Purpose of review
In recent years, new molecular markers have emerged as significant prognostic parameters and as potential targets for molecularly targeted therapy in acute myeloid leukemia (AML). However, prognostic markers cannot guide the decision for a specific treatment, as they are associated with a differential outcome regardless of the given treatment. In contrast, predictive markers indicate a treatment benefit in patients that are characterized through these markers. Thus, predictive markers can guide clinical decision-making.
In young adults, mutations of the nucleophosmin (gene 9NPM1mut) in the absence of concurrent FLT3-internal tandem duplication (ITD) (FLT3-ITDneg) have impressive prognostic and, beyond prognostication, predictive properties. This NPM1mut/FLT3-ITDneg genotype predicts equivalent favorable outcome after intensive chemotherapy and allogeneic stem cell transplantation, whereas in the absence of this marker clinical outcome was significantly improved after an allogeneic transplantation. In addition, within a retrospective study performed on older adults, the same genotype predicted a significantly improved outcome if all-trans retinoic acid was added to intensive chemotherapy.
The discovery of new prognostic and predictive markers has increased our understanding of leukemogenesis and this may lead to improved prognostication and, more important, to novel genotype-specific treatment strategies.