A trial of temozolomide for children with neuroblastoma that has come back after treatment (NAG 2003 02)

Cancer type:

Children's cancers

Neuroblastoma

Status:

Results

Phase:

Phase 2

This trial was looking at the chemotherapy drug temozolomide for children with neuroblastoma.

Neuroblastoma Open a glossary item is a rare type of cancer that affects young children. Doctors usually treat it with chemotherapy, surgery and sometimes radiotherapy. But sometimes the treatment doesn’t work very well, or the cancer comes back after treatment (recurs). If this happens it is more difficult to treat.

Temozolomide is a chemotherapy drug that doctors use to treat some forms of brain tumour in adults (such as glioma). The researchers think temozolomide may be useful for treating children with neuroblastoma.

The aim of this trial was to see how well temozolomide worked for children with neuroblastoma that had not responded to treatment, or that had come back after treatment.

Summary of results

The trial team found that temozolomide helped some children with neuroblastoma that had not responded to treatment or had come back after treatment.

All the children had temozolomide. They had 5 days of temozolomide every 28 days. Each 28 day period is called a cycle of treatment. Each child had up to 12 cycles of treatment over 12 months.

The trial team were able to analyse the results of 25 children. Of these 25, 10 had responded to temozolomide. The trial team found that

In 5 children the neuroblastoma got smaller – this is called a partial response , 1 of which was a very good partial response
In 2 children the neuroblastoma showed signs of getting smaller – the researchers called this an objective response
In 3 children where the neuroblastoma had spread to other parts of the body these areas had got smaller – the researchers called this a mixed response

The main side effects were a drop in blood cells and infection.

The trial team concluded that temozolomide could work for children with neuroblastoma that had not responded to treatment or had come back after treatment. They say that temozolomide should be looked at further and in combination with another drug.

We have based this summary on information from the team who ran the trial. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item ) and published in a medical journal. The figures we quote above were provided by the trial team. We have not analysed the data ourselves.