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Predisposing Conditions to Pediatric Hepatocellular Carcinoma and Association With Outcomes: Single-center Experience
Elizabeth Cowell1, Kalyani Patel2, Andras Heczey3
1Department of Pediatrics.
Insights
Most children with hepatocellular carcinoma (HCC) lack predisposing liver disease. These children are diagnosed later with advanced disease and have significantly poorer survival outcomes.
Area of Science:
- Pediatric Oncology
- Hepatology
- Cancer Research
Background:
- Hepatocellular carcinoma (HCC) in children is often associated with chronic liver diseases.
- Characteristics and outcomes of pediatric HCC, especially in relation to predisposing conditions, require further elucidation.
Purpose of the Study:
- To characterize pediatric hepatocellular carcinoma (HCC) patients.
- To define outcomes in children with HCC, particularly concerning predisposing liver disease.
Main Methods:
- Retrospective review of 61 pediatric HCC cases (age ≤21) managed between 1996-2016.
- Subgroup analysis based on presence or absence of predisposing conditions.
- Survival analysis using Kaplan-Meier method.
Main Results:
- 44% of patients had predisposing conditions (e.g., cirrhosis, steatosis, genetic disorders).
- De novo HCC (no predisposing condition) patients were diagnosed at a later age (10.2 vs 7.2 years) with larger tumors (>4cm, 100% vs 20%).
- Patients with de novo HCC had significantly decreased overall survival compared to those with predisposing conditions.
Conclusions:
- The majority of pediatric HCC cases occur without predisposing liver disease.
- Children with de novo HCC present with more advanced disease and have worse survival.
- Timely diagnosis and treatment strategies are crucial for improving outcomes in pediatric HCC.
Objectives:
Hepatocellular carcinoma (HCC) has been linked to chronic viral or metabolic liver disease and other conditions. The characteristics of children with HCC have not been fully elucidated and outcomes in children with predisposing liver disease are not well defined.
Methods:
Patients ≤21 years old with HCC managed at our institution and through external consultation between 1996 and 2016 were included. Demographics, clinical history, and pathology were tabulated. Fisher exact test and Wilcoxon test were employed for subgroup comparison, and survival differences were evaluated by Kaplan-Meier method.
Results:
Sixty-one cases of HCC were identified. Seven of 16 patients (44%) at our institution and 18 of 45 consult patients (40%) had a predisposing condition: cryptogenic cirrhosis/steatosis (9), genetic (7), biliary pathology (4), viral hepatitis (1), and other (4). Thirteen of 27 patients with de novo HCC had fibrolamellar HCC. Clinical characteristics were grouped by presence or absence of predisposing conditions: age at diagnosis (7.2 vs 10.2 years, P < 0.05), metastatic disease at presentation (15% vs 44%, P = n.s), and tumor size >4 cm (20% vs 100%, P < 0.05). In patients treated at our institution, 5 of 7 with predisposing conditions received liver transplant and achieved complete remission, whereas only 3 of 9 patients with de novo HCC received curative surgery and this group had decreased median overall survival (P < 0.05).
Conclusions:
The majority of children with HCC did not have predisposing liver or associated disease. These patients were diagnosed later with more advanced stage disease and had significantly decreased overall survival.
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