Hepatoblastoma is a rare liver cancer that primarily affects infants and young children, typically under age three. This cancer originates in liver cells and disrupts the organ’s normal functions, affecting only one to two children per million in the United States. Because the tumors grow slowly, symptoms may not appear until the mass becomes large. Early detection and specialized medical care are important for managing the disease.
Causes and Associated Symptoms
The precise cause of most hepatoblastoma cases is unknown. However, researchers have identified certain genetic conditions and circumstances that increase a child’s risk. Premature birth with a very low birth weight is one of the more common associated factors. Specific genetic syndromes, such as Beckwith-Wiedemann syndrome, a growth disorder, and familial adenomatous polyposis (FAP), an inherited condition causing polyps in the large intestine, are also linked to a higher incidence of hepatoblastoma.
The most common initial sign is a painless mass or swelling in the abdomen that a parent or doctor may feel. As the tumor grows, a child might experience abdominal pain, a feeling of fullness, or a loss of appetite leading to weight loss. Other symptoms can include nausea, vomiting, and jaundice, which is a yellowing of the skin and eyes. In some cases, fatigue, fever, and itchy skin may also be present.
The Diagnostic Process
A diagnosis begins when a physician suspects a liver issue based on a physical exam and reported symptoms. Blood tests are then performed, primarily to measure the level of alpha-fetoprotein (AFP). This protein is often produced in high amounts by hepatoblastoma tumors, making elevated AFP levels a strong indicator of the disease. These blood tests also help assess the overall function of the child’s liver.
Following blood tests, imaging studies visualize the abdomen and liver. An ultrasound is frequently the first imaging test performed because it is non-invasive. To get a more detailed view, doctors will order a computed tomography (CT) scan or a magnetic resonance imaging (MRI) scan. These detailed images help determine the tumor’s size, location, and if the cancer has spread beyond the liver.
A definitive diagnosis requires a biopsy. This procedure involves taking a small sample of the tumor tissue for examination under a microscope by a pathologist. A biopsy is performed under general anesthesia to ensure the child feels no pain. The examination confirms the cancer type and provides information needed for planning treatment.
Treatment Approaches
Once a diagnosis is confirmed, treatment is based on the tumor’s stage. Staging classifies the extent of the disease, and for hepatoblastoma, doctors often use the PRETEXT system. This system categorizes the tumor based on how many liver sections are involved. This staging helps determine the most effective course of action.
The primary treatment is the complete surgical removal (resection) of the tumor. Whether surgery is immediately possible depends on the tumor’s size and location. For children with small, localized tumors (PRETEXT stage I or II), immediate resection may be performed. A successful surgery is a significant factor influencing the long-term outcome.
Chemotherapy is a standard component of treatment. It is often administered before surgery (neoadjuvant chemotherapy) to shrink the tumor and make it easier to remove. After surgery, additional chemotherapy (adjuvant chemotherapy) may be given to destroy any remaining cancer cells and reduce the risk of recurrence.
If a tumor is too large to be removed with a standard resection, a liver transplant may be recommended. This procedure involves removing the entire liver and replacing it with a healthy donor liver. A transplant can offer a chance for a cure, even when the tumor is considered unresectable by other surgical means.
Prognosis and Long-Term Outlook
The prognosis for hepatoblastoma has improved significantly, with survival rates now reaching 80-90% for many patients. The outcome depends on the cancer’s stage, whether the tumor can be completely removed, and its response to chemotherapy. For children with localized tumors that are successfully resected, the survival rate is greater than 85%.
Patients with stage I and II disease treated with surgery and chemotherapy have a cure rate of over 90%. Even when cancer has spread to other parts of the body, such as the lungs, effective treatment can still lead to positive outcomes.
After treatment is completed, long-term follow-up care is necessary. This monitoring is designed to watch for potential late effects of treatment, which can surface months or years later. Regular check-ups help manage health issues and ensure the child’s continued growth. Recurrences are most common within the first two years after treatment, making this a period of careful observation.