A trial looking at etoposide for children and young people with ependymoma (CNS 2001 04)

Trial design
This trial was for children and young people up to 21 years old who had ependymoma. It was for people who’d had treatment, but their brain tumour had not gone away or it had come back. Doctors were not able to remove it with surgery.

The people taking part had etoposide through a drip into a vein. They had treatment 3 days a week for 3 weeks. And then a week with no treatment. Each 4 week period is called a cycle of treatment. They had up to 6 cycles.

The research teams looked at scans before, during and after treatment to see how well it was working.

Results
A total of 25 people joined this trial. They were between 2 and 17 years old. Three people didn’t have treatment as part of the trial. The other 22 people had between 1 and 6 cycles of treatment.

The trial team looked at how well the treatment worked. They were able to assess this in 21 people (96%) who took part. They found that the brain tumour had:

• gone away in 2 people (9%)
• got smaller in 7 people (32%)
• stayed the same in 5 people (23%)
• continued to grow in 7 people (32%)

Diagram 1

They looked at how many people’s cancer had not started to grow again within 1 year and 3 years of joining the trial. They found it was:

• 5 people (23%) at 1 year
• 3 people (14%) at 3 years

They also looked at how many people were living 1 year and 5 years after joining the trial. They found it was:

• 13 people (59%) at 1 year
• 4 people (18%) at 5 years

These results are similar to trials that looked at having etoposide tablets. In this trial people had etoposide into a vein (intravenous treatment).

Side effects
Everyone who had treatment had at least 1 side effect. Some were mild or didn’t last long. But most people had at least 1 that was more severe.

The most common of the more severe side effects was a drop in blood cells. This includes a drop in white blood cells, red blood cells and clotting cells (platelets).

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

Conclusion
The trial team concluded that etoposide may be useful for some children and young people with ependymoma that has come back and can’t be removed with surgery. But it doesn’t work for everyone. They say it might be helpful to use etoposide in combination with other treatments.

They also suggest more work is done looking at biomarkers  to help find out who is likely to benefit from treatment.

More detailed information
There is more information about this research in the reference below. 

Please note, this article is not in plain English. It has been written for health care professionals and researchers.

Journal articles
Phase II study of intravenous etoposide in patients with relapsed ependymoma (CNS 2001 04) 
John R Apps and others
Neuro-Oncology Advances, 2022. Volume 4, Issue 1

Where this information comes from    
We have based this summary on the information in the article above. This has been reviewed by independent specialists (peer reviewed ) and published in a medical journal. We have not analysed the data ourselves. As far as we are aware, the link we list above is active and the article is free and available to view.