What is the prognosis of hepatoblastoma?

Prognosis of Hepatoblastoma

Hepatoblastoma has an excellent prognosis with 5-year survival rates of 80% or higher when the tumor is confined to the liver and appropriate treatment is provided, including complete surgical resection and chemotherapy. 1

Disease Overview and Prognostic Factors

Hepatoblastoma is the most common primary hepatic malignancy in children, typically occurring in young children under 5 years of age. The prognosis depends on several key factors:

• Tumor extent and resectability: The most important prognostic factor is whether complete surgical resection can be achieved
• Response to chemotherapy: Platinum-based chemotherapy can significantly reduce tumor volume
• Metastatic status: Presence of metastases, particularly pulmonary metastases, worsens prognosis
• Tumor confinement: Tumors confined to the liver have better outcomes

Staging and Classification

The PRETEXT (Pretreatment Extent of Disease) classification system is used to determine the extent of disease at diagnosis:

• PRETEXT I-II: Excellent prognosis with >90% survival
• PRETEXT III: Good prognosis with appropriate treatment
• PRETEXT IV: More challenging but still potentially curable with aggressive treatment
• Metastatic disease: Requires more intensive therapy but can still achieve good outcomes if metastases respond to chemotherapy

Treatment Outcomes and Survival Rates

Resectable Disease

• Children with initially resectable hepatoblastoma have 5-year survival rates of >90% 1
• Complete surgical resection combined with adjuvant chemotherapy provides the best outcomes 2

Initially Unresectable Disease

• Neoadjuvant chemotherapy can convert up to 78% of initially unresectable tumors to resectable ones 3
• Children with nonresectable hepatoblastoma who undergo liver transplantation have an 82% 10-year survival rate 1

Metastatic Disease

• Patients with pulmonary metastases can still achieve good outcomes if:
  1. Metastases disappear after chemotherapy, or
  2. Residual metastases can be completely resected with tumor-free margins 1

Special Considerations

Liver Transplantation

• Liver transplantation should be considered for children with hepatoblastoma confined to the liver that is not resectable 1
• Primary liver transplantation for unresectable hepatoblastoma has significantly better outcomes (82% 10-year survival) compared to "rescue" transplantation for recurrence after initial resection and chemotherapy (30% 10-year survival) 1

Recurrent Disease

• Recurrent disease carries a worse prognosis
• Children who receive liver transplantation for recurrence have approximately 30% 10-year survival 1

Referral Timing and Recommendations

Early referral to specialized centers is critical for optimal outcomes:

• Children with nonmetastatic and otherwise unresectable hepatoblastoma should be referred for liver transplant evaluation at diagnosis or no later than after 2 rounds of chemotherapy 1
• Patients with pulmonary metastases can be considered for liver transplantation if metastases clear with chemotherapy or can be completely resected 1

Long-term Outcomes

With modern treatment approaches combining surgery and chemotherapy, long-term disease-free survival is achievable:

• Complete disease remission for 15+ years has been documented in patients receiving optimal combined treatment 4
• The overall survival rate has dramatically improved from 25-30% historically to 75-80% with current combination therapy 2

Hepatoblastoma prognosis has significantly improved over recent decades due to advances in surgical techniques, chemotherapy regimens, and transplantation options, making it one of the more curable pediatric solid tumors when appropriate multidisciplinary care is provided.