Precision-guided treatment in high-risk pediatric cancers
-
Published:2024-06-06
Issue:7
Volume:30
Page:1913-1922
-
ISSN:1078-8956
-
Container-title:Nature Medicine
-
language:en
-
Short-container-title:Nat Med
Author:
Lau Loretta M. S.ORCID, Khuong-Quang Dong-AnhORCID, Mayoh ChelseaORCID, Wong MarieORCID, Barahona Paulette, Ajuyah Pamela, Senapati Akanksha, Nagabushan SumanthORCID, Sherstyuk Alexandra, Altekoester Ann-Kristin, Fuentes-Bolanos Noemi A., Yeung VeronicaORCID, Sullivan Ashleigh, Omer NatachaORCID, Diamond Yonatan, Jessop Sophie, Battaglia Lauren, Zhukova NataliyaORCID, Cui LouiseORCID, Lin AngelaORCID, Gifford Andrew J., Fleuren Emmy D. G., Dalla-Pozza Luciano, Moore Andrew S.ORCID, Khaw Seong-Lin, Eisenstat David D.ORCID, Gottardo Nicholas G.ORCID, Wood Paul J., Tapp Heather, Alvaro Frank, McCowage Geoffrey, Nicholls WayneORCID, Hansford Jordan R.ORCID, Manoharan Neevika, Kotecha Rishi S.ORCID, Mateos Marion K.ORCID, Lock Richard B., Tyrrell VanessaORCID, Haber MichelleORCID, Trahair Toby N.ORCID, Cowley Mark J.ORCID, Ekert Paul G.ORCID, Marshall Glenn M., Ziegler David S.ORCID
Abstract
AbstractRecent 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
Funder
Department of Health | National Health and Medical Research Council Cancer Institute NSW
Publisher
Springer Science and Business Media LLC
Reference24 articles.
1. Wong, M. et al. Whole genome, transcriptome and methylome profiling enhances actionable target discovery in high-risk pediatric cancer. Nat. Med. 26, 1742–1753 (2020). 2. van Tilburg, C. M. et al. The pediatric precision oncology INFORM registry: clinical outcome and benefit for patients with very high-evidence targets. Cancer Discov. 11, 2764–2779 (2021). 3. Church, A. J. et al. Molecular profiling identifies targeted therapy opportunities in pediatric solid cancer. Nat. Med. 28, 1581–1589 (2022). 4. Berlanga, P. et al. The European MAPPYACTS trial: precision medicine program in pediatric and adolescent patients with recurrent malignancies. Cancer Discov. 12, 1266–1281 (2022). 5. Ecker, J. et al. Molecular diagnostics enables detection of actionable targets: the Pediatric Targeted Therapy 2.0 registry. Eur. J. Cancer 180, 71–84 (2023).
Cited by
3 articles.
订阅此论文施引文献
订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
|
|