RUNX1 and bone marrow failure syndrome: The discovery of RUNX1 mutations as the cause of familial platelet disorder (FPD) was pivotal to understanding the implications of this gene in hematological malignancies. FPD is an inherited bone marrow failure syndrome (IBMFS) with quantitative and qualitative platelet abnormalities and a high predisposition to acute myeloid leukemia (AML) [1,2]. IBMFS are genetic disorders characterized by cytopenia and hypoproliferation of one or more cell lineages in the bone marrow [1].