[Treatment outcome of 100 patients with hepatoblastoma based on a new risk stratification]

Z Z Zhen1, J C Liu2, L Zhou2

  • 1Department of Pediatric Oncology, Sun Yat-sen University Cancer Center, State Key Laboratory of Oncology in South China, Guangzhou 510060, China.

Insights

A new risk stratification for hepatoblastoma (HB) shows excellent outcomes for low-risk patients. High-risk hepatoblastoma patients still require improved survival rates and novel treatment strategies.

Area of Science:

  • Pediatric Oncology
  • Hepatobiliary Malignancies
  • Cancer Risk Stratification

Background:

  • Hepatoblastoma (HB) is a rare pediatric liver cancer.
  • Effective risk stratification is crucial for tailoring treatment and improving outcomes in HB patients.
  • Current risk stratification methods may not be optimally suited for all populations, necessitating localized approaches.

Purpose of the Study:

  • To develop and validate a risk stratification method for hepatoblastoma (HB) applicable in China.
  • To explore novel treatment strategies for high-risk HB patients.
  • To evaluate the efficacy of a comprehensive treatment approach based on the new risk stratification.

Main Methods:

  • A cohort of 100 newly diagnosed pediatric HB patients (under 18) were stratified into four risk groups (extremely low, low, intermediate, high) based on clinical stage, AFP level, and pathology.
  • Treatment protocols varied by risk group, including surgery alone, C5V, C5VD, and alternating C5VD/IIV regimens.
  • Chemotherapy response, tumor relapse, progression, and survival rates were assessed over a median follow-up of 20 months.

Main Results:

  • Patients were stratified into extremely low-risk (2%), low-risk (10%), medium-risk (51%), and high-risk (37%) groups.
  • The overall response rate to chemotherapy was 78.3% (65/83 patients).
  • Two-year progression-free survival (PFS) and overall survival (OS) rates were 69.2% and 72.0%, respectively. High-risk patients had significantly lower survival rates (PFS 43.2%, OS 44.8%) compared to other groups.

Conclusions:

  • The developed HB risk classification system is simple, feasible, and effective for implementation in China.
  • Patients classified as extremely low-, low-, and medium-risk demonstrate excellent outcomes with the current comprehensive treatment strategies.
  • Significant improvements in survival rates for high-risk HB patients are needed, highlighting the necessity for developing and investigating new therapeutic interventions.

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