What is the treatment for refractory neuroblastoma? 

Refractory neuroblastoma is when the cancer does not respond as expected to initial (frontline) treatment. Because every child’s situation is different, there is no single standard treatment pathway. Instead, doctors tailor treatment to your child’s needs using the best available evidence. 

Your child’s medical team will consider several factors, including: 

• which treatments your child has already received 

• how the cancer has behaved and whether it has spread 

• your child’s age and overall health 

• biological and genetic features of the tumour (such as ALK changes or MYCN status) 

Treatment often involves a combination of different therapies. 

Chemotherapy 

Children with refractory neuroblastoma may be offered different chemotherapy combinations from those used during frontline treatment. These medicines aim to shrink the cancer, control symptoms, or allow other treatments, such as radiotherapy or surgery to be used. 

The medicines and number of cycles vary depending on how the cancer responds. 

Targeted therapies 

Some children may benefit from targeted therapies that act on specific genetic features of the tumour. For example, children with ALK mutations may be offered ALK‑targeted medicines if these are available and appropriate. 

Your child’s medical team will explain whether any targeted options are relevant and how you might be able to access them. 

Immunotherapy 

Immunotherapy helps the immune system recognise and attack cancer cells. 

This may be offered again, or given in a different way from frontline treatment, depending on how the cancer has behaved. 

Radiotherapy or molecular radiotherapy 

Radiotherapy may be used to treat specific areas of cancer that are causing symptoms or are not responding well to other treatments. Some children may also be offered molecular radiotherapy, such as mIBG therapy, which delivers radiation directly to neuroblastoma cells through a radioactive medicine taken up by the tumour. 

Clinical trials 

Children with refractory neuroblastoma are often considered for clinical trials. 

Trials may offer access to newer treatments that are not yet widely available, or to different ways of using existing treatments. Taking part in a trial is always a choice, and your child will continue to receive the best available care whether you join a trial or not. 

Read more about clinical trials for refractory neuroblastoma. 

Why treatment varies

Refractory disease can behave very differently from one child to another. This means that: 

• treatment plans are highly individual 

• options may change depending on how the cancer responds 

• doctors may use a combination of therapies over time 

• your child’s plan may differ from other children you know 

Your child’s treatment team will talk you through all options, explain why each treatment is being recommended, and support you to make informed decisions.

Further information 

More detailed information about treatment approaches for relapsed and refractory neuroblastoma is available in the joint patient information booklet produced by the Children and Young People’s Cancer Association (CCLG), Solving Kids’ Cancer UK and Neuroblastoma UK.

CCLG: Treatment for relapsed or refractory neuroblastoma (PDF)