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Treatments for neuroblastoma

If a child has neuroblastoma, the healthcare team will include specialists from different areas of medicine. All of these specialists will have experience treating children with neuroblastoma. They will work together to create a treatment plan just for the child. It will be based on your child’s needs and may include a combination of different treatments.

When deciding which treatments to offer for neuroblastoma, your child’s healthcare team will consider the:

  • child’s age
  • stage of cancer
  • risk group
  • child’s overall health

Doctors will closely monitor the child’s response to treatment to decide if treatment should continue, if treatment can be stopped or if they should try other treatments.

Treatment options for neuroblastoma

The following are treatment options for neuroblastoma.


Surgery is the main treatment for localized neuroblastoma (has not spread, or metastasized). It is used to diagnose and stage neuroblastoma. It is also used to completely remove the tumour, if possible.

Chemotherapy may be given after surgery, and sometimes surgery is done again after chemotherapy. This is called second-look surgery. It is used to remove any disease that remains and to see how the cancer is responding to treatment.


Chemotherapy is usually offered for neuroblastoma. It usually includes different combinations of drugs.

Chemotherapy may be used:

  • as the main treatment, with or without surgery (apart from a biopsy which is usually required to diagnose neuroblastoma)
  • after surgery to prevent the cancer from coming back, or recurring (called adjuvant therapy)
  • to shrink the tumour before surgery so it is easier to remove without harming nearby organs, tissues and vessels
  • to treat advanced tumours that can’t be completely removed with surgery

Radiation therapy

Radiation therapy is usually only used to treat high-risk neuroblastoma that is likely to recur. It is also used to quickly shrink tumours in the chest, liver or central nervous system (CNS) that are causing life-threatening symptoms.

Radiation therapy is sometimes used to relieve symptoms of advanced cancer (called palliative radiation therapy).

Stem cell transplant

A stem cell transplant is sometimes used to treat neuroblastoma. It is often used to treat high-risk neuroblastoma or neuroblastoma that recurs, along with high-dose chemotherapy. Other treatments may be used along with stem cell transplant.

Retinoids and immunotherapy

Retinoids and immunotherapy may be offered for high-risk neuroblastoma or neuroblastoma that recurs. Different types of immunotherapy may be used, including interleukins, granulocyte growth factors or monoclonal antibodies. These treatments are usually given in combination at the same time or in sequence. Other treatments may be used along with retinoids or immunotherapy.

Watchful waiting

Watchful waiting may be a treatment option for some neuroblastomas that don’t need to be treated right away. It is sometimes used for babies younger than 12 months who have stage 4S neuroblastoma with favourable prognostic factors and no symptoms.

Follow-up care

Follow-up after treatment is an important part of cancer care. Your child will need to have regular follow-up visits, especially in the first 2 years after treatment has finished. These visits allow the healthcare team to monitor your child’s progress and recovery from treatment and to monitor for late side effects from the treatments received.

Response to treatment

Knowing how well neuroblastoma responds to treatment helps doctors decide whether or not treatment can be stopped or more treatment is needed. Response to treatment is monitored using imaging tests and urine tests. Neuroblastoma cells produce chemical substances called catecholamines. Catecholamines are broken down into homovanillic acid (HVA) and vanillylmandelic acid (VMA). Urine tests can check levels of these acids in the urine to measure response to treatment. CT and MRI are used for measuring the primary tumour, and MIBG and PET are used for measuring bone and marrow metastases. Doctors use the following criteria to measure the response to treatment.

Complete response means that the primary tumour is gone and there are no metastases. Vanillylmandelic acid (VMA) and homovanillic acid (HVA) levels in the urine are normal.

Very good partial response means that the primary tumour is over 90% smaller than before treatment and there is no evidence of metastasis. Vanillylmandelic acid (VMA) and homovanillic acid (HVA) levels in the urine are normal. Some minor changes may be seen in a bone scan but there are no new lesions and all pre-existing lesions are improved.

Partial response means that all tumours are over 50% smaller than before treatment. There are no new metastases. Over 50% of the sites where cancer had spread to the bone are gone.  Only one bone marrow sample shows remaining tumour cells and the HVA or VMA levels in the urine are 50%–90% lower than before treatment.

Mixed response means that there are no new metastases. Some tumours are less than half the size they were before treatment. Other tumours have shrunk but not as much. Any single tumour has grown by less than 25%.

No response, or stable disease, means that there are no new tumours. Some tumours have shrunk, but by less than 50% of their original size. Any other tumour has grown by less than 25%.

Progressive disease means that there is a new tumour, any tumour has grown by over 25% since treatment started or there are cancer cells in the bone marrow that were not there before treatment.

Clinical trials

Many children with cancer are treated in a clinical trial. Clinical trials look at new and better ways to prevent, find and treat cancer. Find out more about clinical trials.

Questions to ask about treatment

Find out more about treatment. To make the decisions that are right for your child, ask the healthcare team questions about treatment.


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