维奈托克与阿扎胞苷对比强化化疗用于首次完全缓解后接受异基因造血干细胞移植的高危急性髓系白血病患者：TROPHY组的多中心研究
Venetoclax and azacitidine compared with intensive chemotherapy for adverse-risk acute myeloid leukemia patients receiving allogeneic hematopoietic stem cell transplantation in first complete remission: A multicenter study of TROPHY group.
作者信息
Wen Qi, Jiang Chuanhe, Liu Xiaodan, Xia Yi, Ma Yilei, Yang Yang, Wang Yu, Chang Yingjun, Wang Luxiang, Zhang Zilu, Huang Xiaojun, Cao Yang, Zhao Yanmin, Hu Xiaoxia, Mo Xiaodong
机构信息
Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing 100044, China.
State Key Laboratory of Medical Genomics, Shanghai Institute of Hematology, National Research Center for Translational Medicine, Shanghai Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China.
出版信息
Chin J Cancer Res. 2025 Jun 30;37(3):417-431. doi: 10.21147/j.issn.1000-9604.2025.03.10.
OBJECTIVE
Adverse-risk acute myeloid leukemia (AML) patients should receive allogeneic hematopoietic stem cell transplantation (allo-HSCT) at first complete remission (CR1). However, the influence of prior therapies [i.e., venetoclax plus azacitidine (VEN-AZA) or intensive chemotherapy (IC)] on post-transplant outcomes remains inconclusive. This multicenter, retrospective study compared the post-transplant outcomes between patients receiving VEN-AZA and those receiving IC before allo-HSCT.
METHODS
This study was based on the transplant database of TROPHY group. Consecutive adverse-risk AML patients receiving allo-HSCT from January 2021 to June 2023 were screened in five Chinese transplant centers. Patients were categorized into VEN-AZA group if they received venetoclax combined with azacitidine as first-line therapy followed by allo-HSCT. Patients who received first-line therapy consisting of a mainstay treatment of cytarabine and anthracycline followed by allo-HSCT were categorized into IC group.
RESULTS
In the total cohort, the 3-year probabilities of overall survival, leukemia-free survival, and event-free survival were better in the IC group than VEN-AZA group, particularly for patients with mutations or mutations. However, the survival of the VEN-AZA group was not superior to that of IC group in patients aged ≥55 years or those with the hematopoietic cell transplantation-comorbidity index scores ≥1 before allo-HSCT. After propensity score matching (median age: VEN-AZA group: 57 years; IC group: 55 years), only the probability of overall survival for the IC group was better than that of VEN-AZA group (93.6% 78.0%, P=0.034) at the 1-year follow-up; however, all of the other clinical outcomes were comparable between the VEN-AZA and IC groups. The mutation was independently associated with post-transplant relapse and survival.
CONCLUSIONS
Our results suggest that IC remains the cornerstone of therapy, whereas VEN-AZA may also be used in younger patients and medically fit patients with adverse-risk AML who are receiving allo-HSCT in CR1.
目的
高危急性髓系白血病（AML）患者应在首次完全缓解（CR1）时接受异基因造血干细胞移植（allo-HSCT）。然而，先前治疗[即维奈克拉联合阿扎胞苷（VEN-AZA）或强化化疗（IC）]对移植后结局的影响仍无定论。这项多中心回顾性研究比较了接受VEN-AZA治疗的患者与在allo-HSCT前接受IC治疗的患者的移植后结局。
方法
本研究基于TROPHY组的移植数据库。在五个中国移植中心筛选了2021年1月至2023年6月期间接受allo-HSCT的连续高危AML患者。如果患者接受维奈克拉联合阿扎胞苷作为一线治疗，随后进行allo-HSCT，则将其分类为VEN-AZA组。接受以阿糖胞苷和蒽环类药物为主的一线治疗，随后进行allo-HSCT的患者被分类为IC组。
结果
在整个队列中，IC组的3年总生存、无白血病生存和无事件生存概率均优于VEN-AZA组，尤其是对于有 突变或 突变的患者。然而，在年龄≥55岁或allo-HSCT前造血细胞移植合并症指数评分≥1的患者中，VEN-AZA组的生存率并不优于IC组。在倾向评分匹配后（中位年龄：VEN-AZA组：57岁；IC组：55岁），在1年随访时，仅IC组的总生存概率优于VEN-AZA组（93.6% 对78.0%，P = 0.034）；然而，VEN-AZA组和IC组的所有其他临床结局相当。 突变与移植后复发和生存独立相关。
结论
我们的结果表明，IC仍然是治疗的基石，而VEN-AZA也可用于年龄较轻且身体状况适合的高危AML患者，这些患者在CR1时接受allo-HSCT。
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