Fibrolamellar Carcinoma: Recent advances and unresolved questions on the molecular mechanisms

摘要

Fibrolamellar hepatocellular carcinoma (FLC) is a rare form of primary liver cancer that affects adolescents and young adults without underlying liver disease. Surgery remains the mainstay of therapy, however most patients are either not surgical candidates, or suffer from recurrence. There is no approved systemic therapy and the overall survival remains poor. Historically classified as a subtype of Hepatocellular carcinoma (HCC), FLC has a unique clinical, histological and molecular presentation. At the genomic level FLC contains a single 400kB deletion in chromosome 19, leading to a functional DNAJB1-PRKACA fusion protein. In this review we detail the recent advances in our understanding of the molecular underpinnings of FLC and outline the current knowledge gaps.