Fibrolamellar carcinoma: An entity all its own

O’Neill, Allison F.; Church, Alanna J.; Perez‐Atayde, Antonio R.; Shaikh, Raja; Marcus, Karen J.; Vakili, Khashayar

doi:10.1016/j.currproblcancer.2021.100770

Cited by 34 publications

(43 citation statements)

References 77 publications

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“…21,22 The transcription of this DNAJ-PKAc chimeric protein is expressed exclusively in FLC, in what has been shown to be a somatic mutation. 21,23 The chimera can subsequently be separated into specific subcellular locations by fusion into the A-Kinase Anchoring Protein (AKAP) signaling complexes. 24,25 Attributed to the DNAJ part, the protein interacts with the chaperonin heat shock protein 70 (Hsp70).…”

Section: Pathophysiologymentioning

confidence: 99%

“…43 At the moment there is one clinical trial recruiting patients for a DNAJB1-PRKACA Fusion Kinase Peptide Vaccine Combined with Nivolumab and Ipilimumab and one trial for Pembrolizumab in pediatric patients. 23,64 Generally, chemotherapy in combination with surgery showed overall worse survival than surgery alone. From our point of view, this might be due to an already advanced tumor stage at time point of surgery necessitating (neo-) adjuvant treatment.…”

Section: Chemotherapymentioning

confidence: 99%

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A Multidisciplinary Approach to the Management of Fibrolamellar Carcinoma: Current Perspectives and Future Prospects

Polychronidis¹,

Murtha-Lemekhova²,

Fuchs³

et al. 2022

OTT

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Fibrolamellar carcinoma (FLC) is a rare primary liver tumor affecting predominantly younger and otherwise healthy patients. Typically, FLC presents with advanced disease due to the paucity of typical symptoms and no history of underlying liver disease. Depending on tumor characteristics and the patient's general condition, surgical treatment is the most promising treatment modality. Aggressive resection and liver transplantation have been utilized and are presently indispensable curative treatment options. Under certain circumstances surgical resection is also possible for metachronous metastases or local recurrence. Recent tumor biology discoveries have contributed to improved diagnostic specificity and systemic treatment options.

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Section: Pathophysiologymentioning

confidence: 99%

Section: Chemotherapymentioning

confidence: 99%

A Multidisciplinary Approach to the Management of Fibrolamellar Carcinoma: Current Perspectives and Future Prospects

Polychronidis¹,

Murtha-Lemekhova²,

Fuchs³

et al. 2022

OTT

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“…These is no standard systemic therapy for patients with FLC, and the management of unresectable FLC has largely been extrapolated from the management of advanced hepatocellular carcinoma (HCC), despite marked biological differences between these cancers [ 10 ]. FLC patients are generally excluded from clinical trials of HCC, and there are limited prospective studies to guide therapeutic selection in the treatment of FLC.…”

Section: Introductionmentioning

confidence: 99%

Clinical Outcomes in Fibrolamellar Hepatocellular Carcinoma Treated with Immune Checkpoint Inhibitors

Chen

Popović

Hsiehchen

et al. 2022

Cancers

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Background: Fibrolamellar hepatocellular carcinoma (FLC) is a rare form of liver cancer primarily affecting children and young adults. Although considered a subset of hepatocellular carcinoma (HCC), FLC has unique molecular and pathologic characteristics, suggesting that it may require different treatment. Immune checkpoint inhibitors (ICIs) are used in the treatment of HCC, but efficacy and safety in FLC has not been characterized. Methods: We performed a multicenter retrospective analysis of patients with FLC to determine responses to ICI therapy. Response rates were assessed based on RECIST 1.1 criteria, and Kaplan–Meier statistics were used for progression-free survival (PFS) and overall survival (OS). Results: FLC tumors were characterized by low tumor mutational burden (TMB) and absent PD-L1 expression. We identified 19 patients who received ICIs, including 15 who received ICI therapy alone [programmed death receptor 1 (PD-1) inhibitor, +/− cytotoxic T lymphocyte antigen-4 (CTLA-4) inhibitor]. Objective tumor responses were observed in 3/19 patients (15.8%), including 2/15 patients (13.3%) who received ICIs alone, all partial responses. Median PFS and OS were 5.5 and 26.0 months, respectively. Grade 3–4 immune related adverse events were observed in 4/19 (21.1%) patients. Conclusions: ICI therapy has modest clinical activity in FLC, and novel therapeutic combinations are needed.

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“…Fibrolamellar hepatocellular carcinoma (FLC) is a rare and often lethal primary liver cancer of adolescents and young adults (1,2). Surgical resection is the current standard of care for FLC; however, this is inadequate to cure patients with locally advanced or metastatic disease.…”

Section: Introductionmentioning

confidence: 99%