
"I am glad that taking part in a trial might help others on their own cancer journey.”
This trial was looking at a new combination of drugs. It was for children with neuroblastoma whose treatment had not worked as well as doctors had hoped.
Neuroblastoma is a rare type of cancer that develops in nerve cells, most commonly in nerve cells of the . It usually affects babies and young children. Neuroblastoma can spread to other parts of the body. This is called
. The most common places for neuroblastoma to spread are the bones, liver and skin.
Children with neuroblastoma usually have a combination of treatments including , chemotherapy and
. Treatment depends on:
Sometimes the treatment doesn’t work very well, or the cancer comes back after treatment (recurs). If this happens it is more difficult to treat.
Vincristine and doxorubicin are used to treat children and young people with cancer. Topotecan is a chemotherapy drug used to treat some adult types of cancer. And it’s already been looked at in trials for some childhood cancers. Earlier studies in childhood cancers had shown that adding topotecan to other drugs could help treatment work better.
This trial aimed to find out:
The trial was for children over 1 year and young people under the age of 20 with advanced neuroblastoma. They were at high risk of their cancer coming back after treatment. And their hadn’t got rid of or shrunk their neuroblastoma very well.
The trial team found that TVD helped some children and young people with advanced, high risk neuroblastoma.
63 children and young people took part. Everyone had TVD. They had treatment in 3 week periods called cycles of treatment.
They had topotecan on days 1 to 5 through their central line. This was followed by vincristine and doxorubicin non stop through a pump over days 5 and 6. They had no treatment in weeks 2 or 3. After the first cycle of treatment the children and young people had tests to find out if treatment had worked.
Everyone had a good enough response so they all had another cycle of treatment over 6 weeks. After the second cycle of treatment, everyone had tests to find out if treatment had continued to work.
The trial team analysed the test results after 2 courses of TVD and:
The neuroblastoma continued to grow during the TVD treatment for 1 child taking part.
For 23 out of the 63 young people, the TVD worked well enough to have the next phase of their treatment plan. This is called high dose treatment.
The researchers also found that TVD worked better for neuroblastoma that had spread to the than to bone. And it didn’t work as well for those with neuroblastoma that stayed the same, or got worse, after first line chemotherapy.
Side effects
The trial team also looked at the of TVD. The main side effects were:
The trial team concluded that TVD can help children and young people with advanced neuroblastoma. They recommend more clinical trials comparing TVD with other first line chemotherapy treatments.
Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.
Dr G. Makin
Cancer Research UK Children's Cancer Trials Team
University of Birmingham
NIHR Clinical Research Network: Cancer
Freephone 0808 800 4040
"I am glad that taking part in a trial might help others on their own cancer journey.”