# Validation of Clinical Assays for Risk Stratification of Children With Pediatric Liver Neoplasms

> **NIH NIH UH3** · BAYLOR COLLEGE OF MEDICINE · 2024 · $316,352

## Abstract

Project Summary
Hepatoblastoma (HB) and hepatocellular carcinoma (HCC) are the most frequently diagnosed liver tumors in
children, with HBs most commonly present in young children less than 5 years of age, and HCCs are more
commonly seen in adolescents. HB- and HCC-patient outcomes and treatment options vary dramatically, with
5-year overall survival rates of over 70% for HB and under 30% for HCC patients. While a combination of
chemotherapy and surgery is effective for lower-risk HBs, 3-year overall survival for high-risk HBs is 50%. High-
dose chemotherapy, which is often ineffective for high-risk HBs, is associated with significant morbidity.
Complete surgical resection is the only chance for a cure for HCC. Molecular biomarkers can help optimize
treatments for patients that will not benefit from chemotherapy or that do not require high dosage chemotherapy
and identify patients that require a combination of aggressive surgery and chemotherapy.
In previous work, we, and our collaborators, identified prognostic biomarkers that distinguish between low- and
high-risk HBs at diagnosis. We proposed and retrospectively evaluated predictive models to classify patients
based on risk—including their need for aggressive therapies. These models identify patients that do not require
aggressive therapies, patients that will benefit from aggressive therapies, and patients with tumors that are more
likely to metastasize and become resistant to chemotherapy. We developed and certified molecular assays to
profile patients and tumors for predictive biomarker used by these models. Here, we propose to prospectively
validate these biomarkers, assays, and models to produce the first validated platform for the molecular diagnosis
and therapy choice for HBs and HCC. We will benefit from a close collaboration with clinical-trial produced data
in the USA and EU, including AHEP1531 (USA), ChILTERN (EU) and iPC (a EU-USA collaboration).

## Key facts

- **NIH application ID:** 10868737
- **Project number:** 5UH3CA271227-02
- **Recipient organization:** BAYLOR COLLEGE OF MEDICINE
- **Principal Investigator:** Dolores Lopez-Terrada
- **Activity code:** UH3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $316,352
- **Award type:** 5
- **Project period:** 2023-07-01 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10868737

## Citation

> US National Institutes of Health, RePORTER application 10868737, Validation of Clinical Assays for Risk Stratification of Children With Pediatric Liver Neoplasms (5UH3CA271227-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10868737. Licensed CC0.

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