• 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

    Treatment for hepatoblastoma is generally aimed at resecting (removing) as much of the tumor as possible while making sure your child still has adequate liver function. Liver tissue can regenerate when removed.

    Surgery

    Complete removal of the tumor through surgery (liver resection) remains very important to achieving a cure in children with hepatoblastoma. If it is deemed that removal would be extremely risky or have a significant chance of leaving tumor behind, then the child may be treated initially with chemotherapy.

    After three or four courses of treatment, resection is often much more readily accomplished. The liver has a remarkable capacity to regenerate. Long-term follow-up studies of children have shown that the liver can continue to grow and often remains very close to what would be the predicted normal size of the liver despite removal of up to 2/3 of the liver in infancy.

    Any tumor in the upper gastrointestinal tract, including the liver, is a very complicated surgery and requires very specialized expertise. At Dana-Farber/Boston Children's, we have surgeons who specialize in pediatric liver surgery and have successfully performed some of the most complicated liver and upper gastrointestinal surgeries in children.

    Chemotherapy

    As mentioned above, chemotherapy is a treatment used in conjunction with surgery and other treatments. Chemotherapy is a drug treatment that works by interfering with the cancer cell's ability to grow or reproduce.

    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, if possible.

    • 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.
    • Certain chemotherapy drugs may be given in a specific order depending on the type of cancer it is being used to treat.

    Chemotherapy can be given:

    • as a pill to swallow
    • as an injection into the muscle or fat tissue
    • intravenously (directly to the bloodstream)
    • intrathecally (directly into the spinal column with a needle)

    Liver transplant

    In very rare cases, when tumors are still inoperable after chemotherapy, doctors may consider a liver transplant. Your child's physician will provide more information on whether this is an option for your child and the process involved.

    What is the long-term outlook for patients with hepatoblastoma?

    There has been a dramatic improvement in outcomes for hepatoblastoma over the past 20 years. Complete surgical removal at diagnosis, with or without chemotherapy, can allow for a survival rate of almost 100%. The survival rate drops to approximately 70% for patients requiring chemotherapy upfront to assist with surgical excision. Patients with metastatic disease have an approximately 20 or 30% survival rate.

    There is preliminary evidence that the cure rate for children with advanced stages of the disease may be improving due to aggressive chemotherapy options and the possibility of a liver transplant. Your child's prognosis greatly depends on:

    • The extent of the disease
    • The size and location of the tumor
    • Presence or absence of metastasis
    • The tumor's response to therapy
    • The age and overall health of your child
    • Your child's tolerance of specific medications, procedures, or therapies

    As with any cancer, prognosis and long-term survival can vary greatly from child to child. Prompt medical attention and aggressive therapy are important for the best prognosis.

    What is the recommended long-term care for children treated for hepatoblastoma?

    Your child should visit a cancer survivorship clinic every year to:

    • manage disease complications
    • screen for early recurrence of cancer
    • manage late effects of treatment

    A typical follow-up visit may include some or all of the following:

    • a physical exam
    • laboratory testing
    • imaging scans

    Through the David B. Perini, Jr. Quality of Life Clinic, childhood cancer survivors receive a comprehensive follow-up evaluation from their cancer care team.

    What is the latest research on hepatoblastoma?

    Dana-Farber/Boston Children's researchers are conducting numerous studies that will help clinicians better understand and treat hepatoblastoma. We participate in the current treatment protocols of the national Children's Oncology Group.

    Other specific types of treatment currently being studied include:

    • Angiogenesis inhibitors – Substances that may be able to prevent the growth of tumors by blocking the formation of new blood vessels that feed the tumors
    • Immunotherapy – A wide range of substances that may be able to involve the body's own immune system to fight cancer or lessen harmful side effects of some treatments

    Treating hepatitis in children

    As discussed above, children who are exposed to hepatitis B infection at an early age are at increased risk for developing hepatoblastoma. Maureen M. Jonas, MD, medical director of the Boston Children's Liver Transplant Program, has led research focused on liver disease in children with emphasis on viral hepatitis.

    • She was the principal investigator in several international trials where drugs that are effective in treating adults with chronic hepatitis B were tested in children.
    • Dr. Jonas's work has been published in the New England Journal of Medicine, Hepatology, Pediatric Infectious Disease Journal and other major medical journals.

    Clinical trials

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

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