Chidamide in HR-AML

Acute myeloid leukemia (AML) patients with fusion gene-positive including core binding factor (CBF), mixed-lineage leukemia (MLL) gene rearrangement have a high incidence of relapse if they have continuously positive measurable residual disease (MRD) or ELN 2022-high risk chromosome abnormality and could not be bridged to allogenetic heamatopoitic stem cell transplantation (allo-HSCT). Histone deacetylase (HDAC) is known to abnormally recruit in these fusion gene-positive AML, and has been proven to be a promising therapy target. Whether HDAC inhibitor chidamide could be used as a maintenance therapy in these AML patients remains unknown. This study aims to evaluate the efficacy and safety of chidamide in the maintenance therapy of high-risk acute myeloid leukemia (AML) patients with core binding factor (CBF) and measurable residual disease (MRD) positivity, or ELN 2022-high risk fusion gene positive.

Inclusion Criteria: 1. Age range ≥18 years, both male and female were eligible. 2. Patients with CFB-AML and MRD positive, or patients with ELN 2022-high risk fusion genes, such as MLL rearrangement, or NUP98 rearrangement, ect. 3. Use of chidamide-based regimens as maintenance therapy for at least 3 months, without undergoing or not planning to undergo allo-HSCT. 4. ECOG ≤4; 5. At screening, laboratory tests meet the following criteria: (1) Complete blood count: hemoglobin (Hb) ≥90 g/L, absolute neutrophil count (ANC) ≥1.5×10⁹/L, platelet count (PLT) ≥90×10⁹/L; (2) Biochemical tests: serum creatinine (Cr) ≤1.5× upper limit of normal (ULN); total bilirubin (TBIL) ≤1.5×ULN; alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤2.5×ULN (for cases with liver metastasis: ≤5×ULN). Exclusion Criteria: 1. Known history of allergy to the study drug. 2. Resistant to chidamide. 3. Unable to take oral medications. 4. Concurrent uncontrolled active infection (including bacterial, fungal, or viral infections). 5. Concurrent uncontrolled major organ failure. 6. Currently participating in other clinical studies that affected the primary objectives of this study. 7. Patients deemed by the investigators to be unsuitable for participation in this study.