Decitabine combined with reduced-intensity conditioning for older patients with acute myeloid leukemia in composite complete remission undergoing allogeneic hematopoietic stem cell transplantation: a multicenter, single-arm, phase 2 trial

作者：Qifa Liu, Zhongli Hu, Na Xu, Yirong Jiang, Zhiping Fan, Fen Huang, Ren Lin, Hua Jin, Yunxin Zeng, He Hai, Ping Zhu, Guopan Yu, Pengcheng Shi, Ruijuan Sun, Xiaojun Xu, Zhangkun Li, Yu Zhang, Jing Sun, Yu Wang, Li Xuan · 发表于：The Lancet Regional Health - Western Pacific · 年份：2025 · DOI：10.1016/j.lanwpc.2025.101664 · 被引用次数：3 · 研究领域：Acute Myeloid Leukemia Research、Hematopoietic Stem Cell Transplantation、Neutropenia and Cancer Infections

Background: Outcomes of older patients with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem-cell transplantation (allo-HSCT) remain unsatisfactory. The primary objective of this trial was to establish whether decitabine combined with reduced-intensity conditioning (RIC) regimen could improve overall survival (OS) for this population in composite complete remission (CRc). Methods: daily intravenously (days -6 to -3). The primary endpoint was 2-year OS rate. All efficacy and safety endpoints were assessed in the intention-to-treat population. This trial is registered with ClinicalTrials.gov (NCT03530085) and is complete. Findings: Between Jan 1, 2021 and Nov 30, 2022, 60 patients were enrolled. With a median follow-up of 35.5 months (IQR 32.5-39.2), 39 patients survived and 21 died. The 2-year OS rate was 67% (95% CI 56-80), which met the primary objective. Within 100 days post-transplantation, the most common grade 3-4 non-hematological treatment-emergent adverse events (TEAEs) were infections (22 [37%]), acute graft-versus-host disease (21 [35%]), and gastrointestinal disorders (16 [27%]). Five (8%) patients died of TEAEs, with one death treatment-related. Interpretation: Decitabine combined with RIC regimen exhibits encouraging OS and acceptable toxicity profile, which might be a suitable therapeutic option for older patients with AML. Funding: National Natural Science Foundation of China; Science and Technology Program of Guangdong Province; National K...