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Precision-guided treatment in high-risk pediatric cancers
Loretta M S Lau1,2,3, Dong-Anh Khuong-Quang4,5, Chelsea Mayoh1,2
1Children's Cancer Institute, Lowy Cancer Research Centre, UNSW Sydney, Sydney, New South Wales, Australia.
Nature Medicine
|June 6, 2024
Summary
Precision-guided treatment (PGT) significantly improves outcomes for children with high-risk cancers. Comprehensive molecular profiling guides PGT, enhancing survival rates and treatment responses in pediatric cancer patients.
Area of Science:
- Pediatric Oncology
- Precision Medicine
- Cancer Genomics
Background:
- Precision medicine offers novel treatment strategies for pediatric cancers.
- Identifying patients who benefit most from precision-guided treatment (PGT) remains a challenge.
Purpose of the Study:
- To evaluate the effectiveness of PGT in children with high-risk cancer.
- To identify factors associated with clinical benefit from PGT.
Main Methods:
- Analysis of consecutive data from 384 high-risk pediatric cancer patients in the ZERO Childhood Cancer PRISM trial.
- Assessment of PGT recommendations and treatments received.
- Comparison of outcomes (objective response rate, progression-free survival) between PGT and standard care or non-molecularly guided targeted agents.
Main Results:
- 256 patients (67%) received PGT recommendations; 110 (29%) received a recommended treatment.
- PGT achieved a 36% objective response rate.
- PGT improved 2-year progression-free survival compared to standard of care (26% vs. 12%) and non-molecularly guided targeted agents (26% vs. 5.2%).
- PGT based on tier 1 evidence, targeting fusions, or initiated before progression showed the greatest benefit.
Conclusions:
- Comprehensive molecular profiling-informed PGT significantly improves outcomes for children with high-risk cancers.
- PGT demonstrates superior efficacy compared to conventional treatments and non-guided targeted therapies.
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