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Increased survival with new treatment for aggressive form of childhood cancer

Karolinska Institutet Mar 22, 2017

An international team of scientists, including researchers from Karolinska Institutet and Karolinska University Hospital, have conducted a clinical study on a new treatment for high–risk neuroblastoma. According to the researchers, the results demonstrate that the new treatment both increases survival and reduces undesirable effects.

“This is a significant step forwards for these seriously ill children,” says consultant Per Kogner, professor of paediatric oncology at the Department of Women’s and Children’s Health, who led the Swedish arm of the study.

The preparation tested is the new pharmaceutical combination busulfan–melphalan, which is given in high doses followed by stem cell transplantation. In all, 1347 children from 18 countries were included in a so called phase 3 study, of which 598 children were randomised to be treated with busulfan–melphalan or standard therapy. The research team found that the survival rate for those who received this new treatment increased by 38 to 50 per cent, while the risk of life–threatening adverse effects decreased from 10 to 4 per cent.

The current collaboration study was published in The Lancet Oncology journal and was coordinated by Professor Ruth Ladenstein at the MedUni Vienna in Austria.
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