Managing fibrolamellar hepatocellular carcinoma

Miral Sadaria Grandhi, Timothy M. Pawlik

Research output: Contribution to journalReview article

Abstract

Introduction: Fibrolamellar hepatocellular carcinoma (FLC) is a rare primary liver malignancy historically designated as a variant of hepatocellular carcinoma (HCC), which comprises less than 1% of all HCC. FLC affects predominantly young people (< 40 years of age) in the absence of underlying liver disease. Diagnosis can be difficult with history and cross-sectional imaging alone, sometimes requiring a core biopsy for definitive diagnosis. Despite presenting with more advanced disease than patients with HCC, FLC has a high resectability rate and improved prognosis over HCC. Unfortunately, most FLC patients will recur and a subset of patients will die of the disease. Currently, FLC has no standardized regimen for systemic therapy. Areas covered: This review covers the diagnosis, management, and recent advancements of FLC. These advancements increase understanding of the genetic profile for FLC and provide potential molecular targets for anticancer therapy. This literature review includes publications from PubMed and clinical trials from ClinicalTrials.gov. Expert opinion: Despite a high resectability rate, many patients with FLC recur. Effective systemic therapy is needed to improve the prognosis of FLC. The recent discovery of chimeric transcript DNAJB1-PRKACA in FLC holds promise in further understanding the pathogenesis of FLC, becoming a diagnostic marker for FLC, and developing molecular targeted therapy for the treatment of FLC. However, given the rare nature of the disease, collaborative efforts on an international, multi-institutional level in clinical trials, tumor registries for tissue and blood banking, and the development of novel systemic therapies are essential for the future advancement of FLC.

Original languageEnglish (US)
Pages (from-to)143-152
Number of pages10
JournalExpert Opinion on Orphan Drugs
Volume5
Issue number2
DOIs
StatePublished - Feb 1 2017
Externally publishedYes

Fingerprint

Hepatocellular Carcinoma
Fibrolamellar hepatocellular carcinoma
Clinical Trials
Tissue Banks
Molecular Targeted Therapy
Therapeutics
Blood Banks
Expert Testimony
Rare Diseases
PubMed
Registries
Publications
Liver Diseases
Neoplasms
History
Biopsy
Liver

Keywords

  • DNAJB1-PRKACA
  • Fibrolamellar hepatocellular carcinoma
  • hepatectomy
  • hepatocellular carcinoma
  • orthotopic liver transplantation
  • recurrence

ASJC Scopus subject areas

  • Pharmacology, Toxicology and Pharmaceutics (miscellaneous)
  • Health Policy
  • Pharmacology (medical)

Cite this

Managing fibrolamellar hepatocellular carcinoma. / Grandhi, Miral Sadaria; Pawlik, Timothy M.

In: Expert Opinion on Orphan Drugs, Vol. 5, No. 2, 01.02.2017, p. 143-152.

Research output: Contribution to journalReview article

Grandhi, Miral Sadaria ; Pawlik, Timothy M. / Managing fibrolamellar hepatocellular carcinoma. In: Expert Opinion on Orphan Drugs. 2017 ; Vol. 5, No. 2. pp. 143-152.
@article{8d3c3fa2cdae451babe41ccded0d86e1,
title = "Managing fibrolamellar hepatocellular carcinoma",
abstract = "Introduction: Fibrolamellar hepatocellular carcinoma (FLC) is a rare primary liver malignancy historically designated as a variant of hepatocellular carcinoma (HCC), which comprises less than 1{\%} of all HCC. FLC affects predominantly young people (< 40 years of age) in the absence of underlying liver disease. Diagnosis can be difficult with history and cross-sectional imaging alone, sometimes requiring a core biopsy for definitive diagnosis. Despite presenting with more advanced disease than patients with HCC, FLC has a high resectability rate and improved prognosis over HCC. Unfortunately, most FLC patients will recur and a subset of patients will die of the disease. Currently, FLC has no standardized regimen for systemic therapy. Areas covered: This review covers the diagnosis, management, and recent advancements of FLC. These advancements increase understanding of the genetic profile for FLC and provide potential molecular targets for anticancer therapy. This literature review includes publications from PubMed and clinical trials from ClinicalTrials.gov. Expert opinion: Despite a high resectability rate, many patients with FLC recur. Effective systemic therapy is needed to improve the prognosis of FLC. The recent discovery of chimeric transcript DNAJB1-PRKACA in FLC holds promise in further understanding the pathogenesis of FLC, becoming a diagnostic marker for FLC, and developing molecular targeted therapy for the treatment of FLC. However, given the rare nature of the disease, collaborative efforts on an international, multi-institutional level in clinical trials, tumor registries for tissue and blood banking, and the development of novel systemic therapies are essential for the future advancement of FLC.",
keywords = "DNAJB1-PRKACA, Fibrolamellar hepatocellular carcinoma, hepatectomy, hepatocellular carcinoma, orthotopic liver transplantation, recurrence",
author = "Grandhi, {Miral Sadaria} and Pawlik, {Timothy M.}",
year = "2017",
month = "2",
day = "1",
doi = "10.1080/21678707.2017.1268050",
language = "English (US)",
volume = "5",
pages = "143--152",
journal = "Expert Opinion on Orphan Drugs",
issn = "2167-8707",
publisher = "Informa Healthcare",
number = "2",

}

TY - JOUR

T1 - Managing fibrolamellar hepatocellular carcinoma

AU - Grandhi, Miral Sadaria

AU - Pawlik, Timothy M.

PY - 2017/2/1

Y1 - 2017/2/1

N2 - Introduction: Fibrolamellar hepatocellular carcinoma (FLC) is a rare primary liver malignancy historically designated as a variant of hepatocellular carcinoma (HCC), which comprises less than 1% of all HCC. FLC affects predominantly young people (< 40 years of age) in the absence of underlying liver disease. Diagnosis can be difficult with history and cross-sectional imaging alone, sometimes requiring a core biopsy for definitive diagnosis. Despite presenting with more advanced disease than patients with HCC, FLC has a high resectability rate and improved prognosis over HCC. Unfortunately, most FLC patients will recur and a subset of patients will die of the disease. Currently, FLC has no standardized regimen for systemic therapy. Areas covered: This review covers the diagnosis, management, and recent advancements of FLC. These advancements increase understanding of the genetic profile for FLC and provide potential molecular targets for anticancer therapy. This literature review includes publications from PubMed and clinical trials from ClinicalTrials.gov. Expert opinion: Despite a high resectability rate, many patients with FLC recur. Effective systemic therapy is needed to improve the prognosis of FLC. The recent discovery of chimeric transcript DNAJB1-PRKACA in FLC holds promise in further understanding the pathogenesis of FLC, becoming a diagnostic marker for FLC, and developing molecular targeted therapy for the treatment of FLC. However, given the rare nature of the disease, collaborative efforts on an international, multi-institutional level in clinical trials, tumor registries for tissue and blood banking, and the development of novel systemic therapies are essential for the future advancement of FLC.

AB - Introduction: Fibrolamellar hepatocellular carcinoma (FLC) is a rare primary liver malignancy historically designated as a variant of hepatocellular carcinoma (HCC), which comprises less than 1% of all HCC. FLC affects predominantly young people (< 40 years of age) in the absence of underlying liver disease. Diagnosis can be difficult with history and cross-sectional imaging alone, sometimes requiring a core biopsy for definitive diagnosis. Despite presenting with more advanced disease than patients with HCC, FLC has a high resectability rate and improved prognosis over HCC. Unfortunately, most FLC patients will recur and a subset of patients will die of the disease. Currently, FLC has no standardized regimen for systemic therapy. Areas covered: This review covers the diagnosis, management, and recent advancements of FLC. These advancements increase understanding of the genetic profile for FLC and provide potential molecular targets for anticancer therapy. This literature review includes publications from PubMed and clinical trials from ClinicalTrials.gov. Expert opinion: Despite a high resectability rate, many patients with FLC recur. Effective systemic therapy is needed to improve the prognosis of FLC. The recent discovery of chimeric transcript DNAJB1-PRKACA in FLC holds promise in further understanding the pathogenesis of FLC, becoming a diagnostic marker for FLC, and developing molecular targeted therapy for the treatment of FLC. However, given the rare nature of the disease, collaborative efforts on an international, multi-institutional level in clinical trials, tumor registries for tissue and blood banking, and the development of novel systemic therapies are essential for the future advancement of FLC.

KW - DNAJB1-PRKACA

KW - Fibrolamellar hepatocellular carcinoma

KW - hepatectomy

KW - hepatocellular carcinoma

KW - orthotopic liver transplantation

KW - recurrence

UR - http://www.scopus.com/inward/record.url?scp=85009968738&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85009968738&partnerID=8YFLogxK

U2 - 10.1080/21678707.2017.1268050

DO - 10.1080/21678707.2017.1268050

M3 - Review article

AN - SCOPUS:85009968738

VL - 5

SP - 143

EP - 152

JO - Expert Opinion on Orphan Drugs

JF - Expert Opinion on Orphan Drugs

SN - 2167-8707

IS - 2

ER -