BACKGROUND: Fibrolamellar hepatocellular carcinoma (FLC) is a rare primary liver tumor presenting earlier in life than nonfibrolamellar hepatocellular carcinoma (NFL-HCC), with distinct epidemiologic and clinical characteristics. Although FLC is believed to have a better prognosis than NFL-HCC, data on treatment and prognosis are scarce. We performed a systematic review to investigate treatment options and clinical outcomes of patients with FLC. STUDY DESIGN: The study is a systematic review of the literature and pooled analysis of individual patient data. RESULTS: A total of 35 series were analyzed, reporting on 575 patients (52% female, elevated alpha-fetoprotein in 10%, cirrhosis in 3%, hepatitis B in 2%), most of whom were treated with partial hepatectomy (55%) or orthotopic liver transplantation (23%). Nineteen studies provided data on 206 individual patients with a median age of 21 years and tumor size of 12 cm. Median overall survival (OS) was 39 months; 1-year, 3-year, and 5-year OS rates were 85%, 53%, and 44%, respectively. For patients treated with liver resection, median OS was 18.5 years and 1-year, 3-year, and 5-year OS were 93%, 80%, and 70%, respectively. Based on data from 15 studies, FLC appeared to follow a relatively indolent course compared with NFL-HCC. CONCLUSIONS: Patients with FLC treated with partial hepatectomy have excellent long-term survival, with 5-year overall survival reaching 70%. Patients fared worse with the use of other therapeutic options including chemotherapy, intra-arterial therapy, and transplantation, although data directly comparing resection vs transplantation were limited.
BACKGROUND:Fibrolamellar hepatocellular carcinoma (FLC) is a rare primary liver tumor presenting earlier in life than nonfibrolamellar hepatocellular carcinoma (NFL-HCC), with distinct epidemiologic and clinical characteristics. Although FLC is believed to have a better prognosis than NFL-HCC, data on treatment and prognosis are scarce. We performed a systematic review to investigate treatment options and clinical outcomes of patients with FLC. STUDY DESIGN: The study is a systematic review of the literature and pooled analysis of individual patient data. RESULTS: A total of 35 series were analyzed, reporting on 575 patients (52% female, elevated alpha-fetoprotein in 10%, cirrhosis in 3%, hepatitis B in 2%), most of whom were treated with partial hepatectomy (55%) or orthotopic liver transplantation (23%). Nineteen studies provided data on 206 individual patients with a median age of 21 years and tumor size of 12 cm. Median overall survival (OS) was 39 months; 1-year, 3-year, and 5-year OS rates were 85%, 53%, and 44%, respectively. For patients treated with liver resection, median OS was 18.5 years and 1-year, 3-year, and 5-year OS were 93%, 80%, and 70%, respectively. Based on data from 15 studies, FLC appeared to follow a relatively indolent course compared with NFL-HCC. CONCLUSIONS:Patients with FLC treated with partial hepatectomy have excellent long-term survival, with 5-year overall survival reaching 70%. Patients fared worse with the use of other therapeutic options including chemotherapy, intra-arterial therapy, and transplantation, although data directly comparing resection vs transplantation were limited.
Authors: Elana P Simon; Catherine A Freije; Benjamin A Farber; Gadi Lalazar; David G Darcy; Joshua N Honeyman; Rachel Chiaroni-Clarke; Brian D Dill; Henrik Molina; Umesh K Bhanot; Michael P La Quaglia; Brad R Rosenberg; Sanford M Simon Journal: Proc Natl Acad Sci U S A Date: 2015-10-21 Impact factor: 11.205
Authors: Dai Liu; Guangfu Li; Diego M Avella; Eric T Kimchi; Jussuf T Kaifi; Mark P Rubinstein; E Ramsay Camp; Don C Rockey; Todd D Schell; Kevin F Staveley-O'Carroll Journal: Oncoimmunology Date: 2017-09-21 Impact factor: 8.110
Authors: Joshua N Honeyman; Elana P Simon; Nicolas Robine; Rachel Chiaroni-Clarke; David G Darcy; Irene Isabel P Lim; Caroline E Gleason; Jennifer M Murphy; Brad R Rosenberg; Lydia Teegan; Constantin N Takacs; Sergio Botero; Rachel Belote; Soren Germer; Anne-Katrin Emde; Vladimir Vacic; Umesh Bhanot; Michael P LaQuaglia; Sanford M Simon Journal: Science Date: 2014-02-28 Impact factor: 47.728