Treatment for neuroblastoma

The treatment your child will receive for neuroblastoma depends on many factors including the stage and risk-group of the cancer, the location of the cancer, and your child’s age.

Because neuroblastoma is a very variable disease, treatment pathways for one child compared to another can look very different. Your doctor will follow the best plan for your child given their individual circumstances.

Treatment by risk group

There is a vast difference between treatments and outcomes for low, intermediate, and high-risk neuroblastoma.

The treatment for low-risk disease is usually minimal- in some cases the tumour can go away on its own, without needing treatment but just close monitoring.

More extensive treatment such as surgery or chemotherapy may be needed for intermediate-risk disease, but this will depend on your child’s particular circumstances.

High-risk neuroblastoma requires particularly intensive therapies which can make your child quite ill, and in some cases lead to life-long side effects. Even within this risk group, there is a lot of variation, so the treatment pathway your child takes may look different to the ‘typical’ pathway, or others that you are aware of. Your treatment team will always be following the best options for your child’s circumstances.

Possible treatments

On this page:

  • Induction chemotherapy
  • Surgery
  • High-dose chemotherapy
  • Stem cell transplant
  • Radiotherapy
  • Immunotherapy

Induction Chemotherapy

What is induction chemotherapy?

The main aim of induction chemotherapy is to reduce the size of the primary tumour to make it easier to remove as much of it as possible during surgery.

Chemotherapy uses drugs to kill cancer cells. It's delivered through a central line - a tube that feeds into one of the large veins in the chest. This will be fitted under general anaesthetic to minimise the distress caused by intravenous injections.

Types of induction chemotherapy

There are different chemotherapy regimens used in the induction phase but the most common in the UK are Rapid COJEC and Modified N7.

    • This is a combination of five agents (cisplatin, vincristine, carboplatin, etoposide, and cyclophosphamide) that is delivered directly into your child’s bloodstream through their central line.

      This therapy is administered in eight cycles, separated by intervals of ten days, and is completed within 70 days of the first treatment.

      In high-risk patients, the use of Rapid COJEC is to give high doses of chemotherapy over a shorter time which may improve survival.

    • Modified N7 uses the drugs cyclophosphamide, doxorubicin, vincristine, cisplatin, and etoposide.

      One cycle lasts three-four days every three weeks, and there are five cycles.

      Your child will also be given a 'growth factor' known as G-CSF as an injection, given daily between chemotherapy cycles to reduce the risk of infection.

Stem cell harvesting

During induction chemotherapy, your child will have some of their 'peripheral haematopoietic stem cells' taken from their blood. These are cells that can develop into any of the different types of blood cells and so are very important for carrying oxygen, fighting infection, and preventing bleeding.

During chemotherapy, these cells can get damaged, and this can limit the amount of therapy your child can have. This is because it takes some time for the blood to recover and for blood counts to improve. Taking some cells while they are healthy (known as stem cell harvest) and storing them during the induction phase means that these cells can be replaced later. This will improve your child’s ability to make new blood cells.

Side effects of chemotherapy

Chemotherapy damages cancer cells that divide rapidly with the aim of destroying them. But other types of cells also divide, like skin, hair, and nail cells. This means the chemotherapy can also kill these normal cells. Damaged normal cells can repair and recover over time.

Short-term side effects

  • Tiredness/weakness
  • Sore mouth
  • Hair loss
  • Feeling sick/being sick
  • Diarrhoea
  • Loss of appetite- in some cases your child may need a nasogastric (NG) tube to provide nutrition directly to the stomach if they are unable to eat.
  • Increased chance of infection

These problems are temporary, and steps can be taken to manage them as best as possible with support from your healthcare provider.

Chemotherapy drugs can be very harsh on the developing cells in children. This means they can also cause long-lasting side effects. You can speak with your healthcare provider to understand more about this, and the impact it may have on your child after they have finished treatment.

    Induction chemotherapy patient information video


    Usually, after induction chemotherapy, surgery will be performed to remove as much of the primary tumour (the tumour that developed first) as possible.

    At this point, the hope is that the tumour will have reduced in size since diagnosis so that the surgery will be as successful as possible.

    The surgeon will remove the tumour, along with some healthy cells around it, with the aim to cause little disruption to surrounding structures and organs.

    The tumour that has been removed is then sent for further testing in the laboratory. This can provide a clearer picture of the genetic makeup of that tumour as well as identifying how much or how little of the tumour is active.

      Surgery patient information video

      High-dose chemotherapy

      Following surgery, a child with high-risk neuroblastoma will have high-dose chemotherapy to clear any remaining cancer cells.

      High-dose chemotherapy is a strong cancer treatment. It uses higher doses of chemotherapy drugs than standard chemotherapy. The goal is to kill any cancer cells left over after induction chemotherapy and surgery. This treatment may increase the chances of a cure or longer-term control of the cancer.

      High-dose chemotherapy is also known a consolidation therapy. The drugs used are busulfan and melphalan.

      Stem cell treatment

      High-dose chemotherapy kills cancer cells throughout the body. This includes in the bone marrow where neuroblastoma cells can hide. This treatment is called ‘myeloablative’ because it destroys bone marrow cells. The term ‘myelo’ means bone marrow, and ‘ablative’ means to destroy.

      The bone marrow is important as it is where new blood cells are made. So, patients need a stem cell transplant. Before treatment, stem cells are collected and saved. After the treatment, the stem cells are given back to the patient. This helps the patient to make new blood cells to replace the ones that were destroyed during the treatment.


      High-dose chemotherapy causes the white blood cell count to drop very low. This means patients are at high risk of getting sick from infection. To keep them safe, they stay in a special hospital room that protects them from germs until they are no longer at risk. While in the hospital, a family member can be with them all the time, and other family members can visit too.

      Side effects of high-dose chemotherapy

      High-dose chemotherapy can be tough on the body and can cause life-threatening problems for four in 100 patients.

      It takes several weeks for blood counts to recover after treatment. During this time the child is at risk of infection and can experience stomatitis (mouth sores) and mucositis (inflammation and ulceration of the gastrointestinal tract). This can cause difficulty in eating, swallowing, and talking. To manage these symptoms, children receive antibiotics, anti-fungal medications, and blood products. They can also be given nutritional support if they have trouble eating.

      Other adverse events include general wellbeing, infection and veno-occlusive disease (VOD).

      Studies continue to assess long term effects.

        High-dose chemotherapy patient information video

        Stem cell transplant

        After high-dose chemotherapy, a stem cell transplant may be used to help your child recover and produce new blood cells.

        Stem cell transplant involves taking stem cells from your child and freezing them. These cells are given back via a drip after high-dose chemotherapy. This helps restore the bone marrow and blood cell production. Since the child’s own cells are used there is no risk of rejection as there could be with donor stem cells.

        The transplanted stem cells begin making new blood cells within a couple of weeks. White blood cells are made first, followed by platelets and red blood cells. Until this happens your child is at risk of infection and stays in a special hospital room in isolation.

        Once this is complete and dependant on the risk group, your child may undergo radiotherapy on the area around their primary tumour site.

          Stem cell harvest patient information video


          Radiotherapy uses high-energy radiation to kill cancer cells. The radiation damages the DNA of the cancer cells, which makes it hard for them to grow and divide.

          Radiotherapy can be used after induction chemotherapy, surgery, and high-dose chemotherapy. The treatment targets the original (primary) tumour and any other areas of the body where the cancer may have spread. The goal is to reduce the risk of the cancer returning in that area.

          The plan for radiotherapy is different for each person. High-risk neuroblastoma is usually treated with daily radiation for three to five weeks. The total dose of radiation may be divided into smaller doses, called fractions, to minimise side effects.

          Radiotherapy is given using a special machine. Your child will not feel anything during treatment. Sometimes, your child may need general anaesthesia as they will need to be very still for the treatment.

          Radiotherapy may cause short-term side effects like skin reactions, nausea or diarrhoea, and tiredness. Depending on the type of radiation being given, there may be precautions that need to be taken when coming into contact with others. You can find out more about this from your healthcare provider.

            Radiotherapy patient information video


            Immunotherapy is a cancer treatment that harnesses the body’s own immune system. It aims to keep your child in remission and prevent the cancer from returning by training the immune system to recognise and destroy cancer cells. It is also called antibody therapy.

            The type of immunotherapy used for high-risk neuroblastoma is given into the bloodstream and lasts for six months.


            Cells have molecules on their surface which can be targeted by immunotherapies. GD2 is a molecule found on the surface of neuroblastoma cells but not on normal cells. In neuroblastoma, GD2 promotes the growth and survival of cancer cells. Anti-GD2 therapy is a type of immunotherapy which targets GD2 on the surface of neuroblastoma cells.

            The standard anti-GD2 therapy used for neuroblastoma is called Dinutuximab beta. Solving Kids’ Cancer UK worked alongside a range of other charities to push for the outcome of National Institute for Health and Care Excellence (NICE) recommending the life-saving drug for NHS use in England and Wales.

            Dinutuximab beta is a type of manmade antibody called a monoclonal antibody. It works by sticking to the surface of neuroblastoma cells and marking them as targets for the patient’s immune cells to destroy.

            Dinutuximab beta treatment typically involves five rounds of therapy, each lasting 35 days. Each round of treatment involves a 24-hour intravenous infusion for 10 days, followed by a 25-day rest period.

            As with many treatments for neuroblastoma, there are side effects which your child may or may not experience.

              Side effects of immunotherapy

              • Pain
              • Fever
              • Nerve problems, such as numbness or tingling in the limbs
              • Low blood pressure
              • Vomiting and diarrhoea
              • Capillary leak syndrome, where proteins leak from small blood vessels in the body causing swelling and low blood pressure

              Your child may experience other side effects, and you can speak with your treatment team about the best way to manage these.

              Retinoic acid

              Alongside anti-GD2 immunotherapy, your child may also be treated with a retinoid called 1,3-cis-retinoic acid (isoretinoin). This can reduce the risk of the cancer coming back after high-dose chemotherapy and stem cell transplant, by helping cancer cells to differentiate into normal cells.

              It is taken as a capsule twice a day for two weeks, followed by a two-week break. Usually, it is recommended to be taken for six months, but your treatment team will advise on the best course of action depending on your child’s circumstances.

              Immunotherapy patient information video

              After treatment

              Your child will have regular appointments with their doctor after they have completed treatment. This is to see how they are recovering, look for any long-lasting effects of treatment, and look for any signs of the neuroblastoma returning.

              Adjusting to life after treatment can be a difficult and challenging process for families. Our Family Support team offers a range of post-treatment support services that can help you at this time. Our Family Support Coordinator, Hayley, shares her experience as a parent in the video below, from our Neuroblastoma Parent Global Symposium.

              Post-treatment support: A parent's perspective

              This information is PIF TICK accredited which means it has been produced with robust guidelines that make sure it is accurate and trustworthy.

              Resource production date: 04/05/2021

              Last Reviewed: 29/03/2023
              Next Review: 15/03/2026

              Read more about the PIF TICK