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International Journal of Hematology

26-04-2024 | Acute Myeloid Leukemia | Progress in Hematology

CEBPA mutations in acute myeloid leukemia: implications in risk stratification and treatment

Authors: Feng-Ming Tien, Hsin-An Hou

Published in: International Journal of Hematology

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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 (CEBPAbZIP−inf) confer a survival benefit, and CEBPAbZIP−inf replaced CEBPA double mutations (CEBPAdm) 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 CEBPAbZIP−inf die of AML despite intensive treatment. This review aims to provide a comprehensive summary of the heterogeneities observed in AML with CEBPAdm and CEBPAbZIP−inf, 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.
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Metadata
Title
CEBPA mutations in acute myeloid leukemia: implications in risk stratification and treatment
Authors
Feng-Ming Tien
Hsin-An Hou
Publication date
26-04-2024
Publisher
Springer Nature Singapore
Published in
International Journal of Hematology
Print ISSN: 0925-5710
Electronic ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-024-03773-5
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