CEBPA mutations in acute myeloid leukemia: implications in risk stratification and treatment.
Journal
International journal of hematology
Journal Volume
120
Journal Issue
5
Pages
541 - 547
ISSN
1865-3774
Date Issued
2024-11
Author(s)
Abstract
Mutations in CCAAT enhancer binding protein α (CEBPA) occur in approximately 10% of patients with de novo acute myeloid leukemia (AML). Emerging evidence supports that in-frame mutations in the basic leucine zipper domain of CEBPA (CEBPA) confer a survival benefit, and CEBPA replaced CEBPA double mutations (CEBPA) as a unique entity in the 2022 World Health Organization (WHO-2022) classification and International Consensus Classification (ICC). However, challenges remain in daily clinical practice since more than 30% patients with CEBPA die of AML despite intensive treatment. This review aims to provide a comprehensive summary of the heterogeneities observed in AML with CEBPA and CEBPA, and will discuss the prognostic implications of concurrent mutations and novel mechanistic targets that may inform future drug development. The ultimate goal is to optimize clinical management and to provide precision medicine for this category of patients.
Subjects
Acute myeloid leukemia
CEBPA
Leukemogenesis
Prognosis
SDGs
Type
journal article