State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China.
Hematol Oncol. 2024 Jan;42(1):e3232. doi: 10.1002/hon.3232. Epub 2023 Oct 4.
Recurrence following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the major cause of treatment failure in patients with myeloid malignancy. Azacytidine (AZA) maintenance is a promising therapy to prevent relapse and improve survival. We conducted a prospective, one-arm study involving 78 patients with myeloid malignancy at a high risk of recurrence who were enrolled between September 2019 and April 2022. Furthermore, 102 matched historical controls were selected using propensity score matching. With a median follow-up time of 19.6 (3.5-91.7) months, AZA maintenance therapy significantly improved relapse-free survival (RFS; log-rank test, p = 0.01). The AZA and control groups had a 1-year RFS of 87.7% (95% confidence interval [CI], 0.80-0.96) and 72.2% (95% CI, 0.64-0.82), respectively, with a hazard ratio (HR) of 0.21 (95% CI, 0.09-0. 47; p < 0.01). There were no grade 4 adverse effects or deaths related to AZA. Refractory patients with favorable/intermediate-risk acute myeloid leukemia (AML) benefited more from AZA maintenance therapy than those with adverse-risk AML according to the European Leukemia Net guidelines (RFS in favorable/intermediate-risk AML, HR = 0.29, 95% CI, 0.11-0.79; RFS in adverse-risk AML, HR = 0.57, 95% CI, 0.21-1.6; p for interaction = 0.03). Our findings suggest that AZA maintenance therapy following allo-HSCT was safe and could reduce the incidence of relapse, particularly for refractory patients with favorable/intermediate-risk AML.
异基因造血干细胞移植（allo-HSCT）后复发是髓系恶性肿瘤患者治疗失败的主要原因。阿扎胞苷（AZA）维持治疗是一种有前途的预防复发和提高生存率的方法。我们进行了一项前瞻性、单臂研究，纳入了 78 例髓系恶性肿瘤高复发风险患者，这些患者于 2019 年 9 月至 2022 年 4 月入组。此外，使用倾向评分匹配选择了 102 例匹配的历史对照。中位随访时间为 19.6（3.5-91.7）个月，AZA 维持治疗显著改善了无复发生存（RFS；对数秩检验，p=0.01）。AZA 组和对照组 1 年 RFS 分别为 87.7%（95%CI，0.80-0.96）和 72.2%（95%CI，0.64-0.82），风险比（HR）为 0.21（95%CI，0.09-0.47；p<0.01）。没有与 AZA 相关的 4 级不良事件或死亡。根据欧洲白血病网指南，与不良风险 AML 相比，有利/中危急性髓系白血病（AML）的难治性患者从 AZA 维持治疗中获益更多（有利/中危 AML 的 RFS，HR=0.29，95%CI，0.11-0.79；不良风险 AML 的 RFS，HR=0.57，95%CI，0.21-1.6；p 交互=0.03）。我们的研究结果表明，allo-HSCT 后 AZA 维持治疗是安全的，可以降低复发率，特别是对有利/中危 AML 的难治性患者。
