Genetic mutations appear to play a central role in explaining why Black patients with acute myeloid leukemia experience worse survival outcomes than White patients, according to analysis of more than 30 years of clinical trial data. The study examined 10 phase 2 and phase 3 trials involving newly diagnosed adults with AML, where participants received identical, highly controlled medical care.
The controlled nature of clinical trials proves crucial for understanding the disparity. Lead researcher Shella Saint Fleur-Lominy explained that because trial participants receive close medical oversight with regular monitoring, observed differences in outcomes become “less likely related to social factors” and point instead toward untested genetic mutations as the explanation. She told Medscape Medical News “there could be other mutations that we don’t know about.”
The NPM1 mutation exemplifies how genetic factors create racial disparities in leukemia survival. This specific mutation produces significantly worse outcomes in Black patients compared to White patients with the same mutation. Andrew Stiff and colleagues documented “evidence of underlying biological differences in patients with NPM1 mutations with respect to genetic ancestry.” Wang and colleagues found that “growing evidence suggests that NPM1 mutations do not seem to confer as favorable a prognostic impact on Black patients as on White patients treated with intensive chemotherapy.”
Venetoclax, approved by the FDA in late 2018, demonstrates how targeting genetic mutations can eliminate disparities. Before this drug became available, non-Hispanic Black patients had a 22 percent higher hazard of death than non-Hispanic Whites. After venetoclax approval, Wang reported “worse [overall survival] was not observed for [non-Hispanic Blacks],” effectively erasing the survival gap through mutation-specific treatment
See: “Genetic mutations and racial disparities in leukemia survival” (January 30, 2026)Â
