Azacytidine was approved based on a randomized, multicenter, double-blind, placebo-controlled study (AG120-C-009, NCT03173248) that included 146 patients with newly-diagnosed AML with an IDH1 mutation who met at least one of the following criteria: age 75 years or older, baseline Eastern Cooperative Oncology Group performance status of 2, severe cardiac or pulmonary disease, hepatic impairment with bilirubin >1.5 times the upper limit of normal, creatinine clearance <45 mL/min, or other comorbidity. Here, IDH1 is linked to acute myeloid leukemia.