Prognosis and monitoring of core-binding factor acute myeloid leukemia: current and emerging factors

Abstract

Core-binding factor acute myeloid leukemia (CBF-AML) – including AML with t(8;21) and AML with inv(16) – accounts for about 15% of adult AML and is associated with a relatively favorable prognosis. Nonetheless, relapse incidence may reach 40% in these patients. In this context, identification of prognostic markers is considered of great interest. Due to similarities between their molecular and prognostic features, t(8;21) and inv(16)-AML are usually grouped and reported together in clinical studies. However, considerable experimental evidences have highlighted that they represent two distinct entities and should be considered separately for further studies. This review summarizes recent laboratory and clinical findings in this particular subset of AML and how they could be used to improve management of patients in routine practice.

Keywords:

• acute myeloid leukemia
• CBFB-MYH11
• core binding factor
• minimal residual disease
• mutational analysis
• prognostic markers.
• RUNX1-RUNX1T1

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

Key issues

• Patients with core-binding factor acute myeloid leukemia are considered to have a favorable prognosis but constitute in fact a heterogeneous population: relapse incidence may reach 40% and not all may be cured.

• Recurrent molecular abnormalities, especially KIT mutations, could improve risk stratification and define therapeutic targets.

• Minimal residual disease monitoring of therapy has been shown to be a significant prognostic factor and could be used for treatment stratification.

• AML with t(8;21) and those with inv(16) should be reported separately in future studies.

• Participation in clinical trials and the collection of samples for correlative science analysis is recommended for all patients with newly diagnosed core-binding factor acute myeloid leukemia.