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A clonal hematopoietic disease, acute myeloid leukemia (AML) is characterized by abnormal proliferation of early precursors of myeloid cells and blasts. It represents a heterogeneous disease group with a considerably complex biology and pathophysiology. Various translocations, chromosome copy number changes, and mutations have been described in AML, and a few of them help define the diagnosis, prognosis, and management. Cytarabine and anthracycline-based combination regimens followed by the allogeneic stem cell transplant remain the optimal treatment in most cases. However, older age and decreased tolerance to conventional therapies pose a major challenge for the conventional therapies, leading to the development of effective and less toxic therapy modalities as reviewed in this article.

Navigating through Mutations in Acute Myeloid Leukemia. What Do We Know and What Do We Do with It?