Clinicopathological features and treatment outcomes of patients with fibrolamellar hepatocellular carcinoma: a retrospective multicenter study

Abstract

Background: Fibrolamellar Hepatocellular Carcinoma (FLHCC) is a rare primary liver tumour that is distinct from conventional HCC in both histopathological and clinical features. There is no clear consensus on its treatment.

Objective: Assess the demographics, prognosis, and treatment- particularly systemic therapies-of FLHCC patients across multiple centers in Türkiye.

Design: Retrospective.

Settings: Multicenter.

Patients and methods: Patients with histopathological diagnosis of pure fibrolamellar hepatocellular carcinoma.

Main outcome measures: Treatment responses of metastatic stage patients were evaluated. Progression-free survival (PFS) and overall survival (OS) analyses of metastatic stage first-line treatments were performed.

Sample size: 39 patients with FLHCC.

Results: The 5-year survival for all patients was 48%, 80% in stage 1, 57% in stage 2, 53% in stage 3 and 0% in stage 4. The median PFS for chemotherapy (n=10) and sorafenib (n=6) patients in the metastatic stage first series was 5.7 months and 2.8 months, respectively (P=.031). Median OS was 12.1 months for chemotherapy and 8.8 months for sorafenib (P=.853) in the metastatic stage.

Conclusion: Conventional chemotherapies, especially gemcitabine and oxaliplatin combination can be used as systemic treatment options. Immunotherapies, chemoimmunotherapy and immunotherapy plus anti vascular endothelial growth factor combinations may be considered by clinicians.

Limitations: Small sample size and the variability of the treatment modalities administered in the patients.

Keywords: DNAJB1-PRKACA; Fibrolamellar HCC; Gemcitabine-oxaliplatin; Immunotherapy; Sorafenib.

Figure 1.

Kaplan-Meier curves of patients with Fibrolamellar hepatocelüler carcinoma by stages.

Figure 2.

Progression free survival and overall survival curves by treatments group.