Abstract
To understand how myelodysplastic syndromes (MDS) transform to AML and to describe how transformation can be predicted and prevented. Recent genomic analyses have shown that MDS progression to AML is associated with clonal expansion and clonal evolution. Mutation profiles of MDS change during progression and new mutations in signaling genes and transcription factors emerge. AML transformation can be predicted by several parameters including International Prognostic Scoring System IPSS risk category and transfusion requirements. The prognostic relevance of the acquisition of some gene mutations (i.e., IDH1 and 2, CBL, FT3, RAS, NPM1, TP53, and ASXL1) has to be prospectively validated. The most effective preventive therapy for AML transformation is allogeneic stem cell transplantation. Hypomethylating agents have been associated with prolonged time to AML transformation even in patients who did not achieve an objective response. The recent progress in the understanding of the molecular events leading to transformation and the event of new effective therapies open new avenues for a better prediction and prevention of AML transformation in patients with MDS.
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