Pediatric hepatocellular carcinoma: challenges and solutions

Area of Science:

• Pediatric Oncology
• Hepatocellular Carcinoma Research
• Chemotherapy Efficacy

Background:

• Hepatocellular carcinoma (HCC) is exceptionally rare in children, hindering large-scale clinical trials.
• Pediatric HCC shows sensitivity to chemotherapy agents like cisplatin and doxorubicin (PLADO), unlike in adults.
• Current treatment paradigms emphasize systemic therapies and surgical resection.

Purpose of the Study:

• To review current treatment strategies for pediatric HCC.
• To highlight the chemotherapy sensitivity of childhood HCC.
• To identify gaps in knowledge and future research directions for pediatric HCC.

Main Methods:

• Review of existing literature on pediatric HCC treatment.
• Analysis of survival data based on treatment modalities.
• Comparison of pediatric HCC treatment approaches with adult HCC.

Main Results:

• Complete resection offers 80%-90% 5-year survival for nonmetastatic pediatric HCC.
• Adjuvant chemotherapy (PLADO) is common but its superiority over observation is unproven.
• Survival is poor (<20% at 3 years) for unresectable pediatric HCC, even with neoadjuvant chemotherapy.
• Combinations like PLADO with sorafenib aim to achieve resectability; sorafenib with gemcitabine/oxaliplatin shows promise.
• Milan criteria for liver transplantation are not applicable to pediatric nonresectable liver tumors.

Conclusions:

• Pediatric HCC, though rare, responds to chemotherapy, guiding treatment towards systemic drugs and resection.
• While survival is high post-resection, the role of adjuvant chemotherapy needs clarification.
• Unresectable cases have poor outcomes, necessitating novel therapeutic strategies.
• Research into molecular mechanisms is vital for developing targeted therapies and personalized medicine in pediatric HCC.