Less intensive chemotherapy avoids irreversible side effects in some childhood cancers

8 Jul 2015, 6:19 p.m.

Children with a rare type of cancer called Wilms’ tumour who are at low risk of relapsing can now be given less intensive treatment, avoiding a type of chemotherapy that can cause irreversible heart problems in later life, reports a team led by researchers at Great Ormond Street Hospital (GOSH).The move follows the results of a trial, published in the Lancet* today, showing that the drug doxorubicin can be safely omitted from treatment without affecting patients’ chances of survival.

Wilms’ tumour is a type of kidney cancer that affects around 80 children a year in the UK, most under the age of seven. Until now, about one half of these patients would have received doxorubicin as part of their treatment, which carries a small risk of heart problems in later life. Now only about one quarter of children at the greatest risk of relapse need the drug.

Doxorubicin is helpful in treating many types of cancer. But with around nine in 10 of children with Wilms' tumour now cured, doctors wanted to find out whether it could be safely left out from the treatment of those who had a low chance of relapse, without affecting survival chances.

As part of the study, which was led by Kathy Pritchard-Jones, consultant oncologist at GOSH and researcher at the UCL Institute of Child Health, 583 children with stage II or stage III Wilms' tumour, who were at intermediate risk of relapsing, took part in a 10 year trial from 2001-2011 across 26 European countries. The results showed that 96.5 per cent of children whose treatment included doxorubicin survived for five years or more, compared with 95.8 per cent of children who did not receive the drug.  Although there were slightly more relapses among the children who avoided doxorubicin, they could all be successfully re-treated, meaning there was no impact on the overall risk of death.

The researchers are now carrying out further work, funded by Cancer Research UK, to pinpoint genetic changes in tumour samples from these patients that could be used to better predict the chance of relapse, so treatment can be tailored accordingly.

Professor Kathy Pritchard-Jones said: “Thanks to the results of this trial fewer children with this disease will have to have treatment that could cause them lifelong-side effects without much benefit. 

Professor Pam Kearns, professor of paediatric oncology and Cancer Research UK’s senior clinical advisor, said: “This is a very important trial that has changed clinical practice for this type of Wilms’ Tumour.