Fibrolamellar carcinoma (FLC) is a rare cancer of the liver that most commonly affects children and young adults. There is no clear standard of care for the disease, whose response to treatment seems to be very different from that of hepatocellular carcinoma. We present a case of FLC in a patient in her mid 30s that recurred and persisted despite resection and multiple lines of treatment. Following transcriptomic analysis, a combination of ipilimumab (anti-CTLA4) and nivolumab (anti-PD-1) led to complete remission, although common biomarkers for immune checkpoint blockade were all negative in this case. The patient is still in remission. Here, combined checkpoint blockade guided by novel transcriptomic analysis led to complete remission after failure of several lines of treatment.
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http://dx.doi.org/10.1136/jitc-2022-005620 | DOI Listing |
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9743402 | PMC |
BMJ Case Rep
January 2025
General Surgery, Hospital Universitario HM Sanchinarro, Madrid, Spain
Fibrolamellar hepatocellular carcinoma (FHCC) is a rare variant of hepatocellular carcinoma (HCC), characterised by a poorer prognosis in later stages compared with conventional HCC due to a high rate of local recurrence, lymph node metastasis and peritoneal metastasis. Conventional chemotherapy is generally ineffective, making surgery the only potentially curative treatment. Currently, surgery is also indicated in cases of recurrence, always aiming for an R0 resection.
View Article and Find Full Text PDFBMJ Case Rep
January 2025
General Surgery, Post Graduate Institute of Medical Education and Research, Chandigarh, India
Fibrolamellar hepatocellular carcinoma (FL-HCC) is a primary liver tumour presenting at a young age. Aggressive surgery of FL-HCC is the mainstay of management unlike other malignancies where metastatic stage precludes curative surgery. There are limited reports of response of FL-HCC to systemic therapies predominantly owing to its rarity.
View Article and Find Full Text PDFNat Commun
December 2024
Laboratory of Cellular Biophysics, The Rockefeller University, New York, NY, USA.
Fibrolamellar Hepatocellular Carcinoma (FLC) is a rare liver cancer characterized by a fusion oncokinase of the genes DNAJB1 and PRKACA, the catalytic subunit of protein kinase A (PKA). A few FLC-like tumors have been reported showing other alterations involving PKA. To better understand FLC pathogenesis and the relationships among FLC, FLC-like, and other liver tumors, we performed a massive multi-omics analysis.
View Article and Find Full Text PDFFibrolamellar carcinoma (FLC) is a rare malignancy disproportionately affecting adolescents and young adults with no standard of care. FLC is characterized by thick stroma, which has long suggested an important role of the tumor microenvironment. Over the past decade, several studies have revealed aberrant markers and pathways in FLC.
View Article and Find Full Text PDFLancet
December 2025
Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center, and Harvard Medical School, Boston, MA, USA.
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