Acute Myeloid Leukemia Patients with High-Risk Karyotypes Benefit from Decitabine in Combination with Modified CAG.
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This study aims to investigate the genetic characteristics of Acute Myeloid Leukemia (AML) patients and identify which patients derive the greatest benefit from a low-intensity regimen of decitabine combined with modified Cytarabine + Aclarubicin + Granulocyte Colony-Stimulating Factor (D-CAG) or intensive chemotherapy (IA regimen). We retrospectively analyzed cytogenetic and molecular data of 331 newly diagnosed AML patients and examined the associations between genetic characteristics, risk status, treatment approaches, and clinical outcomes. The median follow-up duration was 45 months (range: 2-120 months). Patients receiving IA therapy achieved higher complete remission (CR) rates (79.3% vs. 66.4%, P < 0.05), while objective response rates (ORR) remained comparable between the two treatment arms. Both in the total cohort and among favorable-risk patients, the median overall survival (OS) was significantly reduced in the D-CAG group compared to the IA group (P < 0.05). For intermediate- and high-risk patients who had not undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT), median OS outcomes were comparable between the two treatment regimens. Patients harboring TET2 (Tet methylcytosine dioxygenase 2), NRAS (Neuroblastoma RAS viral oncogene homolog), or biallelic CEBPA mutations demonstrated superior OS in the IA group compared to the D-CAG group (P < 0.05). Notably, older patients with complex or monosomal karyotypes exhibited longer median OS than their younger counterparts (P < 0.05). In conclusion, D-CAG may represent a more suitable therapeutic option for AML patients with high-risk karyotypic profiles.