Retrospective study on the clinical outcomes and characteristics of acute myeloid leukemia: different outcomes in the same risk group
Year: 2025
Type: article
Study design: Observational Study
Abstract: Background Acute myeloid leukemia (AML) remains a prognostically heterogeneous malignancy despite advances in molecular risk stratification. While the 2022 European Leukemia Network (ELN) guidelines refine risk classification, their accuracy in predicting survival outcomes across genetic requires validation. Methods We conducted a retrospective analysis of 154 newly diagnosed AML patients at Daping Hospital, integrating next-generation sequencing (NGS)-based genetic profiling, 2022 ELN risk classification, and clinical outcomes. Results The most frequent mutations were FLT3 (26.6%), DNMT3A (21.4%), NPM1 (18.2%), CEBPA (17.5%), and TET2 (15.6%). Median overall survival (OS) and progression-free survival (PFS) were 22.9 months and 14.1 months, respectively. Hematopoietic stem cell transplantation (HSCT), female sex, age < 60 years, and normal karyotype emerged as favorable prognostic factors. No significant differences were observed between allogeneic (allo-HSCT) and autologous HSCT (ASCT). Idarubicin, cytarabine, etoposide (IA ± E) chemotherapy yielded superior survival, while azacitidine+venetoclax (AZA+VEN) regimens underperformed. Conversely, TP53 and KIT mutations correlated with inferior survival, while NPM1, CEBPA mutations predicted longer survival. Notably, significant survival heterogeneity existed within 2022 ELN risk groups, particularly among patients with FLT3 , CEBPA , or TET2 mutations. Conclusions The ELN risk classification demonstrate limitations in prognostication, particularly for patients with FLT3 , CEBPA , or TET2 mutations. Our findings highlight the necessity for refined risk models incorporating additional molecular markers ( KIT ) and mutation interactions to enhance personalized prognostication. Gene coexistence is also a factor that needs to be considered when determining patient prognosis. Trial registration The study was registered on the Chinese clinical trial registry (ChiCTR) platform (No. ChiCTR2500096484).
Source: PeerJ
DOI: 10.7717/peerj.20436
Authors: Fanqiao Meng, Maoyuan Xiang, Huan Xie, Yu Liu, Yan Qi, Dongfeng Zeng
Institutions: Army Medical University, Daping Hospital
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $1,395
Award: G8943444576
OpenAlex ID: W4417028909
