A trial looking at treatment for children and young people with an ependymoma (SIOP 99)

Cancer type:

Brain (and spinal cord) tumours
Children's cancers

Status:

Results

Phase:

Phase 3

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

Ependymoma is a rare type of brain tumour. When this trial was done, doctors usually used surgery followed by radiotherapy to treat ependymomas. 

In this trial, they wanted to find out if chemotherapy would be a useful treatment. They gave this after surgery and before radiotherapy.

Some people in this trial had a combination of chemotherapy called VEC. This is the drugs vincristine, etoposide and cyclophosphamide

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.

Trial design
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.

Results
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

Side effects
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
  • infection

We have more information about the side effects of vincristine, etoposide and cyclophosphamide in our Cancer drugs section.

Genetic changes
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. 

Conclusion
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 (peer reviewed Open a glossary item) and published in a medical journal. The figures we quote above were provided by the trial team who did the research. We have not analysed the data ourselves.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Professor Richard Grundy
Dr Maura Massimino

Supported by

Associazione Bianca Garavaglia 
Brainstrust 
Cancer Research UK Children's Cancer Trials Team
Children with Cancer UK
Fighting Ependymoma
The Joe Foote Research Foundation
The James Tudor Foundation
University of Birmingham
NIHR Clinical Research Network: Cancer

Freephone 0808 800 4040

Last review date

CRUK internal database number:

249

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Cara took part in a clinical trial

A picture of Cara

"I am glad that taking part in a trial might help others on their own cancer journey.”

Last reviewed:

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