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

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

EpSSG RMS 2005 trial part 1 randomisation diagram

Results of Part 1
The median  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

EpSSG RMS 2005 trial graph 1

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

EpSSG RMS 2005 trial graph 2

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 ) 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 , 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

EpSSG RMS 2005 trial part 2 randomisation diagram

Results for Part 2
The median 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

EpSSG RMS 2005 trial graph 4

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 ) 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.