Phase II study of dual epigenetic targeting with chidamide and azacitidine in patients with high-risk acute myeloid leukemia after allo-HSCT

作者：Yaxue Wu, Chao‐Ling Wan, Kai-Wen Tan, Yang Zhang, Yanming Zhang, Hao Zhang, Depei Wu, Suning Chen, Ying Wang, Xiao Ma, Haiping Dai, Chongsheng Qian, Shanshan Jiang, Haiyan Bao, Xiaohui Hu, Zheng Li, Shengli Xue · 发表于：Clinical Epigenetics · 年份：2025 · DOI：10.1186/s13148-025-01987-w · 被引用次数：2 · 研究领域：Acute Myeloid Leukemia Research、Hematopoietic Stem Cell Transplantation、Histone Deacetylase Inhibitors Research

BACKGROUND: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is potentially the only curative option for high-risk acute myeloid leukemia (AML) patients. However, disease relapse remains the principal cause of treatment failure of these patients, and outcomes of salvage treatments are poor. This research seeks to evaluate the efficacy and safety of a dual epigenetic targeting maintenance therapy with chidamide and azacitidine (AZA) in patients with high-risk AML post-allo-HSCT. METHODS: ) subcutaneously daily for 5 days, respectively. Treatment started as early as 3 months after transplantation. All patients were in complete remission before each maintenance cycle. A total of 6 cycles was recommended. RESULTS: The 2-year cumulative incidence of relapse (CIR) was 8.4% (95% CI 4.4-12.4%), 2-year relapse-free survival (RFS) was 91.6% (95% CI 87.6-95.6%), and overall survival (OS) was 97.9% (95% CI 95.8-100%). At the end of the follow-up period, 4 patients relapsed, of which 1 patient died of leukemia recurrence; the other three patients underwent second allo-HSCT and are still alive in remission. The most common adverse events (AEs) were hematological toxicity. No grade > 3 AEs were noted. Graft-versus-host disease (GVHD) occurred in 29.2% (14/48) of patients. Six cases (6/48, 12.5%) were complicated with infection. No treatment-related mortality occurred. CONCLUSIONS: Dual epigenetic targeting maintenance treatment with CHI-AZA has an acceptable toxicity profile a...