Rare Tumors Program North American Hepatocellular Carcinoma Registry

About the North American Hepatocellular Carcinoma Registry

Hepatocellular carcinoma (HCC) is a rare cancer of the liver affecting approximately 90 children in the US each year. It is the second most common type of liver cancer in children after hepatoblastoma. Among children older than five, it is the most common type of liver cancer. Children who have hepatocellular carcinoma are typically diagnosed when their parents or doctor notice an abdominal mass or fullness.

Complete surgical removal of hepatocellular carcinoma is the critical component of therapy. Chemotherapeutic approaches have limited efficacy, but may provide longer survival. Alternative therapeutic options include administration of chemotherapy (trans-arterial chemoembolization, TACE) or radioactive yttrium-90 loaded microspheres (trans-arterial radioembolization, TARE) into the blood vessels supplying the liver tumor. Additionally, the tumors can be damaged by radiofrequency ablation (RFA) directly. These approaches; however, cannot eliminate cancer cells which have spread outside of the liver.

Given the poor outcome of children with hepatocellular carcinoma who have metastatic disease, there is an urgent need to find effective therapies and immunotherapy may fill this need. Our center has developed an immunotherapeutic approach using T cells using glypican-3-specific chimeric antigen receptor. More information is available here: NCT02932956.

Registry goals

• To enroll all children diagnosed with hepatocellular carcinoma in North America and collect clinical and biological information
• To serve as a resource for patients, their families and treating physicians
• To ensure optimal treatment of children with this rare form of liver cancer
• To serve as a resource for researchers involved in pediatric liver cancer research

Why to Enroll

Due to the rarity, there are still unanswered questions for this disease:

• Are there children that may not need chemotherapy after successful surgical resection or transplantation?
• What is the optimal therapy for children with metastatic HCC?
• Are there predictive factors that could guide intensity of chemotherapy after surgery?
• What is the frequency of fibrolamellar variant form of HCC in children?
• What is the frequency of glypican-3 positivity in pediatric HCC?

The North American Hepatocellular Carcinoma Registry was established at Texas Children’s Hospital to find answers to these questions and improve outcome of children with this rare form of liver cancer.

How to enroll

If your child (age <18) is diagnosed with hepatocellular carcinoma and you are interested in finding more information about this registry, please contact us by emailing raretumors@texaschildrens.org or by calling 832-824-6835.

Registry personnel

Andras Heczey, MD, Pediatric Oncologist
Valeria Smith, MD, Pediatric Oncologist
Sanjeev A. Vasudevan, MD, Pediatric Surgical Oncologist
Prakash Masand, MD, Pediatric Radiologist
Dolores Lopez-Terrada, MD, PhD, Pediatric Pathologist
Rajkumar Venkatramani, MD, Pediatric Oncologist
Josalind Randall, research coordinator