Huo Wenxuan, Shen Yifan, Huang Jiayu, Yang Yang, Fan Shuang, Zhao Xiaosu, Wen Qi, Wang Luxiang, Jiang Chuanhe, Cao Yang, Mo Xiaodong, Xu Yang, Hu Xiaoxia
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.
National Clinical Research Center for Hematologic Diseases, Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University, Institute of Blood and Marrow Transplantation, Collaborative Innovation Center of Hematology, Soochow University, Suzhou, 215006, China.
Front Med. 2025 Feb;19(1):90-100. doi: 10.1007/s11684-024-1091-5. Epub 2024 Dec 7.
The cooccurrence of NPM1, FLT3-ITD, and DNMT3A mutations (i.e., triple mutation) is related to dismal prognosis in patients with acute myeloid leukemia (AML) receiving chemotherapy alone. In this multicenter retrospective cohort study, we aimed to identify whether allogeneic hematopoietic stem cell transplantation (allo-HSCT) could overcome the poor prognosis of DNMT3ANPM1FLT3-ITD AML across four transplant centers in China. Fifty-three patients with triple-mutated AML receiving allo-HSCT in complete remission were enrolled. The 1.5-year probabilities of relapse, leukemia-free survival, and overall survival after allo-HSCT were 11.9%, 80.3%, and 81.8%, respectively. Multivariate analysis revealed that more than one course of induction chemotherapy and allo-HSCT beyond CR1 were associated with poor survival. To our knowledge, this work is the largest study to explore the up-to-date undefined role of allo-HSCT in patients with triple-mutated AML. Our real-world data suggest that allo-HSCT could overcome the poor prognosis of DNMT3ANPM1FLT3-ITD in AML.
NPM1、FLT3-ITD和DNMT3A突变同时出现（即三重突变）与单纯接受化疗的急性髓系白血病（AML）患者的不良预后相关。在这项多中心回顾性队列研究中，我们旨在确定在中国的四个移植中心，异基因造血干细胞移植（allo-HSCT）是否能够克服DNMT3A NPM1 FLT3-ITD AML的不良预后。纳入了53例完全缓解后接受allo-HSCT的三重突变AML患者。allo-HSCT后1.5年的复发概率、无白血病生存率和总生存率分别为11.9%、80.3%和81.8%。多变量分析显示，超过一个疗程的诱导化疗以及CR1之后的allo-HSCT与生存不良相关。据我们所知，这项研究是探索allo-HSCT在三重突变AML患者中尚未明确作用的最大规模研究。我们的真实世界数据表明，allo-HSCT可以克服AML中DNMT3A NPM1 FLT3-ITD的不良预后。
