Abstract |
CCAAT/enhancer-binding protein alpha (CEBPA) mutations are a favorable prognostic factor in adult acute myeloid leukemia (AML) patients; however, few studies have examined their significance in pediatric AML patients. Here we examined the CEBPA mutation status and clinical outcomes of pediatric AML patients treated in the AML-05 study. We found that 47 (14.9%) of the 315 evaluable patients harbored mutations in CEBPA; 26 cases (8.3%) harbored a single mutation (CEBPA-single) and 21 (6.7%) harbored double or triple mutations (CEBPA-double). After excluding core-binding factor-AML cases, patients harboring CEBPA mutations showed better overall survival (OS; P=0.048), but not event-free survival (EFS; P=0.051), than wild-type patients. Multivariate analysis identified CEBPA-single and CEBPA-double as independent favorable prognostic factors for EFS in the total cohort (hazard ratio (HR): 0.47 and 0.33; P=0.02 and 0.01, respectively). CEBPA-double was also an independent favorable prognostic factor for OS (HR: 0.30; P=0.04). CEBPA-double remained an independent favorable factor for EFS (HR: 0.28; P=0.04) in the normal karyotype cohort. These results suggest that CEBPA mutations, particularly CEBPA-double, are an independent favorable prognostic factor in pediatric AML patients, which will have important implications for risk-stratified therapy.
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Authors | H Matsuo, M Kajihara, D Tomizawa, T Watanabe, A M Saito, J Fujimoto, K Horibe, K Kodama, M Tokumasu, H Itoh, H Nakayama, A Kinoshita, T Taga, A Tawa, T Taki, S Tanaka, S Adachi |
Journal | Blood cancer journal
(Blood Cancer J)
Vol. 4
Pg. e226
(Jul 11 2014)
ISSN: 2044-5385 [Print] United States |
PMID | 25014773
(Publication Type: Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
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Chemical References |
- CCAAT-Enhancer-Binding Proteins
- CEBPA protein, human
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Topics |
- Adolescent
- CCAAT-Enhancer-Binding Proteins
(genetics)
- Child
- Child, Preschool
- Cohort Studies
- Disease-Free Survival
- Female
- Humans
- Infant
- Leukemia, Myeloid, Acute
(genetics)
- Male
- Mutation
- Polymorphism, Genetic
- Prognosis
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