Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.
A trial looking at treatment for children and young people with an ependymoma (SIOP 99)
This trial looked at radiotherapy with or without chemotherapy for people with a type of brain tumour called an ependymoma. It was for children and young people from 3 to 21 years old.
The trial was open for people to join between 1999 and 2007. The team published long term results in 2022.
More about this trial
In this trial, they wanted to find out if chemotherapy would be a useful treatment. They gave this after surgery and before radiotherapy.
The main aim of this study was to find out if chemotherapy could help stop the cancer coming back and help people live longer.
Summary of results
The research team found that vincristine, etoposide and cyclophosphamide chemotherapy might be a useful treatment for ependymoma.
This trial was for children and young people from 3 to 21 years old. They all had an ependymoma and were due to have surgery to remove it.
If the surgeons were able to remove all of the tumour, they had radiotherapy after their operation.
If the surgeons weren’t quite able to remove all of the tumour, they had chemotherapy after their operation. And then radiotherapy after that.
A total of 74 children and young people had treatment as part of this trial. They all had surgery to begin with.
The surgeons were:
- able to remove all of the tumour in 33 people
- not able to remove all of the tumour in 41 people
Most of those who didn’t have all of their tumour removed during surgery had chemotherapy. The research team looked at how well this worked in 29 people.
They hoped the cancer would either go away or get smaller in at least 45% of people. They found it did in 65% of people. The cancer had:
- gone away in 9 people (31%)
- got a lot smaller in 10 people (34%)
- stayed the same in 4 people (14%)
- continued to grow in 6 people (21%)
The research team then looked at how well people were doing 10 years after joining the trial.
They looked at the number of people who didn’t have signs that their cancer had started to grow again. They found it was:
- nearly half (47%) of all those in the trial
- more than 6 out of 10 (63%) of those who had all of their tumour removed
- more than 3 out of 10 (34%) of those who hadn’t had all of their tumour removed
They also looked at how many people were living. They found it was:
- about 6 out of 10 (61%) of all those in the trial
- nearly 7 out of 10 (68%) of those who had all of their tumour removed
- more than 5 out of 10 (54%) of those who hadn’t had all of their tumour removed
Everyone who had chemotherapy had at least one side effect. Many were mild or didn’t last long. But some were more severe.
The most common side effects of chemotherapy were:
- a drop in white blood cells
- a drop in blood clotting cells (platelets)
- feeling or being sick
The research team looked for changes in some genes and proteins. They wanted to see if there was a link between them and how well treatment worked.
They found 3 changes which seemed to affect how well people did.
The trial team concluded that VEC chemotherapy could be a useful treatment for people whose tumour cannot be completely removed with surgery. And that it didn’t cause too many side effects.
They suggest more work is done to find more about out how well it works. And to look in more detail at who would benefit most from chemotherapy.
Where this information comes from
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (
How to join a clinical trial
Professor Richard Grundy
Dr Maura Massimino
Associazione Bianca Garavaglia
Cancer Research UK Children's Cancer Trials Team
Children with Cancer UK
The Joe Foote Research Foundation
The James Tudor Foundation
University of Birmingham
NIHR Clinical Research Network: Cancer