Fibrolamellar variant of hepatocellular carcinoma does not have a better survival than conventional hepatocellular carcinoma--results and treatment recommendations from the Childhood Liver Tumour Strategy Group (SIOPEL) experience

Eur J Cancer. 2013 Aug;49(12):2698-704. doi: 10.1016/j.ejca.2013.04.012. Epub 2013 May 15.


Purpose: Fibrolamellar hepatocellular carcinoma (FL-HCC) and conventional hepatocellular carcinoma (HCC) cases in two consecutive paediatric HCC trials were analysed to compare outcome and derive treatment implications.

Patients and methods: Data of 24 FL-HCC (24% PRETEXT IV) and 38 HCC (42% PRETEXT IV) cases from SIOPEL-2 and -3 (1995-1998, 1998-2006) were analysed. Patients were treated according to SIOPEL-2 and -3 high-risk protocol (carboplatin+doxorubicin alternating with cisplatin; seven preoperative, three postoperative cycles) or with primary surgery followed by chemotherapy as indicated.

Results: Thirteen of 24 FL-HCC (54%) and 32/38 HCC (84%) were initially treated with chemotherapy. Eight FL-HCC (33%) and five HCC patients (13%) had primary surgery. Partial response was observed in 31% of FL-HCC versus 53% of HCC patients (p=0.17). Complete resection was achieved in ten FL-HCC and seven HCC patients (p=0.08). Three-year event free survival (EFS) was 22% for FL-HCC versus 28% for HCC. Overall survival (OS) was not significantly different at 3 years follow up (42% for FL-HCC versus 33% for HCC, p=0.24). EFS/OS Kaplan-Meier curves did not differ significantly, with median follow up of 43 (FL-HCC) and 60 (HCC) months. No significant correlation was found between potential prognostic factors and OS. In the entire cohort nine out of 23 (39%) patients with complete resection or orthotopic liver transplantation versus 34/39 (87%) without successful surgical treatment, died.

Conclusions: Long-term OS in FL-HCC and HCC is similar. With low response rates, complete resection remains the treatment of choice.

Trial registration: NCT00003912 NCT00077389.

Keywords: Antineoplastic agents; Carcinoma; Clinical oncology; Fibrolamellar hepatocellular carcinoma; Hepatocellular; Hepatoma; Liver diseases; Liver neoplasms; Neoplasms; Paediatrics.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Carboplatin / administration & dosage
  • Carcinoma, Hepatocellular / drug therapy*
  • Carcinoma, Hepatocellular / surgery
  • Child
  • Child, Preschool
  • Cisplatin / administration & dosage
  • Cohort Studies
  • Combined Modality Therapy
  • Doxorubicin / administration & dosage
  • Female
  • Hepatectomy / methods
  • Humans
  • Infant
  • Kaplan-Meier Estimate
  • Liver / drug effects*
  • Liver / pathology
  • Liver / surgery
  • Liver Neoplasms / drug therapy*
  • Liver Neoplasms / surgery
  • Male
  • Treatment Outcome


  • Doxorubicin
  • Carboplatin
  • Cisplatin

Supplementary concepts

  • Fibrolamellar hepatocellular carcinoma

Associated data