What is a Pancreatoblastoma?
Pancreatoblastoma (PBL) is a rare cancer which originates from the early cells that line the glands in the pancreas. Pancreatoblastoma may develop in any part of the pancreas, but usually it is located in the head or main part of the pancreas. When it grows, it can infiltrate the neighboring structures. It is the commonest pancreatic tumour 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. Rarely is it associated with familial adenomatous polyposis (FAP).
Is there a risk of brothers / sisters developing 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 (cancer of the kidney) and hepatoblastoma (cancer of the liver) while patients with FAP – to the colon cancer. Genetic investigations and counseling are recommended in cases in which PBL is found in children with 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?
Pancreatoblastomamay present with abdominal pain, vomiting, weight loss, jaundice (yellow colour of the skin) and bleeding from intestines. In some patients a firm mass may be felt in the upper abdomen. These symptoms are seen also in other 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 scan (US). 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) scan –which uses magnetism to build up a very detailed picture of your body
- CT (computerised tomography) scan – which uses x-rays to build up a three-dimensional picture of the inside of your body.
Because 70% of PBL may 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 in monitoring the treatment effects (it should lower along with chemotherapy or after tumour resection). Also lactate dehydrogenase (LDH) level in the blood may be elevated – especially when a child has metastatic foci in the liver.
Are there different stages of the disease?
The staging of PBL is based on the tumor size (T), the involvement of regional lymph nodes (N) and the presence of distant metastases (spread or M). Also the completeness of tumour surgery is an important factor in staging of PBL. PBL is usually diagnosed in advanced stages – when the tumour of pancreas is large, spreads to surrounding organs and/or distant sites (which are called metastases). The most common sites of metastases are: liver, lungs and regional lymph nodes.
What about the Pancreatoblastoma treatment?
Complete surgical remaoval of th PPL is very important. In some patients 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 is usually performed. Surgery may be done later – after chemotherapy has been given to shrink the tumour.
Pancreatoblastoma is sensitive to chemotherapy and it seems to improve the chances of long term cure. The type of chemotherapy so far is not defined completely, but drugs called doxorubicin and cisplatin are the most frequent used. Most children receive chemotherapy, especially when the only surgery at diagnosis is a biopsy. The number of chemotherapy cycles varies, and depends on the stage and how easy surgery has been.
The role of radiotherapy remains controversial and experts in multidisciplinary meetings will usually discuss if there is a role for this in your child’s case.
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 tumour with surgery.
What research is happening for Pancreatoblastoma?
PBL is a very rare tumor which makes research difficult. Nevertheless, there are groups in USA and Europe that promote clinical and basic research on PBL. Specific genetic alterations have been found that allows 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 PBL in many ways:
- Collecting data of children with PBL among all European countries
- Creating guidelines for diagnosis and treatment with the goal to optimize the chance of cure for all children
- Giving advice in difficult cases to the responsible clinician at a European level and worldwide
- Collaborating with other Groups to improve knowledge on PBL