Introduction: Aberrant expression of miRNAs has been linked with the initiation and progression of several cancers. However, their role as prognostic biomarkers in pediatric core binding factor AML (CBF-AML) is still unclear. The objective of the present study was to identify differentially expressed miRNAs and their prognostic significance in pediatric CBF-AML patients. Methods: Bone marrow samples from 28 pediatric CBF-AML patients and 27 pediatric controls were obtained after ethical approval. Based on our previous findings, expression levels of 11 miRNAs and their selected potential targets were analysed using TaqMan advanced miRNA assay and SYBR Green-based qRT-PCR, respectively. Differential miRNA expression was correlated with clinicopathological parameters and survival outcomes. Results: Upregulation of miR-100-5p, miR-4446-3p, and miR-335-3p and downregulation of miR-409-3p, miR-151a-3p, miR-196b, miR-4772-5p, and miR-758-3p was observed in pediatric CBF-AML patients as compared to controls. Low miR-196b expression was associated with poor disease-free and overall survival and low expression of miR-4772-5p was found to be an independent predictor of poor event-free survival. Except PAX-5, other predicted gene targets were found to be upregulated in pediatric CBF-AML patients. Conclusion: miR-196b and miR-4772-5p may be considered as independent prognostic biomarkers in pediatric CBF-AML. The miRNA-mRNA interaction network suggests potential involvement of miRNAs, which needs to be further, confirmed using mechanistic studies.
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