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Yang et al. Hepatoma Res 2023;9:48 https://dx.doi.org/10.20517/2394-5079.2023.68 Page 17 of 21

Targeted therapy is a type of and the foundation of precision medicine. The latter needs to identify
biological information of genes, RNA, and proteins to stratify patients, selecting patients most likely to
benefit from a specific therapy. Therefore, it is crucial to determine the precise targets and select the
appropriate agents for the optimized efficacy of treatment. Although high-throughput sequencing of tumor
tissue or peripheral blood can provide more comprehensive genetic profiling, the molecular heterogeneity
and biological regulation of cancer as iCCA never occur in genomics alone. In the near future, new
advances in epigenomics, transcriptomics, proteomics, and metabolomics will be indispensable for the
improvement of tailored treatments for patients with iCCA.

CONCLUSION
In the past decade, the recognition of the molecular and genomic characteristics of iCCA has led to a
paradigm shift in treatment approaches and strategies. Next-generation sequencing revealed that the
majority of iCCA patients harbor at least one actionable mutation, prompting numerous clinical trials to
evaluate the efficacy and safety of novel therapies targeting tumor-specific molecules or pathways. Although
promising results have been obtained in extensive trials, large-scale randomized phase 3 trials of first-in-
class agents and innovative regimens are encouraged to benefit the population with iCCA.

DECLARATIONS
Authors’ contributions
Contribute equally to this work, and were major contributors in collecting literature and drafting the
manuscript: Yang F, Qiu Y
in charge of the methodology and administrated the project and reviewed the manuscript: Yi B

Availability of data and materials
All data generated or analysed during this study are included in this published article and its additional
information files.

Financial support and sponsorship
This study was financially supported by the National Natural Science Foundation of China (Dr Bin Yi, No.
82173213) and by the Shanghai Natural Science Foundation (Dr Bin Yi, No.21Y11912500).

Conflicts of interest
All authors declared that there are no conflicts of interest.

Ethical approval and consent to participate
Not applicable.

Consent for publication
Not applicable.

Copyright
© The Author(s) 2023.

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