In the ALFA-0701 clinical trial, the subset analyses pointed out the benefit of the addition of GO on 2-year event-free survival (EFS), RFS and OS in NPM1-mutated AML patients, mainly in the subgroup presenting with activating signaling mutations [83], while the AMLSG 09-09 German phase III study showed an improved RFS and a reduced cumulative incidence of relapse in NPM1-mutated AML, due to deeper reduction of NPM1-mutated transcript levels across all treatment cycles [84,85]. This evidence concerns the gene NPM1 and acute myeloid leukemia.