A new study published by Zhou et al in JAMA Network Open independently verified the value of a system that assesses hepatoblastoma risk in children. The researchers also discovered the potential for tumor histology to predict a patient’s hepatoblastoma prognosis.
Hepatoblastoma is a rare childhood liver cancer, usually seen within the first 3 years of a child’s life, with 50 to 70 cases occurring in the United States each year.
Risk Stratification for Hepatoblastoma
The risk assessment process, or risk stratification, has the potential to improve success rates for children undergoing hepatoblastoma treatment. Risk stratification can also reduce unnecessary exposure to chemotherapy. Often, treatment for low-risk cases is too aggressive, or liver transplants are not prioritized for high-risk cases.
In 2016, leading hepatoblastoma researchers from around the world created the Children’s Hepatic Tumor International Collaboration–Hepatoblastoma Stratification (CHIC-HS). Information such as age at diagnosis, whether the tumor has spread within the body, and alpha fetoprotein level (which is usually increased in liver cancer) contributes to the CHIC-HS risk categorization. This database of clinical trial data aims to establish a common approach to staging and risk stratification. The CHIC-HS system has yet to be globally adopted, however, due to a lack of validation.
“Independent validation is valuable for others to use this risk stratification,” emphasized first study author Shengmei Zhou, MD, of Children’s Hospital Los Angeles and the Keck School of Medicine, University of Southern California. “It gives them confidence.”
Validating the CHIC-HS System
The study retrospectively tested the CHIC-HS system on an independent cohort of patients diagnosed and treated at Children’s Hospital Los Angeles from 2000 to 2016. The team examined the electronic medical records, imaging, and pathology of 96 patients.
The investigators confirmed that the CHIC-HS system successfully predicts how much risk a tumor poses. Children in the lower-risk categories improved with minimal treatment; those in higher-risk groups required a more intense treatment approach.
The CHIC-HS system also corresponded with long-term patient outcomes after treatment including overall and progression-free survival.
For 84 of the patients included in the study, tumor histology collected before treatment was available for analysis. Having a cohort of this size offered a unique opportunity to assess the relationship between histologic characteristics in the tumor, risk category, and patient survival.
The investigators discovered that certain histologic features predicted patient risk and long-term outcomes, including relapse and survival rates.
“Histology may help further enhance the risk stratification scale,” added senior author Leo Mascarenhas, MD, MS, of Children’s Hospital Los Angeles. “Our hope is that our work will be proven in the ongoing Pediatric Hepatic International Tumor Trial.”
Ongoing research is focusing on the identification of genetic markers that may further contribute to risk stratification and inform hepatoblastoma treatment.
“This cancer, from being a pretty deadly cancer, is now highly curable in a lot of patients,” said Dr. Mascarenhas. “A lot of our goals should really be aimed at limiting the toxicity of treatment in these patients and, for those with high-risk disease, figuring out how to improve their outcomes.”
Disclosure: This research was funded in part by the National Center for Advancing Translational Science and the National Cancer Institute. The research also received support from the Society for Pediatric Pathology Young Investigator Research Grant and the Names Family Foundation. For full disclosures of the study authors, visit jamanetwork.com.
