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According to the Cancer Council about 750 children aged 0-14 are diagnosed with cancer each year in Australia.

Recent advancements in paediatric cancer research have highlighted the potential of precision-guided treatment (PGT) in managing high-risk cases. This study from researchers in New South Wales delves into the efficacy of PGT for children with aggressive cancers, presenting a more targeted approach to therapy that could revolutionise outcomes for this vulnerable group.

The ZERO Childhood Cancer Precision Medicine Program’s PRISM trial conducted a comprehensive analysis on 384 children diagnosed with high-risk paediatric cancers, all of whom had a predicted cure rate of less than 30 per cent. Using whole-genome sequencing and other precision medicine techniques, the trial aimed to pinpoint molecular targets that could inform tailored treatment strategies. Participants were followed for at least 18 months, and the effectiveness of PGT was evaluated against standard treatments and non-guided targeted therapies.

The study found that two thirds of the participants were given PGT recommendations and about a third received the proposed treatments. The effectiveness of PGT was notably superior. PGT led to a significant improvement in two-year progression-free survival rates (26 per cent) compared to the standard care group (12 per cent) and the group receiving unguided targeted therapies (5 per cent). These results underscore the potential of precision medicine in identifying and applying more effective treatment modalities for paediatric cancer patients.

These findings suggest PGT can substantially enhance treatment outcomes for children with high-risk cancers. By adding comprehensive molecular profiling to treatment planning, PGT personalises patient care and could lead to better survival rates and quality of life. However, the study also recognises the need for broader clinical adoption and more extensive research to refine and validate the benefits of precision medicine in paediatric oncology.


Further information

Precision-guided treatment in high-risk pediatric cancers: Nature Medicine

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