Hepatoblastoma

What is hepatoblastoma?

Hepatoblastoma is a rare cancer that starts in the liver. It is the most common form of childhood liver cancer, and it almost always develops in very young children, most often before the age of three. The word comes from “hepato” (liver) and “blastoma” (a tumor made of immature cells). The tumor is thought to grow from hepatoblasts, which are early liver cells that normally mature into healthy liver tissue before or shortly after birth. In hepatoblastoma, some of these cells keep growing in an uncontrolled way and form a mass.

Hepatoblastoma is different from the liver cancers seen in adults. Adult liver cancer, called hepatocellular carcinoma, usually develops in a liver that has been damaged over many years by conditions such as hepatitis or cirrhosis. Hepatoblastoma, by contrast, usually appears in an otherwise healthy liver and is considered an embryonal tumor, meaning it is linked to how tissue develops early in life.

Although a cancer diagnosis in a young child is frightening for any family, hepatoblastoma is one of the childhood cancers that often responds well to treatment when it is found and managed by an experienced pediatric cancer team. It is usually cared for by pediatric oncologists (doctors who treat cancer in children), pediatric surgeons, and specialists in liver disease. At Acibadem, this care is coordinated through the pediatrics department together with pediatric oncology and surgery.

Hepatoblastoma symptoms

Hepatoblastoma symptoms can be subtle at first, and many children have no obvious complaints until the tumor has grown large enough to be felt or to press on nearby organs. Because most affected children are toddlers or infants, they cannot describe how they feel, so parents and caregivers often notice physical changes first.

• A lump or swelling in the abdomen, often on the right side where the liver sits, sometimes noticed while bathing or dressing the child
• A belly that looks enlarged or feels firm
• Abdominal pain or discomfort, which may show up as fussiness, crying when the belly is touched, or a change in how the child moves
• Loss of appetite and poor feeding
• Weight loss or failure to gain weight as expected
• Nausea and vomiting
• Tiredness, weakness, or paleness, which can be a sign of anemia (a low red blood cell count)
• Fever without a clear infection
• Yellowing of the skin or eyes (jaundice), which is less common but can occur if the tumor blocks the flow of bile
• Early signs of puberty in a very young child, which is rare and happens when the tumor releases a hormone called beta-hCG

Symptoms may differ depending on how large the tumor is and whether it has spread. A small tumor confined to one part of the liver may cause few or no symptoms and may be found by chance during an examination for another reason. A larger tumor is more likely to cause a visible or palpable mass, pain, and digestive problems. If the cancer has spread beyond the liver, most commonly to the lungs, a child may develop a cough or breathing difficulty, although this is not typical at the time of diagnosis. Back pain can occur if the tumor presses on nerves or the spine.

It is important to remember that many of these signs, such as a swollen belly, poor appetite, or fever, are common in young children and are usually caused by ordinary conditions. Having one or more of these symptoms does not mean a child has hepatoblastoma. However, a firm abdominal mass or steadily enlarging abdomen should always be checked by a doctor.

Causes and risk factors

In most children, doctors cannot identify a specific cause for hepatoblastoma. The tumor is believed to arise from genetic changes in developing liver cells, but these changes usually happen by chance and are not inherited from the parents. Nothing a parent did or did not do during pregnancy is known to cause hepatoblastoma, and there is no evidence that it is caused by infection or by anything in a child’s diet.

Research has identified several factors that are associated with a higher-than-average chance of developing hepatoblastoma. Having a risk factor does not mean a child will develop the cancer; most children with these factors never do.

• Premature birth and very low birth weight. Babies born very early or weighing very little at birth appear to have an increased risk, which is one reason hepatoblastoma is sometimes discussed in the context of neonatal care.
• Beckwith-Wiedemann syndrome. This is an overgrowth condition present from birth that can cause an unusually large body size, a large tongue, and other features. Children with this syndrome are known to have a higher risk of certain childhood tumors, including hepatoblastoma, and are often screened regularly.
• Hemihyperplasia (hemihypertrophy). In this condition, one side of the body grows larger than the other. It shares some of the same tumor risks as Beckwith-Wiedemann syndrome.
• Familial adenomatous polyposis (FAP). This inherited condition, caused by a change in the APC gene, leads to many polyps in the colon and a high risk of bowel cancer in adulthood. Children in families with FAP have an increased risk of hepatoblastoma.
• Certain other genetic conditions, such as trisomy 18, Aicardi syndrome, and glycogen storage disease type I, have also been linked to hepatoblastoma in some children.

Because of these associations, children with a known genetic syndrome may be offered regular screening with blood tests and abdominal ultrasound during early childhood. Your doctor may discuss whether this is appropriate for your child.

Diagnosis

If a doctor suspects a liver tumor, the diagnosis is confirmed through a combination of physical examination, blood tests, imaging, and in many cases a biopsy. The goal is not only to confirm that the mass is hepatoblastoma but also to understand exactly where it is in the liver and whether it has spread, because this guides treatment.

• Physical examination. The doctor gently feels the abdomen for a mass, checks for signs of jaundice or anemia, and asks about symptoms, growth, feeding, and family history.
• Alpha-fetoprotein (AFP) blood test. AFP is a protein normally made by the liver of a developing baby. Its level is naturally high in newborns and falls over the first year of life. In most children with hepatoblastoma, the AFP level is much higher than expected for their age. AFP is also used later to track how the tumor responds to treatment and to watch for recurrence.
• Other blood tests. A complete blood count checks for anemia, and liver function tests assess how well the liver is working.
• Abdominal ultrasound. This painless scan uses sound waves and is often the first imaging test performed. It can show the size and location of a liver mass.
• CT or MRI scan. These detailed scans provide a clearer picture of the tumor, its relationship to blood vessels, and how many sections of the liver are involved. Young children often need sedation to stay still for these scans.
• Chest CT scan. Because hepatoblastoma can spread to the lungs, doctors usually image the chest to check for spread.
• Biopsy. A small sample of the tumor is removed, either with a needle or during surgery, and examined under a microscope by a pathologist. This confirms the diagnosis and identifies the tumor’s subtype, which can influence treatment decisions.

Doctors describe the extent of the tumor using a staging system. One widely used system, called PRETEXT (PRE-Treatment EXTent of disease), divides the liver into four sections and records how many are involved by the tumor before any treatment. It also notes whether the tumor involves major blood vessels, has broken through the liver capsule, or has spread to distant sites. This staging helps the team decide whether the tumor can be removed by surgery right away, whether chemotherapy should be given first to shrink it, or whether a liver transplant may be needed.

Hepatoblastoma treatment options

Hepatoblastoma treatment is planned by a multidisciplinary team and is usually based on the tumor’s stage, its subtype, the AFP level, and the child’s overall health. The two main pillars of treatment are surgery and chemotherapy, and most children receive both. Observation alone is not a standard approach for hepatoblastoma, because the tumor is a cancer that requires active treatment.

Surgery

Removing the entire tumor with surgery is the most important part of treatment and offers the best chance of cure. The operation, called a liver resection or partial hepatectomy, removes the part of the liver containing the tumor. The liver is remarkable in its ability to regrow, and in children the remaining healthy liver typically regenerates in the weeks after surgery. Whether surgery can be done first depends on where the tumor sits and how close it is to major blood vessels.

Chemotherapy

Chemotherapy uses medicines that kill rapidly dividing cancer cells. In hepatoblastoma, a drug called cisplatin is the mainstay, sometimes combined with other agents depending on risk group. Chemotherapy may be given before surgery (neoadjuvant chemotherapy) to shrink a tumor that is too large or too close to vital structures to remove safely, and it is usually also given after surgery to destroy any remaining cancer cells. Chemotherapy is given through a vein, often via a small implanted device called a central line or port, over several cycles.

Chemotherapy has side effects. Cisplatin in particular can affect hearing, so children are monitored with hearing tests during and after treatment. Other possible effects include nausea, hair loss, lowered immunity, and effects on the kidneys. Your child’s team will monitor closely and adjust treatment as needed.

Liver transplant

If the tumor involves so much of the liver that it cannot be removed while leaving enough healthy tissue behind, and if it has not spread outside the liver, a liver transplant may be recommended. In this operation, the entire liver is removed and replaced with a donor liver, often from a living relative. After transplant, the child needs lifelong medicines to prevent rejection and regular follow-up.

Treatment of spread disease

When hepatoblastoma has spread to the lungs, chemotherapy is used to shrink these deposits, and in some cases surgery is performed to remove any that remain. Treatment plans in this situation are individualized.

Supportive care and rehabilitation

Alongside cancer treatment, children receive supportive care to manage pain, nutrition, infections, and emotional well-being. Nutrition support is often needed because appetite and weight can be affected. After surgery, children gradually return to normal activity, and physical therapy may help some children regain strength. Families are also supported by nurses, social workers, and child psychologists, since a young child’s treatment affects the whole household.

Living with hepatoblastoma and outlook

Parents naturally want to know the hepatoblastoma survival rate. It is honest to say that the outlook for children with hepatoblastoma has improved substantially over recent decades, largely because of effective chemotherapy combined with surgery and, where needed, transplant. In many cases, particularly when the tumor is confined to the liver and can be completely removed, children are cured and go on to live full lives. The outlook is more uncertain when the cancer has spread beyond the liver, when the AFP level is unusually low at diagnosis, or when the tumor cannot be fully removed. Even in these situations, treatment can be effective, and doctors will discuss the specific circumstances of your child rather than relying on general figures.

After treatment ends, children need long-term follow-up. This typically includes regular AFP blood tests and imaging to watch for recurrence, which is most likely in the first few years after treatment. Because chemotherapy can have late effects, follow-up also includes hearing checks, kidney function tests, heart monitoring where relevant, and attention to growth and development. Children who have had a liver transplant require ongoing care from a transplant team.

Most children who complete treatment develop normally, attend school, and take part in ordinary activities. Some may live with hearing loss from chemotherapy and benefit from hearing aids and educational support. Emotional recovery matters as much as physical recovery, both for the child and for parents and siblings, and many families find it helpful to connect with support services or other families who have been through a similar experience.

Frequently asked questions

What is the hepatoblastoma survival rate?

There is no single figure that applies to every child, because the outlook depends on the tumor’s stage, whether it can be removed surgically, the AFP level, and how well the tumor responds to chemotherapy. In general, children whose tumors are confined to the liver and completely removed have a good chance of cure, while the outlook is less certain when the cancer has spread. Your child’s oncologist is the best source of information about your child’s specific situation.

What are the first hepatoblastoma symptoms parents usually notice?

The most common first sign is a swollen abdomen or a firm lump on the right side of the belly, often discovered during bathing or dressing. Other early clues may include poor appetite, fussiness, vomiting, or slower weight gain. Because these symptoms overlap with many minor childhood illnesses, a doctor’s examination is needed to determine the cause.

Is hepatoblastoma hereditary?

In most cases, no. Hepatoblastoma usually arises from genetic changes that occur by chance in developing liver cells and are not passed down in families. However, a small proportion of cases occur in children with inherited conditions such as familial adenomatous polyposis or Beckwith-Wiedemann syndrome. If there is a family history of these conditions, your doctor may recommend genetic counseling and screening.

How is hepatoblastoma treatment different from adult liver cancer treatment?

Hepatoblastoma is a different disease from adult liver cancer. It tends to respond well to chemotherapy, which plays a central role in treatment alongside surgery. Adult hepatocellular carcinoma often develops in a damaged liver and is treated with a different set of approaches. Children with hepatoblastoma are treated by pediatric oncology teams using protocols designed specifically for childhood liver cancer.

Can hepatoblastoma come back after treatment?

Recurrence is possible, most often within the first few years after treatment ends, which is why regular follow-up with AFP blood tests and imaging is important. If hepatoblastoma does return, further treatment with chemotherapy, surgery, or in some cases transplant may be considered. Rising AFP levels are often the first sign of recurrence and can appear before symptoms.

Will my child need a liver transplant?

Most children with hepatoblastoma do not need a transplant. It is generally considered when the tumor cannot be safely removed by partial resection, even after chemotherapy, and has not spread outside the liver. Your child’s surgical and transplant teams will assess this based on detailed imaging and the response to treatment.

When to see a doctor

Any young child with a persistent or growing swelling in the abdomen should be examined by a doctor promptly. Early evaluation allows treatment to begin sooner if a liver tumor is found. Seek urgent medical attention if your child has any of the following:

• A firm lump or mass in the abdomen, or a belly that is visibly getting larger
• Severe or worsening abdominal pain, especially with a swollen, hard, or tender belly
• Yellowing of the skin or the whites of the eyes
• Repeated vomiting, refusal to feed, or signs of dehydration such as very few wet diapers
• Unusual paleness, extreme tiredness, or lethargy
• Persistent fever with no clear cause
• Unexplained weight loss or failure to gain weight
• Difficulty breathing or a persistent cough in a child known to have a liver tumor
• Signs of early puberty in an infant or toddler

If your child is already being treated for hepatoblastoma, contact the treating team without delay for fever, unusual bleeding or bruising, severe vomiting, or any sudden change in condition, since chemotherapy can lower the body’s ability to fight infection.