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Updated: Aug 17, 2025
A Three-Dimensional Spheroid Model to Investigate the Tumor-Stromal Interaction in Hepatocellular Carcinoma
Published on: September 30, 2021
State of the art and perspectives in pediatric hepatocellular carcinoma
Graziana Digiacomo1, Roberta Pia Serra2, Elena Turrini2
1Department of Medicine and Surgery, University of Parma, Parma, Italy.
Insights
Pediatric liver cancers, hepatoblastoma (HB) and hepatocellular carcinoma (HCC), have different prognoses. While surgery offers a cure for HB, pediatric HCC has a poor outlook, prompting research into new treatments like targeted therapies.
Area of Science:
- Pediatric Oncology
- Hepatology
- Cancer Genetics
Background:
- Hepatoblastoma (HB) and pediatric hepatocellular carcinoma (HCC) are rare childhood liver cancers.
- HB is more common and has a better prognosis (>80% 5-year survival) than pediatric HCC (<30% 5-year survival).
- Current treatments for pediatric HCC, including cisplatin and doxorubicin (PLADO) chemotherapy, show limited efficacy.
Purpose of the Study:
- To review the current understanding of pediatric HCC, including treatment challenges and emerging therapeutic strategies.
- To highlight the role of molecular pathways, such as Wnt/CTNNB1, in pediatric HCC pathogenesis.
- To explore the potential of targeted therapies and immunotherapies for improving pediatric HCC outcomes.
Main Methods:
- Review of existing literature on pediatric HB and HCC.
- Analysis of clinical trial data, including the PHITT trial investigating sorafenib.
- Exploration of genetic profiling studies to identify molecular drivers of pediatric HCC.
Main Results:
- Pediatric HCC has a dismal prognosis compared to HB, with limited response to standard chemotherapy.
- Sorafenib, a multi-kinase inhibitor, is being investigated in the PHITT phase III trial for pediatric HCC.
- The Wnt/CTNNB1 pathway is implicated in the pathogenesis of pediatric HCC, similar to HB and adult HCC.
Conclusions:
- Surgery remains the primary curative treatment for pediatric HCC, but novel therapeutic approaches are urgently needed.
- Targeted therapies and immunotherapies, informed by molecular profiling, hold promise for improving pediatric HCC treatment.
- Further research into the genetic landscape of pediatric HCC is crucial for developing effective biological and immunotherapeutic agents.
Abstract:
Hepatoblastoma (HB) and pediatric hepatocellular carcinoma (HCC) are rare primary malignant liver cancers in children and young adults. HB is the most common and accounts for about 70 % cases; it is usually diagnosed during the first 3 years of life. Instead, pediatric HCC is uncommon, and it is associated with a poor prognosis. Overall, the prognosis of pediatric HCC is dismal with 5-year event-free survival of <30 % as compared to >80 % for HB. Surgery approaches, either resection or transplant, remain the best chance for the cure of pediatric HCC. However, chemotherapy can be helpful as an adjuvant or neoadjuvant treatment. International groups have done trials in pediatric HCC with a chemotherapy regimen, based on cisplatin and doxorubicin (PLADO) as for HB, but the efficacy is limited. Sorafenib, a multi-kinase inhibitor, following positive results in adults and in a pilot study in children, is now tested in conjunction with chemotherapy in the PHITT phase III clinical trial. Some studies have been exploring the genetic profiles of patients to find biological hallmarks that determine the aggressiveness of pediatric HCC. Pathways involved in growth and differentiation are dysregulated and as demonstrated in HB and adult HCC, an important role of the Wnt/CTNNB1 pathway in the pathogenesis of pediatric HCC is also emerging. An extended molecular analysis of tumor samples could give information about pathways as possible targets of biological and immunotherapeutic agents bringing new pharmacological options for the treatment of pediatric HCC.
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