Purpose of review: Core binding factor acute myeloid leukemia (CBF-AML) corresponds to two distinct subtypes of AML characterized by recurrent favorable chromosome translocations, namely t(8;21) and inv(16)/t(16;16). Given the relatively good outcome of patients with CBF-AML, when treated with intensive chemotherapy including high-dose cytarabine, they are generally not considered as candidates for intensification with allogeneic stem cell transplantation in the first complete remission. The optimal treatment strategy (place of stem cell transplantation, best postremission chemotherapy, role of targeted agents) remains, however, to be defined in these patients.
Recent findings: The biological and prognostic heterogeneity of both CBF-AML subtypes, including gene mutation and gene expression profiles as well as molecular response to therapy, has been recently described.
Summary: These new insights in the heterogeneity of CBF-AML suggest that a tailored approach might be preferred to a unique predefined strategy to treat these patients.