What type of study is this?

This is a Cohort Study study (also classified as: Human Clinical Trial).

December 5, 2025PeerJ1 citationsOpen Access

Retrospective study on the clinical outcomes and characteristics of acute myeloid leukemia: different outcomes in the same risk group

FMFanqiao MengArmy Medical University HXHuan XieArmy Medical University YQYan QiTongji University

Key Points

Median overall survival was 22.9 months with significant variability observed based on molecular factors.
The most common mutations included TP53 and FLT3, impacting prognosis and treatment outcomes.
Retrospective analysis integrated next-generation sequencing and clinical outcomes in 154 acute myeloid leukemia patients.
Hematopoietic stem cell transplantation's effectiveness varies, emphasizing the need for refined risk classifications.

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).

Ask AI

Helpful

Bookmark

View Full Paper