Hepatoblastoma treatment & prognosis — understanding liver cancer in children

Hepatoblastoma is a cancer that starts in the liver and arises from immature liver cells called hepatoblasts — cells that form the liver during fetal development. It is the most common liver cancer in children and almost always occurs in children under five years of age, with the highest incidence in infants and toddlers under three years. Most cases arise without an identifiable cause or family history. A small number of children with hepatoblastoma have certain associated conditions — such as Beckwith-Wiedemann syndrome (an overgrowth condition) or familial adenomatous polyposis — and in these children, liver surveillance may have already been planned. Premature infants with very low birth weight have a modestly higher risk compared to the general population, though the reason for this is not fully understood. As with all paediatric liver tumours, the condition is not caused by anything a parent did during pregnancy.

The most common early sign of hepatoblastoma is a firm, painless swelling or lump in the upper right part of the abdomen, often noticed by a parent while bathing or dressing the child. Because the liver is a large organ on the right side of the abdomen, a growing tumour can become quite large before causing discomfort. The abdomen may appear visibly distended. Other symptoms that sometimes occur include unexplained weight loss or poor appetite, a general sense of feeling unwell, persistent nausea or vomiting, and occasionally pallor or anaemia. Jaundice (yellowing of the skin or eyes) is uncommon at presentation but can occur. A raised level of a blood protein called alpha-fetoprotein (AFP) — detected on a blood test — is a key diagnostic marker for hepatoblastoma. If your child has any of these symptoms, a doctor should assess them promptly.

When a liver tumour is suspected, the first investigation is an abdominal ultrasound scan, which shows the size, location, and character of the liver mass. This is followed by a CT scan or MRI of the abdomen and chest to map the tumour in detail, assess which segments of the liver are involved, and look for spread to the lungs or other organs. A blood test measuring alpha-fetoprotein (AFP) is essential — AFP is produced by hepatoblastoma cells and a significantly elevated level strongly supports the diagnosis. A biopsy (taking a small tissue sample from the tumour) confirms the diagnosis and is used to examine the tumour’s histological type. All these results are reviewed together by a multidisciplinary paediatric tumour board — a hepatologist, paediatric surgeon, medical oncologist, radiologist, and pathologist — before a treatment plan is discussed with the family.

Staging in hepatoblastoma describes how far the cancer has spread and how much of the liver is involved. The most widely used international staging system for hepatoblastoma (PRETEXT, used by the International Childhood Liver Tumours Strategy Group, SIOPEL) divides the liver into four sections and records how many sections contain tumour, whether the tumour involves key blood vessels or bile ducts, and whether it has spread beyond the liver. PRETEXT stage I means tumour in one section only; PRETEXT IV means all four liver sections are involved. Staging determines whether the tumour can be removed with surgery straight away, or whether pre-operative chemotherapy is needed first to shrink it enough for safe surgical removal. In some very advanced or multi-focal cases, a liver transplant may be considered. Staging is therefore directly linked to the sequence and type of treatment your child will receive.

Hepatoblastoma treatment combines surgery and chemotherapy, and the sequence depends on staging. For tumours that can be removed straight away, surgery (partial liver resection — removing the part of the liver containing the tumour) is performed first, followed by chemotherapy to eliminate any remaining cancer cells. For tumours that are too large or too centrally located to remove safely at diagnosis, pre-operative chemotherapy is given first to shrink the tumour; this allows safer surgical removal a few weeks later. The liver’s remarkable ability to regenerate means that even after removing a substantial portion, it can grow back close to its original size over time. In cases where chemotherapy cannot shrink the tumour enough for partial resection, liver transplantation may be considered at specialist centres. AFP levels in the blood are tracked throughout treatment as a marker of response — a falling AFP level is a reassuring sign that the treatment is working. Every child’s plan is designed and reviewed by a multidisciplinary tumour board.

After completing treatment, children are followed closely with regular imaging (ultrasound, CT, or MRI) and blood tests including AFP to monitor for any signs of recurrence. The follow-up schedule is most intensive in the first two years and gradually becomes less frequent over time. Children who had a partial liver resection need monitoring of liver function and overall growth. Those who received chemotherapy may need cardiac function monitoring depending on the agents used. Long-term, many children go on to live healthy, active lives after successful hepatoblastoma treatment. At CION, we walk this journey with your family — not just through treatment, but through the follow-up phase and beyond, so that your child’s recovery is supported at every step. We will plan the follow-up programme clearly, so nothing falls through the cracks.