Treatments for neuroblastoma

Some children with low risk neuroblastoma may not need treatment as the cancer may go away by itself. This is called regression. Your child has regular check ups to make sure the cancer is getting smaller (monitoring). This is usually for children up to 18 months of age.

This type of monitoring also depends on other factors including:

• if they are experiencing symptoms and how these are affecting your child

• gene or chromosome changes

• if the cancer is in one area of the body (localised)

Having very close monitoring means your child’s doctor can start treatment early if the cancer is growing, before it makes them unwell.

It can seem very worrying when you first hear that your child won’t be having treatment. But your child’s doctor and nurse will talk you through why this is the best option for your child at the time.

How often your child see’s their doctor for monitoring depends on the age of your child. Generally, they see them every 3-6 weeks for the first few visits. The time between visits then gradually increases as the cancer gets smaller. If they think the cancer is growing, they increase how often they see your child or start treatment depending on their findings.

As part of the check up your child will usually have a:

• physical examination by their doctor

• scan - this might be an MRI scan, CT scan or ultrasound scan

• pee (urine) sample to check for substances called catecholamines. Neuroblastoma cells make these, and the body gets rid of them in urine

Your child’s surgeon tries to remove the whole cancer, or as much of it as possible. The exact type of surgery depends on where the cancer is in the body.

When your child might have surgery

Most children with neuroblastoma have surgery at some point during their treatment. Surgery might be the only treatment your child needs if the cancer hasn't spread and it's possible to remove it all.

Your child might have chemotherapy first to shrink the cancer to a smaller size. This is neo adjuvant chemotherapy. The aim of this is to make the operation easier and safer. Your child stays in hospital after their surgery. How long they stay depends on the type of surgery they have, this might be for around 5-7 days.

Most children will go to the high dependency unit (HDU) and a few may go to intensive care unit (ICU) after their operation. On these units your child usually has one to one nursing care. These units can be quite busy and noisy places. But they allow your child to be carefully monitored by the nursing and surgical team.

As soon as your child’s doctors are sure they are recovering well, they move back to the children’s ward.

Children with small localised cancers may not need HDU or ICU. They will go to recovery after their operation, then back to the ward.

Side effects of neuroblastoma surgery

Your surgeon talks with you about the risks and benefits of neuroblastoma surgery. The main side effects include:

• infection

• bleeding

• pain

Other possible side effects depend on where the neuroblastoma is in the body.

Chemotherapy uses drugs to kill cancer cells. Children with intermediate or high risk neuroblastoma have chemotherapy.

Children with low risk neuroblastoma might also have chemotherapy, but this is less common. They might have chemotherapy because:

• their symptoms are causing particular problems, such as with their liver

• the cancer might be making breathing difficult

• there are changes in the cancer cells meaning it is more likely to come back

What happens

Your child has a course of several cycles of treatment. The number of cycles they have will depend on:

• the risk group of your child’s cancer

• if the cancer has any gene or chromosome changes

• if the neuroblastoma is causing any symptoms

Doctors usually give a combination of chemotherapy drugs, usually 2 or more different ones.

For low and intermediate risk neuroblastoma, children most commonly have:

• carboplatin

• etoposide

Sometimes this group of children might also have the CADO regimen which contains:

• cyclophosphamide

• vincristine

• doxorubicin

For high risk neuroblastoma, children usually have all of the following:

• carboplatin

• etoposide

• cyclophosphamide

• vincristine

• busulphan

• melphalan

• cisplatin

Your child will have a central line put in before they start treatment. This is a tube that is put into one of the large veins in the chest. They can have their chemotherapy drugs through the central line and nurses can take blood from it. It means that they need fewer needles. A central line is kept in place throughout treatment.

We have more information on the different types of central lines which is written for adults with cancer. But there are pictures and more detailed information that might be helpful to look at.

Read more about central lines

Side effects

Your child’s doctor will go through the risks and benefits of chemotherapy for neuroblastoma. The side effects depend on the drugs your child is having. Not everyone gets bad side effects from chemotherapy drugs. And the team looking after your child are very experienced in managing the side effects of chemotherapy.

Chemotherapy works by damaging dividing cells. Cancer cells divide much more often than most normal cells. So, chemotherapy damages cancer cells and can destroy them. But some types of normal cells divide very often too. This happens in tissues that need a steady supply of new cells, such as:

• the skin

• hair

• nails

Chemotherapy can also damage those cells, and this causes side effects. But the damaged normal tissues can repair themselves and recover.

Some of the main side effects of chemotherapy include:

• tiredness and weakness

• an increased risk of getting an infection

• a sore mouth

• hair thinning or hair loss

• feeling or being sick

• breathlessness and looking pale

• constipation or diarrhoea

Cisplatin and hearing loss

One of the common chemotherapy drugs used in neuroblastoma is cisplatin. A possible side effect of cisplatin is hearing loss. Some babies, children and young people can have a treatment called sodium thiosulfate. This is to try and reduce the risk of hearing loss. Your child has this 6 hours after the end of their cisplatin treatment.

Your child can only have this treatment if:

• they are between the age of 1 month and 17 years

• they have a solid cancer such as neuroblastoma that is not high risk

• the cancer has not spread to other areas of the body

Sodium thiosulfate also has side effects. The common side effects include feeling or being sick. Your child’s blood tests might show changes to levels of minerals and salts. This includes low levels of phosphate and potassium, and a high level of sodium.

Get more information on individual chemotherapy drugs, including possible side effects

About half the children diagnosed with neuroblastoma have high risk disease when diagnosed. Some of these children need more intensive treatment.

These children have high doses of chemotherapy and a stem cell transplant. It is commonly called a stem cell rescue. Some children may have 2 rounds of high does chemotherapy then a stem cell transplant. This is known as a tandem transplant.

This means the high dose chemotherapy destroys the neuroblastoma cells, but it also damages the bone marrow and kills off the cells that make blood cells. These are called stem cells. As part of high dose treatment these stem cells are replaced.

Your child’s transplant uses your child’s own stem cells. This is called an autologous transplant.

What happens

Early on in treatment, your child's stem cells are collected. They are usually collected from their bloodstream. But some children might have stem cells taken directly from their bone marrow.

Once the transplant team have enough stem cells, they are stored until after your child has had their high dose chemotherapy. This is also called conditioning treatment. Your child usually has the chemotherapy drugs:

• busulfan

• melphalan

If your child is having a tandem transplant they have the chemotherapy drug thiotepa beforehand.

After high dose chemotherapy, your child has the stem cells through a drip back into their bloodstream.

Side effects

The high dose chemotherapy can cause side effects. These can be quite severe, but the team caring for your child are experts in managing these side effects. They do everything they can to prevent or support your child through any problems.

The main side effects of this type of intensive treatment include:

• an increased risk of getting an infection

• an increased risk of bruising or bleeding from low blood cell counts

• sickness and diarrhoea

• a sore mouth

• difficulty eating and drinking

• feeling tired and run down

• veno occlusive disease (VOD) - this is when the small blood vessels of the liver get blocked

There are longer term side effects of having intensive chemotherapy. Your child’s team will talk these through with you.

We have information about autologous transplants aimed at adults with cancer. The process is very similar so you might find it helpful.

Having a transplant using your own cells

Radiotherapy means the use of radiation, usually x-rays to kill cancer cells.

When might your child have radiotherapy?

Your child might have radiotherapy after surgery. This is to reduce the risk of the cancer coming back. They are likely to have radiotherapy if they have either:

• intermediate neuroblastoma

• high risk neuroblastoma

Children with low risk neuroblastoma might have radiotherapy, but this is less common. This is only if their symptoms are causing them a lot of problems. For example, if the cancer is making breathing difficult.

After your child’s surgery the surgeon may have used a clip to mark an area of cancer they couldn’t remove. This is to help plan and target radiotherapy to this area.

What happens

Radiotherapy is usually aimed at the cancer from outside the body. This is called external beam radiotherapy.

Your child has treatment in the radiotherapy department. Radiotherapy machines are very big and they vary slightly in how they look and how they work.

To have treatment they lie on a couch and the machine gives the treatment. The machine doesn't touch your child. They don’t feel anything during treatment.

Play specialists are often available to help your child get used to having radiotherapy. The team at your hospital are used to treating children with cancer. They will explain everything clearly to you and to your child.

Proton beam therapy

Proton beam therapy is a type of radiotherapy.

It uses protons rather than high-energy x-rays to kill cancer cells. Protons are tiny particles from the centre of atoms. They produce a sudden burst of energy when they stop, which stays inside the cancer. This means there is less damage to healthy cells around the cancer.

Proton beam therapy has been shown to work well for some children’s neuroblastoma.

Your child’s specialist will talk with you if they think proton therapy might be beneficial for your child.

There are only 2 proton beam therapy centres in the UK and these are at:

• The Christie Hospital in Manchester

• University College London Hospital

At the moment children needing this service need to travel to one of these hospitals.

We have more information about proton beam therapy but it is aimed at adults with cancer.

Find out more about proton beam treatment

Molecular radiotherapy

Molecular radiotherapy is also called targeted radiotherapy. Your child might need molecular radiotherapy if their neuroblastoma is high risk. This is a type of internal radiotherapy. They might have it:

• before a stem cell transplant

• because chemotherapy is not working

• if the neuroblastoma has come back after earlier treatment

Molecular radiotherapy is similar to the mIBG scan that your child had at diagnosis. The main difference being that your child gets a higher dose of the radioactive iodine.

Find out more about having a mIBG test

mIBG is a drug that the doctors use to treat neuroblastoma. Radioactive iodine is attached to the mIBG. Your child has it through a vein into the bloodstream (intravenously).

The mIBG travels around the body and is picked up by the cancer cells. The amount of radioactive iodine attached to the mIBG is high enough to kill the cancer cells. But it is not too high to cause serious side effects to surrounding tissues

This treatment can damage the bone marrow and kill off the cells that make blood cells. So children having this treatment might also need a stem cell transplant.

As it’s a type of internal radiotherapy, any child having mIBG treatment needs to be cared for on their own. This is because any of their bodily fluids such as their pee and saliva will contain radiation. So it’s important to reduce the risk to other people coming into contact with it.

It’s normal to feel a bit nervous about this at first. The specialist team caring for your child are used to looking after children who need to isolate. So they will let you know what to expect and how best you and your family might cope.

Some of the precautions you will follow include:

• wearing a radiation monitor

• wearing aprons, gloves and shoe covers when in the room

• staying behind the lead shields as much as possible

• minimal close contact such as cuddling at the start of treatment

• staying in the relative’s area just outside their room when they are sleeping, playing or resting

Immunotherapy treatment uses your child’s immune system to fight cancer. It works by helping the immune system recognise and attack the cancer cells.

A type of immunotherapy treatment your child might have is a monoclonal antibody. Monoclonal antibodies are man made versions of proteins that our immune system makes.

Some monoclonal antibodies can recognise and attach to specific proteins on neuroblastoma cells. This can help the immune system recognise the neuroblastoma cells and kill them.

The monoclonal antibody treatment used for neuroblastoma is called anti GD2. It is called dinutuximab beta.

Neuroblastoma cells contain a substance called disialoganglioside 2 (GD2) on their cancer cells. Dinutuximab beta works by seeking out the GD2 substances and locking onto it. Once locked on it triggers the immune system to kill any cancer cells.

Your child might have dinutuximab beta as their first treatment if they:

• are 12 months old and over

• have high risk neuroblastoma

• have had some response to chemotherapy

• have had high dose treatment and a stem cell transplant

• have not already had anti GD2 immunotherapy

Some children might have dinutuximab beta if their cancer has come back (relapsed) or not gone away (refractory). But they would also have to meet the above recommendations.

How your child has anti dinutuximab beta treatment

Your child has the immunotherapy by injection into the bloodstream (intravenously). Most children have it through their central line.

Your child has dinutuximab beta as a cycle of treatment. Each cycle is 35 days. They usually have 5 cycles in total.

Your child has this with a drug called isotretinoin. This is a man made form of vitamin A.

Eflornithine is a treatment for some children with high risk neuroblastoma. It is a maintenance treatment. Eflornithine is not licensed in the UK. It is only available through self-funding. Your child’s doctor is this best person to speak to about this medicine, they can also discuss if this is the best option for them.

It’s scientific name is difluoromethylornithine or DFMO for short. This medicine works by blocking an enzyme that helps neuroblastoma cells grow.

The UK neuroblastoma clinical trials Group and CCLG Neuroblastoma Special Interest Group have a statement about this medicine and how it works. This can be found on the CCLG website.

Go to the CCLG information on neuroblastoma

Or you can read more about it on the SIOPEN neuroblastoma research network.

Go to the SIOPEN neuroblastoma research network

The charity Solving Kids’ Cancer UK has the latest information about UK access.

Go to Solving Kids’ Cancer Charity UK