disease risk.
The study supports integrating ACS10 into future clinical protocols, especially to guide induction therapy strategies in populations historically at greater risk of poor outcomes. Personalised treatment, the authors note, may be key to improving survival and closing the racial gap in AML care.
Reference
Marrero RJ et al. Cytarabine pharmacogenomics and outcomes among children and young adults with acute myeloid leukemia. JAMA Netw Open. 2025;8;(6):e2516296.
