In a small, multicenter, phase Ib/II, dose-escalation trial of APR-246 and AZA in adult patients with TP53 mutant MDS or oligoblastic AML (≤30% blasts), ORR by International Working Group (IWG) 2006 criteria was 100%, with nine patients achieving CR and two achieving CR based on bone marrow (NCT03072043). This evidence concerns the gene TP53 and acute myeloid leukemia.