The combination of AZA and SL-401 (tagraxofusp), the anti-CD123-directed cytotoxin, which consists of recombinant interleukin-3 fused to a truncated diphtheria toxin, is currently being tested in an ongoing clinical trial (ClinicalTrials.gov Identifier: NCT03113643) for patients with MDS or AML. This evidence concerns the gene IL3RA and acute myeloid leukemia.