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Turcot Syndrome

Turcot syndrome is a variant of familial adenomatous polyposis (FAP) that has the characteristics of FAP with additional tumours outside of the gastrointestinal tract, most notably colon and brain cancers. Like FAP, it is caused by an alteration in the adenomatous polyposis coli (APC) gene, which is a type of tumour suppressor gene, or in MLH1 and PMS2, which are mismatch repair genes.

Familial cancer syndromes, also known as hereditary cancer syndromes, are rare conditions that cause an increased risk of cancer as the result of inherited genetic mutations in certain cancer-related genes. They can affect both adults and children, however they generally develop in people at a younger age than normal. While familial cancer syndromes are not classified as cancer, they are equally as severe and can be life-threatening as they are associated with the development of various tumours throughout the body. Having a familial cancer syndrome does not guarantee the development of cancer, however the risk of developing cancer is higher than those who do not have a familial cancer syndrome.

Turcot syndrome is generally diagnosed equally among the sexes, and is often diagnosed before the age of 20. However, anyone can develop this disease.

Types of Turcot Syndrome

There are two primary types of Turcot syndrome that are classified by the genetic mutations causing the disease.

Type 1 Turcot Syndrome

Type 1 Turcot syndrome, also known as ‘true’ Turcot syndrome, is caused by mutations in the MLH1 gene or PMS2 gene, which are DNA mismatch repair genes (MMR). MMR genes are responsible for recognising and repairing errors in our DNA sequence that may occur during DNA replication. A mutation in any one of the MMR genes means that an error in the DNA sequence cannot be repaired, and may cause an accumulation of genetic mutations. This type of Turcot syndrome is an autosomal recessive disease, which means that both parents have to contribute a defective gene for you to develop it.

Type 2 Turcot Syndrome

Type 2 Turcot syndrome is caused by a mutation in the APC gene, which is a tumour suppressor gene. Tumour suppressor genes act to inhibit cell growth to prevent overproduction of cells and the development of tumours. It is an autosomal dominant disease, which means that you have a 50% chance of developing the condition if one of your parents has the disease.

Turcot syndrome is most commonly associated with the development of colorectal and brain tumours, however it has also been linked to other types of tumours. Some examples include:

The following tumours may also have an increased incidence in people with Turcot syndrome, however more research is needed to confirm:

Treatment

When cancers are detected, they are staged and graded based on size, metastasis, and how the cancer cells look under the microscope. Staging and grading helps your doctors determine the best treatment for you. However, each patient with Turcot syndrome will present with a unique disease behaviour, with varying tumour locations and symptoms. As such, there is no one treatment method that will work for everyone, and there is no standard staging system for this disease. Instead of staging and grading, your doctor will recommend a treatment plan based on the following factors:

  • Type of tumours present.
  • Whether the tumours are malignant (cancerous) or benign (non-cancerous).
  • Tumour location.
  • Whether or not malignant tumours have metastasised.
  • Your age.
  • General health.
  • Your treatment preferences.

Your doctor may also recommend genetic testing, which analyses your tumour DNA and can help determine which treatment has the greatest chance of success. They will then discuss the most appropriate treatment option for you.

Treatment options for tumours associated with Turcot syndrome may include:

  • Surgery to remove as much of the tumour(s) as possible – these will vary based on tumour type and location.
  • Chemotherapy.
  • Radiation therapy.
  • Clinical trials.
  • Palliative care.

Cancer Screening

Once a diagnosis of Turcot syndrome has been confirmed, implementing a targeted screening plan becomes essential due to the increased risk of developing certain cancers.  The content of this plan will vary from person to person based on the genetic mutation involved, your family’s history of cancer and the types of cancers that may be present. It will also outline the routine tests you should have and how regularly you should have them.

Because of how rare Turcot syndrome is, there hasn’t been any official screening recommendations made for this condition. Instead, your doctor will create a targeted screening program for you based on your individual circumstances.

Screening options for Turcot syndrome may evolve as new technologies are developed and our understanding of the condition grows. It is essential to discuss your individual circumstances with your healthcare team to determine the most appropriate screening plan for you.

Risk factors

Turcot syndrome is caused by mutations in any of the following genes:

  • MLH1 (type 1 Turcot syndrome).
  • PMS2 (type 1 Turcot syndrome).
  • APC (type 2 Turcot syndrome).

The first two genes are known as mismatch repair (MMR) genes, and are known to cause type 1 Turcot syndrome. MMR genes are responsible for recognising and repairing errors in our DNA sequence that may occur during DNA replication. A mutation in any one of the MMR genes means that an error in the DNA sequence cannot be repaired, and may cause an accumulation of genetic mutations. This can predispose a person to cancer.

APC is a tumour suppressor gene, which acts to inhibit cell growth to prevent overproduction of cells and the development of tumours.

Symptoms

The symptoms of Turcot syndrome often vary by the type(s) of tumours present. General symptoms of Turcot syndrome may include:

  • The presence of multiple polyps in the gastrointestinal tract.
  • Café-au-lait spots on the skin.
  • Anaemia.
  • Rectal bleeding.
  • Diarrhoea.
  • Fatigue.
  • Abdominal pain.
  • Neurological symptoms, such as:
    • Headaches.
    • Changes in vision.
    • Seizures.
    • Difficulties with vision, hearing, and/or speaking.
    • Difficulties walking and/or loss of balance.
  • Unexplained loss of appetite/weight loss.
  • Nausea and/or vomiting.

Symptoms related to specific tumours can be found on our knowledgebase.

Not everyone with the symptoms above will have cancer, but see your general practitioner (GP) if you are concerned.

Diagnosis

If your doctor suspects you have tumours associated with Turcot syndrome, they may order some of the following tests to confirm the diagnosis and refer you to a specialist for treatment. The tests required for diagnosis will often vary based on the symptoms present, and where the tumour(s) are suspected to be located.

  • Physical examination.
  • Genetic testing.
  • Neurological examination.
  • Blood tests.
  • Imaging tests, potentially including:
    • MRI (magnetic resonance imaging).
    • CT (computed tomography) scan.
    • PET (positron emission tomography) scan.
    • Ultrasound.
  • Exploratory procedures, potentially including:
    • Colonoscopy.
    • Flexible sigmoidoscopy.
    • Other types of endoscopy as indicated.
  • Biopsy.

References

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