A trial looking at the treatment of children and young people with rhabdomyosarcoma (EpSSG RMS 2005 STS 2006 04)

Cancer type:

Children's cancers
Sarcoma
Soft tissue sarcoma

Status:

Results

Phase:

Phase 3

This trial looked at chemotherapy for children and young people who after initial treatment for rhabdomyosarcoma had a high chance of it coming back. 

Rhabdomyosarcoma is a type of soft tissue sarcoma

The trial was open between 2003 and 2016 for people to join. Results for this trial were published in 2018 and 2019. 

Cancer Research UK supported this trial.

More about this trial

Rhabdomyosarcoma affects cells in muscle tissue. Children who have this cancer are usually treated with:

Some of the children in this trial had treatment according to an internationally agreed protocol (called EpSSG RMS 2005). These protocols were established as a result of earlier trials.

The researchers followed a large number of children to learn more about existing treatments and their side effects. They hoped this would lead to improvements in treatment in the future.

Many children with rhabdomyosarcoma can be cured. But there are some children whose rhabdomyosarcoma is more difficult to treat. This trial also aimed to find out if this group of children and young people benefited from:

  • adding doxorubicin to the existing chemotherapy drugs (standard treatment Open a glossary item)
  • having chemotherapy (vinorelbine and cyclophosphamide) for a further 6 months

Summary of results

The trial team found that adding doxorubicin to the standard treatment didn’t extend the lives of these children and young people. 

About Part 1 of this trial
This was a phase 3 trial. 484 children took part.

It was a randomised trial. Everyone who took part was put into a treatment group. Neither they nor their doctor chose which group they were in:

  • 242 children and young people had the standard chemotherapy treatment
  • 242 children and young people had the standard chemotherapy treatment plus doxorubicin

Results of Part 1
The median Open a glossary item follow up for people in this group was just over 5 years (63.9 months). 

The team looked at how many children and young people were alive at 3 years and didn’t have a sign that their cancer was coming back or getting worse. They found it was:

  • 63 children and young people out of every 100 (63.3%) in the standard chemotherapy group 
  • 68 children and young people out of every 100 (67.5%) in the doxorubicin group

They also looked at the total number of children and young people who were alive 3 years after treatment. They found it was:

  • 81 children and young people out of every 100 (80.6%) in the standard chemotherapy group
  • 78 children and young people out of every 100 (78.3%) in the doxorubicin group

Side effects
The researchers looked at the worst side effects in each group. They found that more children and young people in the doxorubicin group had these side effects than those in the standard treatment group. 

These included:

  • a drop in the number of blood cells causing an increased risk of infection, bruising and bleeding
  • an increase in the number of infections and sickness
  • diarrhoea or constipation

Conclusion for Part 1
The committee that looks at the running and safety of the trial (Data Monitoring Committee Open a glossary item) advised to stop the trial early as adding doxorubicin didn’t work much better than the standard treatment. And those who had doxorubicin had more side effects.

The team concluded that the standard chemotherapy for rhabdomyosarcoma should remain as it is.  

Summary of results for Part 2
The team found that having vinorelbine and cyclophosphamide after initial treatment extended the lives of children and young people with rhabdomyosarcoma. And they could have the 6 cycles of treatment Open a glossary item, each cycle 4 weeks apart, as an outpatient.

About part 2 of this trial
This was a phase 3 trial

Everyone had standard chemotherapy treatment or standard chemotherapy plus doxorubicin. They could then take part in this trial if there was no sign of their cancer after the initial treatment. 

It was a randomised trial. Everyone who took part was put into a treatment group. Neither they nor their doctor chose which group they were in.

371 children and young people took part:

  • 186 children and young people didn’t have chemotherapy after their initial treatment
  • 185 children and young people continued with vinorelbine and cyclophosphamide after their initial treatment

Results for Part 2
The median Open a glossary item follow up for children and young people in this group who were still alive was just over 5 years (60.3 months). 

They looked at the total number of children and young people who were still alive 5 years after treatment. They found it was:

  • 74 out of every 100 children and young people (73.8%) who didn’t continue with chemotherapy 
  • 87 out of every 100 children and young people (86.5%) who continued to have chemotherapy

Side effects
The most common side effects were:

  • a drop in the number of blood cells causing an increased risk of infection, bruising and bleeding
  • infection
  • feeling or being sick
  • bowel problems such as constipation
  • weakness or abnormal sensation in the lower arms and legs
  • low levels of potassium in the blood

Conclusion
The trial team concluded for children and young people with rhabdomyosarcoma who had no sign of their cancer after initial treatment having vinorelbine and cyclophosphamide:

  • extended their lives
  • was safe and acceptable 

This is now the standard treatment for children and young people with rhabdomyosarcoma that has a high chance of coming back after initial 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 Meriel Jenney

Supported by

Cancer Research UK
Children's Cancer and Leukaemia Group (CCLG)
NIHR Clinical Research Network: Cancer
Cancer Research UK
Children's Cancer Trials Team University of Birmingham
NCRI Children’s Cancer and Leukaemia Clinical Studies Group

Other information

This is Cancer Research UK trial number CRUK/06/027.

Freephone 0808 800 4040

Last review date

CRUK internal database number:

765

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

Over 60,000 cancer patients enrolled on clinical trials in the UK last year.

Last reviewed:

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