• Hepatocellular Carcinoma Overview

    Hepatocellular carcinoma, also called liver carcinoma, is a rare, aggressive type of liver cancer. It is more frequently diagnosed in adults and is often seen in conjunction with underlying liver disease (for example, cirrhosis). When diagnosed in pediatric patients, hepatocellular carcinoma is often sporadic but can be linked to hepatitis infection or genetic conditions.

    Hepatocellular Carcinoma Treatment at Dana-Farber/Boston Children's

    Patients with hepatocellular carcinoma are treated at Dana-Farber/Boston Children's through the Liver Tumor Program. We have some of the most experienced pediatric liver cancer oncologists and surgeons in the world, as well as internationally recognized pediatric subspecialists who use sophisticated technology and therapies to maximize outcomes for our patients. Continue reading to learn more about hepatocellular carcinoma or visit the Liver Tumor Program homepage to learn about our expertise.

    Symptoms & Diagnosis

    The symptoms of hepatocellular carcinoma may vary, depending on the size and location of the tumor. Common symptoms include:

    • A large abdominal mass or a swollen abdomen
    • Pain on the right side that may extend to the back and shoulder
    • Back pain from compression of the tumor
    • Decreased appetite and weight loss
    • Vomiting
    • Jaundice
    • Fever
    • Itching skin
    • Anemia

    A child’s physician may order a number of different tests to diagnose hepatocellular carcinoma. In addition to a medical history and physical exam, these may include:

    • Blood tests, including a complete blood count and blood chemistries
    • Liver and kidney function tests
    • Alpha-fetoprotein, a protein often secreted by this type of tumor 
    • Liver ultrasound
    • Liver and chest computerized tomography (CT or CAT) scan
    • Liver magnetic resonance imaging

    Ultimately, the diagnosis is confirmed by a biopsy of the tumor. Hepatocellular carcinoma can spread to parts of the abdomen and the lung.  Therefore, a full work-up at diagnosis is necessary to define disease extent. After all tests are completed, doctors will be able to outline the best treatment options.

    Treatment & Care Options

    Specific treatment for hepatocellular carcinoma will be determined by your child's physician based on some or all of the following factors:

    • Your child's age, overall health, and medical history
    • Extent of the disease
    • Your child's tolerance for specific medications, procedures, and therapies
    • How your child's physician expects the disease to progress
    • Your opinion or preference

    Hepatocellular carcinoma is often less responsive to chemotherapy than hepatoblastoma, the most common liver tumor in children. Treatment for hepatocelluar carcinoma therefore relies less on chemotherapy and is generally aimed more towards resecting (removing) as much of the tumor as possible, while making sure your child still has adequate liver function.

    Surgery may be more difficult for patients with hepatocellular carcinoma, as this type of cancer has often spread by the time it is diagnosed, can involve multiple parts of the liver and can occur in patients with underlying liver disease. For this reason, ongoing research and experimental therapeutics are of utmost importance.


    In some instances, children diagnosed with hepatocellular carcinoma require treatment with chemotherapy. Chemotherapy works by interfering with the cancer cells’ ability to grow or reproduce, and while it can be quite effective in treating certain cancers, the agents do not differentiate normal healthy cells from cancer cells. Because of this, there can be many adverse side effects during treatment.

    Being able to anticipate these side effects can help the care team, parents, and child prepare, and, in some cases, prevent, these symptoms from occurring. Dana-Farber/Boston Children’s is fully-equipped to treat symptoms related to chemotherapy. Each child is assigned a comprehensive team of providers, which includes a primary attending physician, a primary fellow or trainee, a primary nurse, and a psychosocial provider.

    For patients with a new diagnosis of hepatocellular carcinoma, we offer enrollment in the most current collaborative Children’s Oncology Group consortium trial. Two of our physicians – Allison O’Neill, MD and Christopher Weldon, MD, PhD – were actively involved in developing the most recent international liver tumor trial entitled PHITT: Pediatric Hepatic Malignancy International Therapeutic Trial. This trial will study approaches to reducing treatment intensity for patients with low-risk disease and will also collect and analyze tumor samples from enrolled patients with the hopes of identifying unique therapies.

    For patients with recurrent hepatocellular carcinoma unresponsive to chemotherapy, we have expertise in the use of:

    • antiangiogenic drugs that interfere with the formation of blood vessels
    • targeted agents that interrupt genetic pathways important for tumor growth
    • immunotherapies that capitalize on the patient’s own immune system

    Our use of interventional techniques to deliver drugs directly to the tumor, and experimental therapeutics to provide alternative and novel therapies for advanced disease, set us apart from other programs.


    In addition to medical therapies, surgery is a necessary component of therapy for all liver tumors. To remove a tumor, surgery may involve removing a portion of the liver (partial hepatectomy), or in rare cases, the entire liver (total hepatectomy) replacing it with a liver from an organ donor (liver transplant). On even rarer occasions, multiple organs may require transplantation (multivisceral transplant).

    Surgery involving the liver is extraordinarily complex and requires a surgeon well-versed in this specialized skill. Because liver tumors in children are rare, few surgeons see enough cases to develop extensive expertise. At Dana-Farber/Boston Children’s, our experts specialize in pediatric liver surgery and have successfully performed some of the most complicated liver operations in children. In seamless collaboration with Boston Children’s, the nation’s top-ranked pediatric hospital, we have built a multidisciplinary team for each patient that draws from our oncology, pediatric gastroenterology, interventional radiology, and surgery divisions.

    Patients around the world choose us for their care. Within the past 15 years, our surgeons have performed nearly 50 liver tumor surgeries, a large number given the rarity of these diseases. Since 2001, we have performed 25 liver transplants and 3 multivisceral (multiple organs) transplants for patients with liver tumors. Our advanced capabilities include:

    • autotransplantation - removing the liver to allow dissection of an advanced tumor from the organ, while replacing the remaining liver following removal of the cancer
    • blood vessel or bile-duct reconstruction
    • heart-lung bypass to remove a tumor that has grown into the heart

    Very few sites can offer conventional surgery, transplant, and the ancillary staff necessary to care for unique complex cases.

    Interventional Radiology

    Interventional radiology refers to techniques that allow delivery of therapy directly to the site of disease. In the case of liver tumors, interventional procedures can involve:

    • radiofrequency ablation - destruction of tumors through direct delivery of heat
    • transarterial embolization - disruption of the blood supply to the tumor
    • transarterial chemoembolization - direct delivery of chemotherapy to the tumor
    • Yttrium-90 - direct delivery of a radioactive compound to the tumor

    These measures can prevent tumors from growing and/or shrink tumors as a bridge to surgery. While these procedures have been available to adult patients for years, they have yet to be routinely utilized for pediatric liver tumor patients. We are one of the few pediatric sites in the United States able to offer these specialized therapeutic approaches to our patients.


    We work in concert with a comprehensive radiation therapy oncology program paired with Brigham and Women’s Hospital.


    We offer remote consultations through online second-opinion platforms such as Grand Rounds and MOREHealth, thereby reaching patients and families at every corner of the globe.


    Our clinical research program offers unique access to clinical trials in which children can receive the most up-to-date liver cancer treatments. A major focus of our research program is the rapid translation of scientific discoveries from the laboratory to the bedside to benefit patients. This includes discoveries from our pediatric laboratories along with close collaboration with our adult colleagues and those in the pharmaceutical industry. Allison O’Neill, MD, Medical Director of the Liver Tumor Program, has a particular interest in the treatment of hepatocellular carcinoma and has dedicated her clinical and research efforts to better understanding this disease.

    Through this research, our physicians work to improve therapeutic approaches and continually improve outcomes. We have a prospective liver tumor registry which allows us to collect important clinical information from our patients, assisting in the care of patients we will treat years down the road. We offer genomic tumor profiling for every patient we see, allowing us to study relevant genetic pathways in large cohorts of children with these rare diseases. We hope to offer this option remotely for patients, going forward.

    Our new molecular pathology laboratory will allow the rapid identification of rare liver tumors at the time of diagnosis while working to discover new genetic patterns useful for diagnosis and treatment in the future. Laboratory researchers are investigating mechanisms by which to grow liver tumor cells in culture dishes as well as in mouse and zebrafish models. This will allow novel drug testing that can inform additional therapies for hard-to-treat tumors.

    Clinical Trials

    For many children with rare or hard-to-treat conditions, clinical trials provide new options.

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