• Hepatoblastoma

    Hepatoblastoma is a rare type of childhood cancer that occurs in the liver. Children who have had hepatitis B infection at an early age, those born prematurely and of low birth weight, or those who have had biliary atresia (blockage of the tubes that carry bile-20) are at increased risk of developing hepatoblastoma. A number of genetic conditions also increase the risk for this disease.

    The primary functions of the liver include filtering and storing blood, processing the food we eat and making vital proteins for the body to use. It has a right lobe and a left lobe.

    • Most hepatoblastoma tumors grow in the right lobe.
    • This disease primarily affects children from infancy to about five years of age; most cases appear during the first 18 months of life.
    • Hepatoblastoma cancer cells can spread (metastasize) to other areas of the body.
    • The most common sites of metastasis are the lungs and the abdomen.

    Hepatoblastoma Treatment at Dana-Farber/Boston Children's

    Patients with hepatoblastoma are treated at Dana-Farber/Boston Children's through our Liver Tumor Program. We have some of the most experienced pediatric liver cancer oncologists and surgeons, as well as internationally recognized pediatric subspecialists who use sophisticated technology and therapies to maximize outcomes for our patients.

    We also have one of the largest, most sophisticated pathology services of its kind in the country. Because hepatocellular carcinoma (another type of liver tumor) is sometimes misdiagnosed as hepatoblastoma, it is important to have liver tumors reviewed by pediatric pathologists who specialize in rare pediatric tumors and can make a correct diagnosis.

    What causes hepatoblastoma?

    Although the exact cause of liver cancer is unknown, there are a number of genetic conditions that are associated with an increased risk for developing hepatoblastoma, including the following:

    • Beckwith-Wiedemann syndrome
    • Hemihypertrophy
    • Familial adenomatous polyposis
    • Tyrosinemia
    • Glycogen storage disease type I
    • Galactosemia
    • Alpha-1 antitrypsin deficiency

    Children who have a hepatitis B infection at an early age, or those who have biliary atresia (a blockage in the tubes that carry bile), are also at increased risk for developing hepatoblastoma.

    What are the symptoms of hepatoblastoma?

    Each child may experience symptoms differently, and symptoms may vary depending on the size of the tumor and the presence and whether the tumor has spread.

    Your child's symptoms may include:

    • A large abdominal mass, or swollen abdomen
    • Weight loss, decreased appetite
    • Abdominal pain
    • Vomiting
    • Jaundice (yellowing of the eyes and skin)
    • Fever
    • Itching skin
    • Anemia (pale skin and lips from decreased number of red blood cells)
    • Back pain from compression of the tumor

    The symptoms of hepatoblastoma may resemble other conditions or medical problems. Always consult your child's physician for a diagnosis.

    How is hepatoblastoma diagnosed?

    Ultimately, the diagnosis is confirmed by a biopsy of the tumor. Hepatoblastoma can spread to the lungs, and rarely to the bone. 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.

    In addition to a complete medical history and physical examination, diagnostic procedures for hepatoblastoma may include:

    • Biopsy – A sample of tissue removed from the tumor and examined under a microscope; your child's surgeon may also look at the liver using an instrument called a laparoascope, a small tube with a light on the end.
    • Complete blood count (CBC) – A measurement of size, number, and maturity of different blood cells in a specific volume of your child's blood.
    • Additional blood tests – Blood chemistries, evaluation of liver and kidney functions, and genetic studies.
    • Alpha-fetoprotein (AFP) test – Alpha-fetoprotein (AFP) levels in the blood can be used to diagnose and follow your child's response to treatment.
    • Multiple imaging studies, including:
      • X-ray – A diagnostic test which uses invisible electromagnetic energy beams to produce images of internal tissues, bones, and organs on film.
      • Computerized tomography scan (also called a CT or CAT scan) – A CT scan shows detailed images of any part of the body, including the bones, muscles, fat, and organs. CT scans are more detailed than general x-rays.
      • Magnetic resonance imaging (MRI) – A diagnostic procedure that uses a combination of large magnets, radiofrequencies, and a computer to produce detailed images of organs and structures within the body.
      • Ultrasound (also called sonography) – Ultrasounds are used to view internal organs as they function, and to assess blood flow through various vessels.
      • Liver scans – Pictures or x-rays taken after a dye has been injected that is absorbed by liver tissue. These are used to detect tumors and liver abnormalities.

    After we complete all necessary tests, our experts meet to review and discuss what they have learned about your child's condition. Then we will meet with you and your family to discuss the results and outline the best possible treatment options.

    How is hepatoblastoma classified?

    Staging is the process of determining whether cancer has spread and, if so, how far. There are various staging symptoms that are used for hepatoblastoma. Always consult your child's physician for information on staging. One method of staging hepatoblastoma is the following:

    • Stage I – Usually a tumor that can be completely removed with surgery.
    • Stage II – Usually a tumor that can mostly be removed by surgery; very small amounts of the cancer are left in the liver.
    • Stage III – Usually a tumor that cannot be completely removed; or cancer cells also have been found in the lymph nodes.
    • Stage IV – Cancer that has spread to other parts of the body.
    • Recurrent – The disease has returned after it has been treated; it may come back in the liver or in another part of the body.

    What are the treatments for hepatoblastoma?

    Specific treatment for hepatoblastoma 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

    Hepatoblastoma is often quite responsive to chemotherapy, therefore treatment generally involves both chemotherapy and surgical resection of the tumor.


    Most children diagnosed with hepatoblastoma require treatment with chemotherapy. Chemotherapy works by interfering with the cancer cells’ ability to grow or reproduce.

    • Chemotherapy is introduced to the bloodstream and then travels throughout the body to kill cancer cells.
    • Different groups of chemotherapy drugs work in different ways to fight cancer cells and shrink tumors.
    • Often, a combination of chemotherapy drugs is used.

    While chemotherapy 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 hepatoblastoma, 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 increasing treatment intensity for those with more aggressive disease. The trial will also collect and analyze tumor samples from enrolled patients with the hopes of identifying unique therapies.

    For patients with recurrent hepatoblastoma 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 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 more rare 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.

    What is the latest research on hepatoblastoma?

    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.

    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 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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