How I Treat TP53-Mutated Acute Myeloid Leukemia and Myelodysplastic Syndromes

Simple Summary TP53 mutations are seen in 5–10% of de novo MDS and AML, but 25–40% of therapy-related MDS and AML. Despite the addition of recent drugs to the common regimen and improvement of post transplantation survival, these particular myeloid malignancy subtypes remain a challenge for hematologists around the globe. In this article, we aim at reviewing the biology and most recent advances in the treatment of TP53 MDS and AML. Abstract TP53-mutated acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are among the myeloid malignancies with the poorest prognosis. In this review, we analyze the prognosis of these two diseases, focussing particularly on the extent of the mono or biallelic mutation status of TP53 mutation, which is largely correlated with cytogenetic complexity. We discuss the possible/potential improvement in outcome based on recent results obtained with new drugs (especially eprenetapopt and magrolimab). We also focus on the impact of allogeneic hematopoietic stem cell transplantation (aHSCT) including post aHSCT treatment.