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Significance t(8;21) acute myelogenous leukemia (AML) often presents different degrees of aberrant myeloid differentiation. Although most patients respond to current chemotherapy regimens, a sizeable number of patients are prone to relapse. Thus, the cellular heterogeneity and disease outcome may need exploration. Here, by integrating high-throughput DNA/RNA sequencing with phenotypic analysis, heterogeneous leukemic cell populations were identified. CD34+ CD117dim myeloblasts had the characteristics of granulocyte-monocyte progenitors and might be responsible for drug-resistance. CD34+ CD117bri and AM cells were blocked later than CD34+ CD117dim cells, and AM cells were sensitive to chemotherapy. t(8;21) AML patients with a high proportion of CD34+ CD117dim cells had inferior outcomes. The identification of the CD34+ CD117dim proportion as a potential prognostic factor may lead to new tools for future tailored therapeutic strategies.

Multidimensional study of the heterogeneity of leukemia cells in t(8;21) acute myelogenous leukemia identifies the subtype with poor outcome