More accurate disease classification now means some young children with neuroblastoma will have less intensive treatment with better survival, according to research published in the Journal of Clinical Oncology*.
In a pan-European trial, researchers found that screening the tumours of children under the age of one with advanced neuroblastoma for a gene called MYCN could save them from unnecessary chemotherapy.
The number of MYCN genes are known to be increased in about 25 per cent of neuroblastomas. Tumours with increased numbers of this gene behave much more aggressively.
In this trial, 96 per cent of patients whose neuroblastoma had spread to distant parts of the body but did not have the amplified gene survived five years or more with little or no treatment.
Neuroblastoma affects around 90 children each year in the UK and across the board, five year survival rates are around 60 per cent. Around 30 children under the age of one are diagnosed with neuroblastoma each year in the UK.
The study was led by Cancer Research UK’s Professor of Paediatric Oncology Professor Andy Pearson at the Institute of Cancer Research and the Royal Marsden Hospital in Sutton with Dr Mary Gerrard at Sheffield Children’s Hospital and conducted by researchers from the International Society of Paediatric Oncology Europe Neuroblastoma Group (SIOPEN) and the Children’s Cancer and Leukaemia Group (CCLG).
They treated 170 babies from nine European countries with neuroblastoma that had spread to distant parts of the body – but who did not have the amplified MYCN gene in their tumours.
These children were treated with up to four courses of chemotherapy. Some children were treated with surgery first if their doctors felt they needed it.
Previously, these children would have been treated with up to 12 courses of chemotherapy.
Professor Pearson said: “This is very positive news for babies with this type of neuroblastoma. We saw an excellent outcome in the children we treated who were spared extra therapy and the side effects that come with it. Chemotherapy can cause children to feel tired, sick and make it more likely they’ll pick up infections, so it’s very important to avoid this where possible. Our current goal is to individualise or personalise treatment for children with neuroblastoma.”
The results of this trial suggest that less intense chemotherapy should be given to this group of babies.
Kate Law, Cancer Research UK’s director of clinical trials, said: “Overall, survival rates for children’s cancers have been rapidly improving over the past three decades. But it’s crucial we support trials like this to refine treatments and reduce the long-term side effects that curative therapies can sometimes cause.
“This study confirms the importance of understanding the personal biology of individual patients in creating successful, tailored treatment regimes.”
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Bagatell, R., Beck-Popovic, M., London, W., Zhang, Y., Pearson, A., Matthay, K., Monclair, T., Ambros, P., & Cohn, S. (2008). Significance of MYCN Amplification in International Neuroblastoma Staging System Stage 1 and 2 Neuroblastoma: A Report From the International Neuroblastoma Risk Group Database Journal of Clinical Oncology, 27 (3), 365-370 DOI: 10.1200/JCO.2008.17.9184
* Significance of MYCN Amplification in International Neuroblastoma Staging System Stage 1 and 2 Neuroblastoma: A Report From the International Neuroblastoma Risk Group Database. Bagatell et al. Journal of Clinical Oncology.
This study was conducted in nine European counties: UK, Italy, France, Spain, Portugal, Norway, Austria, Belgium and Switzerland.
Stages of neuroblastoma
The stage of a cancer tells the doctor how far it has grown or spread. The stage is important because it helps the doctor decide which treatment to give.
There are 4 main stages of neuroblastoma, but stages 2 and 4 are divided into two further groups.
Children in this study were stage 4 or 4S.
Stage 4 means the cancer has spread to parts of the body that are some distance from where it started. Stage 4S is a special case in children younger than one year old when diagnosed, as it has a better outlook than other stages and sometimes the cancer goes away without treatment Neuroblastoma may have spread to the liver or skin, but not to the bones. And no more than 10 per cent of cells in the bone marrow are neuroblastoma cells.
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