New approach optimizes treatment of hepatoblastoma cancer

A study from Children’s Hospital Los Angeles validates a new risk assessment for hepatoblastoma and finds that certain histological features also predict prognosis.

LOS ANGELES, February 14, 2022–(BUSINESS WIRE)–A new study at Children’s Hospital Los Angeles has independently verified the value of a system that assesses the risk of hepatoblastoma in children. Hepatoblastoma is a rare childhood liver cancer typically seen in the first three years of a child’s life, with 50 to 70 cases occurring in the United States each year. The researchers also discovered the potential of tumor histology – the examination of a tumor’s tissue and its structure – to predict a patient’s hepatoblastoma prognosis.

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Leo Mascarenhas, MD, MS, associate director of the Cancer and Blood Disease Institute at Children’s Hospital Los Angeles (Photo: Business Wire)

Leo Mascarenhas, MD, MS, associate director of the Institute of Cancer and Blood Diseases, co-directed the study with Shengmei Zhou, MD, pediatric pathologist and diagnostician at CHLA. The results are published in JAMA Network Open.

Hepatoblastoma risk stratification

The risk assessment process, called risk stratification, has the potential to improve success rates for children undergoing treatment for hepatoblastoma. Risk stratification can also reduce unnecessary exposure to chemotherapy. Often the treatment of 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 CHIC-HS (Children’s Hepatic tumors International Collaboration Hepatoblastoma Stratification) International Collaboration. Information such as age at diagnosis, whether the tumor has spread through the body, and alpha-fetoprotein level (which is usually increased in liver cancer) contribute to CHIC-HS risk categorization. . This database of clinical trials aims to establish a common approach to staging and risk stratification. However, the CHIC-HS system has not yet been adopted globally due to a lack of validation.

“Independent validation is valuable for others to use this risk stratification,” emphasizes first author Dr. Zhou. “It gives them confidence.”

Validation of 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 reviewed electronic medical records, imaging, and pathology of 96 patients.

The researchers confirmed that the CHIC-HS system successfully predicts the risk posed by a tumour. Children in low-risk categories improved with minimal treatment. Those in high-risk groups required a more intense treatment approach.

The CHIC-HS system was also consistent with patients’ long-term outcomes after treatment. These outcomes include overall survival and how long a patient lives without cancer relapse or progression.

For 84 of the patients included in the study, tumor histology collected before treatment was available for analysis. Pretreatment biopsies, which provide tissue samples from the tumor, are not always possible or commonly collected. Having a cohort of this size offered a unique opportunity to assess the relationship between tumor histological characteristics, risk category and patient survival.

The researchers found that certain histological characteristics predicted patient risk and long-term outcomes, including relapse and survival rates. “Histology can help further improve the risk stratification scale,” concludes Dr. Mascarenhas. “Our hope is that our work will be proven in the ongoing international pediatric liver tumor trial.” (NCT03533582, North America; NCT03017326, Europe).

Ongoing research at CHLA is focused on identifying genetic markers that may further contribute to risk stratification and inform the treatment of hepatoblastoma.

“This cancer, which used to be quite a deadly cancer, is now highly curable in many patients,” says Dr. Mascarenhas. “A lot of our goals should really be about limiting the toxicity of treatment in these patients and, for those with high-risk disease, figuring out how to improve their outcomes.”

The other authors of the article are: Jemily Malvar, MS, of Children’s Hospital Los Angeles and Yueh-Yun Chi, PhD, James Stein, MD, Larry Wang, MD, PhD, Yuri Genyk, MD, and Richard Sposto, PhD, from Children’s Hospital Los Angeles and the Keck School of Medicine at USC.

This research was supported in part by grants UL1TR001855 from the National Center for Advancing Translational Science and 5P30CA014089-44 from the National Cancer Institute of the National Institutes of Health of the United States. The research also received support from the Society for Pediatric Pathology Young Investigator Research Grant and the Names Family Foundation.

About Children’s Hospital Los Angeles

Founded in 1901, Children’s Hospital Los Angeles is the highest-ranked children’s hospital in California and fifth in the nation on the prestigious U.S. News & World Report Honor Roll of Best Children’s Hospitals. US News ranks Children’s Hospital Los Angeles in all 10 specialty categories. Clinical care at the hospital is led by faculty physicians from USC’s Keck School of Medicine through an affiliation dating back to 1932. The hospital also operates the largest residency training program in pediatrics at a free-standing children’s hospital in the western United States. The Saban Research Institute at Children’s Hospital Los Angeles houses all of the basic, translational, clinical, and community-based research conducted at the hospital, enabling proven discoveries to reach patients quickly. Our mission: to create hope and build a healthier future. To learn more, follow us on Facebook, Instagram, LinkedIn, YouTube and Twitterand visit our blog at

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Marlen Bugarin
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