Clinical update

NICE recommends drug to treat rare form of cancer

National Institute for Health and Care Excellence proposes drug for high-risk neuroblastoma

National Institute for Health and Care Excellence proposes drug for treating high-risk neuroblastoma

Picture: Getty Images

Essential facts

The National Institute for Health and Care Excellence (NICE) has recommended a drug for the treatment of high-risk neuroblastoma – a rare type of cancer that mainly affects children and young people.

Neuroblastoma is most common in children under the age of five and is estimated to affect approximately 100 children per year. The existing treatments and procedures for neuroblastoma are painful and debilitating, with severe and long-lasting side effects.

The NICE appraisal committee recommended the drug for the NHS in draft guidance. Modelled trials have estimated the survival gain of the drug to be between three to five years, compared to the current treatment option of isotretinoin, according to NICE.  

What’s new

Treatments for high-risk neuroblastoma include chemotherapy, radiotherapy, stem cell transplant, surgery and isotretinoin. Dinutuximab beta is an important, potentially curative option for maintenance treatment of the disease, the committee agreed.

The guidance suggests that dinutuximab beta increases survival and the length of time before the disease progresses, but it does not meet NICE’s criteria for life-extending treatment at the end of life.

Signs and symptoms

The most common symptom of neuroblastoma is a lump in the abdomen. This could make a child’s tummy swell, causing discomfort or pain. 

Occasionally, it can affect the spinal cord. This can cause:

  • Numbness
  • Weakness
  • Loss of movement in the lower part of the body
  • Rarely, neuroblastoma can appear as a lump in the neck, this might cause breathlessness or difficulty swallowing.

Other symptoms depend on where the neuroblastoma starts in the body and whether it is just localised or has spread to other parts of the body. 

In around half of children, the neuroblastoma has spread to another part of the body at diagnosis.

How you can help your patient

A child should be referred to a specialist within two days if they have any of the following:

  • A lump in the abdomen.
  • An enlarged or swollen organ in the tummy.

NICE guidelines make clear that healthcare professionals should take the parent or carer’s concern about their child into account when deciding about a specialist referral.

Expert comment

Stephen Richards is chief executive of the charity Solving Kids' Cancer

‘Neuroblastoma is a tumour that develops from specialised nerve cells left behind from a baby’s development.

'It has one of the lowest survival rates of all childhood cancers, with only 68% of children surviving longer than five years. If a child’s neuroblastoma is considered ‘high-risk’, long-term survival is around 40%. If that cancer returns, survival drops to 10%.

'Anti-GD2 antibody dinutuximab beta is given at the end of standard therapy and harnesses the body’s own immune system to target and attack the cancer. The approval of dinutuximab beta means that children with high-risk neuroblastoma now have the best treatment, giving them the greatest chance of long-term survival here in the UK.’


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