Precision-guided treatment in high-risk pediatric cancers

Loretta M.S. Lau, Dong Anh Khuong-Quang, Chelsea Mayoh, Marie Wong, Paulette Barahona, Pamela Ajuyah, Akanksha Senapati, Sumanth Nagabushan, Alexandra Sherstyuk, Ann Kristin Altekoester, Noemi A. Fuentes-Bolanos, Veronica Yeung, Ashleigh Sullivan, Natacha Omer, Yonatan Diamond, Sophie Jessop, Lauren Battaglia, Nataliya Zhukova, Louise Cui, Angela LinAndrew J. Gifford, Emmy D.G. Fleuren, Luciano Dalla-Pozza, Andrew S. Moore, Seong Lin Khaw, David D. Eisenstat, Nicholas G. Gottardo, Paul J. Wood, Heather Tapp, Frank Alvaro, Geoffrey McCowage, Wayne Nicholls, Jordan R. Hansford, Neevika Manoharan, Rishi S. Kotecha, Marion K. Mateos, Richard B. Lock, Vanessa Tyrrell, Michelle Haber, Toby N. Trahair, Mark J. Cowley, Paul G. Ekert, Glenn M. Marshall, David S. Ziegler

Research output: Contribution to journalArticlepeer-review

1 Citation (Scopus)


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.

Original languageEnglish
Number of pages26
JournalNature Medicine
Publication statusE-pub ahead of print - 6 Jun 2024


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