A trial looking at treatment for children and young people with medulloblastoma (CNS 2001 06)

Cancer type:

Brain (and spinal cord) tumours

Children's cancers

Status:

Results

Phase:

Phase 2

This trial looked at radiotherapy and chemotherapy for children and young people with medulloblastoma that has spread (metastasised).

Medulloblastoma is a type of brain tumour called a primitive neuro-ectodermal tumour (PNET). It is usually treated with surgery, radiotherapy and chemotherapy.

After surgery, children usually have radiotherapy once a day for 4 to 6 weeks. In this trial, they had radiotherapy twice a day instead of once a day. This is called hyperfractionated accelerated radiotherapy, or HART.

There were two parts to this trial. In the first part, children had HART on its own. In the second part, they had a combination of HART and a chemotherapy drug called vincristine.

The aims of this trial were to

See how well HART works for medulloblastoma
Find out more about the side effects

Summary of results

The trial team found that hyperfractionated accelerated radiotherapy (HART) didn’t cause too many side effects, and that it may be a useful treatment for medulloblastoma.

This phase 2 trial recruited 34 children aged 3 to 15 years old who had medulloblastoma that had spread.

The trial was in 2 parts. Everyone taking part had HART. They had radiotherapy twice a day, Monday to Friday, for 5 weeks. In part 1, 7 children had HART on its own. In part 2, 27 children had a combination of HART and vincristine chemotherapy.

Everyone taking part then had what doctors call maintenance chemotherapy, which started a few weeks after they finished radiotherapy.

The research team looked at how well the treatment worked, and found that

More than 9 out of 10 children (94%) were living 1 year after treatment
About 8 out of 10 children (81%) were living 2 years after treatment
About 7 out of 10 children (71%) were living 3 years after treatment

The treatment did cause some side effects. The most common side effects were a sore mouth, feeling or being sick and a drop in blood cells causing an increased risk of infection, bleeding, bruising, tiredness and shortness of breath.

The research team concluded that HART could be a useful treatment for children with medulloblastoma, and that it didn’t cause too many serious side effects. They suggest that it is looked at further in a larger phase 3 trial.

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.