What is a Pancreatoblastoma?
Pancreatoblastoma (PBL) is a very rare cancer which originates from the early cells that line the glands in the pancreas. PBL may develop in any part of the pancreas, but usually it is located in the head or the main part called the body. When PBL grows, it can infiltrate the neighboring structures. Even though it is a very rare tumor, it remains the most common pancreatic tumor in children.
Who gets Pancreatoblastoma and why?
This tumor usually occurs in children under 10 years of age, with a peak incidence of 5 years of age. The cause is unknown but some children have a genetic disease called Beckwith-Wiedemann syndrome. PBL is rarely associated with familial adenomatous polyposis (FAP).
Is there a risk for brothers / sisters to develop the same cancer or other types of cancer?
In families with specific genetic diseases mentioned above there is an increased risk of PBL and some other rare tumors. Patients with Beckwith-Wiedemann syndrome are predisposed to the Wilms’ tumor (a cancer of the kidney) and hepatoblastoma (a cancer of the liver), while patients with FAP – to the colon cancer. Genetic investigations and counseling are recommended in children with PBL and genetic syndromes. In these patients some other locations, such as the kidneys, liver and intestines should be checked on a regular basis.
What are the typical symptoms and signs of Pancreatoblastoma?
Children with PBL may present with abdominal pain, vomiting, weight loss, jaundice (yellow color of the skin) and bleeding from intestines. In some patients, a big, firm mass may be felt in the upper abdomen. These symptoms are seen also in other childhood diseases, so in many cases the initial diagnosis of PBL is made late.
Which investigations are necessary for a child with Pancreatoblastoma?
Most commonly, the first diagnostic examination which detects PBL is an ultrasound (US) scan of abdomen. However, your child may require further tests to check exactly the size and location of the cancer and whether it has spread to other parts of the body. These tests may include:
- MRI (magnetic resonance imaging) – this method uses magnetism to build up a very detailed picture of the body.
- CT (computerized tomography) scan – this method uses x-rays to build up a three-dimensional picture the body.
Because 70% of PBL produce a protein called alpha-fetoprotein (AFP), an elevated level of AFP may be found in the blood of a child with PBL. That is why AFP may be a marker of this cancer at diagnosis and used for monitoring of the treatment’s effects (it should lower along with chemotherapy or after tumor resection). Also lactate dehydrogenase (LDH) level in the blood may be elevated – especially when the tumor has spread to the liver (this is called liver metastases).
Are there different stages of the disease?
The staging of PBL is based on the tumor size (T), the regional lymph nodes involvement (N) and the presence of distant spread (metastases or M). Also the completeness of surgical tumor removal is an important factor in staging of PBL. PBL is usually diagnosed in advanced stages – when the tumor of pancreas is large, spreads to surrounding organs and/or distant sites. The most common sites of metastases are the liver, the lungs and the regional lymph nodes.
What about the Pancreatoblastoma treatment?
In children with PPB it is very important to remove the tumor completely. Sometimes, the complete surgical removal of the tumor may be performed at diagnosis. When the disease is initially difficult to remove or when distant spread is present, a biopsy of the tumor is usually performed. Surgery may be done later – after chemotherapy has been given to shrink the tumor size.
PBL is sensitive to chemotherapy and it seems to improve the chances of long-term cure. The type of chemotherapy has not been defined completely so far, but drugs called doxorubicin and cisplatin are the most frequently used. Most children receive chemotherapy, especially when the tumor resection is not feasible at diagnosis. The number of chemotherapy cycles varies, and depends on the stage and completeness of surgery.
The role of radiotherapy remains controversial and the experts in multidisciplinary meetings will usually discuss if it can be useful for your child.
What are the results of treatment?
The most important factors that determine a good outcome in PBL is the absence of metastases (spread) and a complete removal of the tumor with surgery.
What research is carried out for Pancreatoblastoma?
PBL is a very rare tumor which makes research difficult. Nevertheless, there are groups of experts in the USA and Europe which promote clinical and scientific research on PBL. Specific genetic alterations have been found that allow us to identify patients and families at risk of developing PBL and other rare tumors.
What EXPeRT is doing for children with Pancreatoblastoma?
The EXPeRT group is working for children with PPB in many ways:
- Collecting data of children with PBL from all European countries
- Creating guidelines for diagnosis and treatment of PBL with the goal to optimize the chance of cure for all children
- Providing advice in difficult cases to the leading clinician, both at a European level and worldwide
- Collaborating with other Groups of experts and researchers to increase the knowledge on PBL.