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Precision-guided treatment in high-risk pediatric cancers.


ABSTRACT: Recent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment (PGT). Here we report consecutive data from 384 patients with high-risk pediatric cancer (with an expected cure rate of less than 30%) who had at least 18 months of follow-up on the ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial. A total of 256 (67%) patients received PGT recommendations and 110 (29%) received a recommended treatment. PGT resulted in a 36% objective response rate and improved 2-year progression-free survival compared with standard of care (26% versus 12%; P = 0.049) or targeted agents not guided by molecular findings (26% versus 5.2%; P = 0.003). PGT based on tier 1 evidence, PGT targeting fusions or commenced before disease progression had the greatest clinical benefit. Our data show that PGT informed by comprehensive molecular profiling significantly improves outcomes for children with high-risk cancers. ClinicalTrials.gov registration: NCT03336931.

SUBMITTER: Lau LMS 

PROVIDER: S-EPMC11271405 | biostudies-literature | 2024 Jul

REPOSITORIES: biostudies-literature

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Precision-guided treatment in high-risk pediatric cancers.

Lau Loretta M S LMS   Khuong-Quang Dong-Anh DA   Mayoh Chelsea C   Wong Marie M   Barahona Paulette P   Ajuyah Pamela P   Senapati Akanksha A   Nagabushan Sumanth S   Sherstyuk Alexandra A   Altekoester Ann-Kristin AK   Fuentes-Bolanos Noemi A NA   Yeung Veronica V   Sullivan Ashleigh A   Omer Natacha N   Diamond Yonatan Y   Jessop Sophie S   Battaglia Lauren L   Zhukova Nataliya N   Cui Louise L   Lin Angela A   Gifford Andrew J AJ   Fleuren Emmy D G EDG   Dalla-Pozza Luciano L   Moore Andrew S AS   Khaw Seong-Lin SL   Eisenstat David D DD   Gottardo Nicholas G NG   Wood Paul J PJ   Tapp Heather H   Alvaro Frank F   McCowage Geoffrey G   Nicholls Wayne W   Hansford Jordan R JR   Manoharan Neevika N   Kotecha Rishi S RS   Mateos Marion K MK   Lock Richard B RB   Tyrrell Vanessa V   Haber Michelle M   Trahair Toby N TN   Cowley Mark J MJ   Ekert Paul G PG   Marshall Glenn M GM   Ziegler David S DS  

Nature medicine 20240606 7


Recent research showed that precision medicine can identify new treatment strategies for patients with childhood cancers. However, it is unclear which patients will benefit most from precision-guided treatment (PGT). Here we report consecutive data from 384 patients with high-risk pediatric cancer (with an expected cure rate of less than 30%) who had at least 18 months of follow-up on the ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer (PRISM) trial.  ...[more]

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