Current challenges to underpinning the genetic basis for cholangiocarcinoma.

Current challenges to underpinning the genetic basis for cholangiocarcinoma.
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DOI:
10.1080/17474124.2021.1915128
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发表时间:
2021-05
影响因子:
3.9
通讯作者:
Calvisi DF
Calvisi DF
中科院分区:
医学3区
文献类型:
--
作者:
Cigliano A;Chen X;Calvisi DF

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Cholangiocarcinoma (CCA) is an aggressive cancer type with a dismal prognosis. CCA typically presents at an advanced stage when surgical resection is not feasible, and systemic chemotherapy is generally of limited benefit. Thus, developing effective therapies against this deadly tumor is imperative and remains an unmet need. To provide more effective treatment options, high-throughput approaches have been employed, leading to a better delineation of each CCA subtype’s genomic and transcriptomic landscape as well as the identification of promising candidates for targeted, personalized therapies. In this scenario, the recent approval of pemigatinib, a pan-Fibroblast Growth Factor Receptor (FGFR) inhibitor, for the treatment of the CCA subtype characterized by FGFR2 mutations, represents the first of (presumably) numerous novel therapeutic approaches against this aggressive disease. This review provides an overview regarding the current scenario and knowledge of the genomic landscape occurring in CCA and the potentially actionable molecular aberrations in each CCA subtype. The establishment and advances of high-throughput methodologies applied to genetic and epigenetic profiling are changing the therapeutic landscape of many cancer types, including CCA. These approaches have led to the generation of a large body of data that must be interpreted appropriately and eventually implemented in clinical practice. The following advancements towards precision medicine in CCA management will require designing better clinical trials with improved methods to stratify biliary tumor patients.
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