Precision-guided treatment in high-risk pediatric cancers
- Author(s)
- Lau, LMS; Khuong-Quang, DA; Mayoh, C; Wong, M; Barahona, P; Ajuyah, P; Senapati, A; Nagabushan, S; Sherstyuk, A; Altekoester, AK; Fuentes-Bolanos, NA; Yeung, V; Sullivan, A; Omer, N; Diamond, Y; Jessop, S; Battaglia, L; Zhukova, N; Cui, L; Lin, A; Gifford, AJ; Fleuren, EDG; Dalla-Pozza, L; Moore, AS; Khaw, SL; Eisenstat, DD; Gottardo, NG; Wood, PJ; Tapp, H; Alvaro, F; McCowage, G; Nicholls, W; Hansford, JR; Manoharan, N; Kotecha, RS; Mateos, MK; Lock, RB; Tyrrell, V; Haber, M; Trahair, TN; Cowley, MJ; Ekert, PG; Marshall, GM; Ziegler, DS;
- Journal Title
- Nature Medicine
- Publication Type
- Research article
- 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.
- Publisher
- Springer Nature
- Department(s)
- Laboratory Research
- Publisher's Version
- https://doi.org/10.1038/s41591-024-03044-0
- Open Access at Publisher's Site
https://doi.org/10.1038/s41591-024-03044-0- Terms of Use/Rights Notice
- Refer to copyright notice on published article.
Creation Date: 2024-08-06 05:29:19
Last Modified: 2024-08-06 05:31:19