"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A trial looking at treatment for children and young people with Wilms' tumour (SIOP WT 2001)
This trial looked at improving treatment for children and young people with a type of kidney cancer called Wilms' tumour. The trial was for children and young people up to and including the age of 18. This trial was supported by Cancer Research UK.
More about this trial
Wilms’ tumour (nephroblastoma) is a type of kidney cancer that affects children. When a baby is growing in the womb, the kidneys develop very early on. Sometimes something goes wrong and some of the cells that should have turned into mature kidney cells don’t. These cells can develop into a Wilms’ tumour. It is a rare cancer that affects about 1 in 10,000 children.
Wilms’ tumours are usually treated with chemotherapy and surgery, and sometimes radiotherapy. But doctors wonder if they may be giving more chemotherapy than is necessary for certain types of Wilms' tumours. This extra treatment may be causing side effects and may not be needed to treat the cancer successfully. The chemotherapy drug is called doxorubicin and it can cause heart problems in the long term.
In this trial, they looked at children with stage 2 or stage 3 cancer who have a medium risk of the cancer coming back after treatment. Doctors looked at the Wilms’ tumour cells under a microscope to decide what risk a child had of the cancer coming back.
The aim of the trial was to find if it is safe to exclude doxorubicin from the usual chemotherapy given to children with Wilms' tumour that has a medium risk of coming back.
Summary of results
The trial team found that doxorubicin doesn’t need to be included in chemotherapy treatment for children and young people with stage 2 or stage 3 cancer who have a medium risk of their Wilms’ tumour coming back.
This was a phase 3 trial. 583 children and young people up to the age of 18 took part. They had stage 2 or stage 3 Wilms' tumour and had already had surgery before joining the trial. The children were put into 1 of 2 groups at random.
- 291 had vincristine, actinomycin D and doxorubicin (the usual treatment for Wilms’ tumour when this trial was done)
- 292 had vincristine and actinomycin D
The researchers looked at the number of children living 5 years after treatment. They call this overall survival. They found that there was no significant difference between the 2 different groups.
They also looked at whose cancer hadn’t come back at 2 and 5 years. They found this was at 2 Years
- Just over 9 out of 10 children (92.6%) whose treatment included doxorubicin
- Just under 9 out of 10 children (88.2%) whose treatment hadn’t included doxorubicin
And at 5 years
- Just over 9 out of 10 children (91.8%) whose treatment included doxorubicin
- Just under 9 out of 10 children (85.3%) whose treatment hadn’t included doxorubicin.
Although the cancer came back in slightly more of the children who didn’t have doxorubicin, the trial team say they all had further treatment that worked well.
The children in the doxorubicin group had more side effects. These included heart problems.
The trial team concluded that it is safe to exclude doxorubicin as part of chemotherapy treatment for this group of children without affecting overall survival.
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
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Professor Kathy Pritchard-Jones
Cancer Research UK
Cancer Research UK Children's Cancer Trials Team
University of Birmingham
NIHR Clinical Research Network: Cancer
This is Cancer Research UK trial number CRUK/02/018.