Rational biomarker development for the early and minimally invasive monitoring of AML

Recurrent disease remains the principal cause for treatment failure in acute myeloid leukemia (AML) across age groups. Reliable biomarkers of AML relapse risk and disease burden have been problematic, as symptoms appear late and current monitoring relies on invasive and cost-ineffective serial bone marrow (BM) surveillance. In this report, we discover a set of unique microRNA (miRNA) that circulates in AML-derived vesicles in the peripheral blood ahead of the general dissemination of leukemic blasts and symptomatic BM failure. Next-generation sequencing of extracellular vesicle-contained small RNA in 12 AML patients and 12 controls allowed us to identify a panel of differentially incorporated miRNA. Proof-of-concept studies using a murine model and patient-derived xenografts demonstrate the feasibility of developing miR-1246, as a potential minimally invasive AML biomarker.

Automated summary

Recurrent disease in acute myeloid leukemia (AML) is a major cause of treatment failure across age groups. Current monitoring of relapse risk and disease burden in AML patients is challenging due to the late appearance of symptoms. This study identified a set of unique circulating miRNA in AML-derived vesicles in peripheral blood, showing promise as a potential minimally invasive biomarker for AML.