ArticleNature medicine2024
Precision-guided treatment in high-risk pediatric cancers.
Article in Nature medicine, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03336931 (A Multicenter Prospective Study of the Feasibility and Clinical Value of a Diagnostic Service for Identifying Therapeutic Targets and Recommending Personalised Treatment for Children and Adolescents With High-risk Cancer), which is not on this map. Cited by 49 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Multicenter Prospective Study of the Feasibility and Clinical Value of a Diagnostic Service for Identifying Therapeutic Targets and Recommending Personalised Treatment for Children and Adolescents With High-risk Cancer
Who cites it
49 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Molecular Tumor Boards clinical impact on patient care and structural features: A systematic review and meta-analysis.PLoS medicine · 2026Pooled it
- Factors that influence the uptake of precision-guided treatment recommendations in paediatric cancer: a systematic review.British journal of cancer · 2026Pooled it
- Tumor-derived cell-free DNA detected in cerebrospinal fluid enables minimally invasive profiling of pediatric brain tumors.The Journal of clinical investigation · 2026Trial
- Paediatric biobanking in the era of precision medicine: ethical, regulatory, and scientific challenges from childhood to adulthood.European journal of pediatrics · 2026Review
- The ITCC-P4 PDX Platform Enables Preclinical Testing of Pediatric Cancers.Cancer research · 2026Article
- First-year results of the International Leukemia/ Lymphoma Target Board for pediatric relapsed and refractory hematological malignancies.Haematologica · 2026Article
- High clinical utility of comprehensive multi-omic molecular profiling of rare and hard-to-diagnose pediatric tumors.Research square · 2026Article
- Establishing a Multicenter Personalized Medicine Program in Childhood, Adolescent, and Young Adult Cancer in Spain: The SEHOP-PENCIL Project.JCO precision oncology · 2026Article
- The myeloid cell leukaemia-1 inhibitor MIK665 is a potent therapy in preclinical models of paediatric acute myeloid leukaemia.British journal of haematology · 2026Article
- High clinical utility of comprehensive multi-omic molecular profiling of rare and hard-to-diagnose pediatric tumors.medRxiv : the preprint server for health sciences · 2026Article
- Global, regional, and national trends in 34 cancers through a novel burden-quality-of-care framework: a cross-sectional study.International journal of surgery (London, England) · 2026Article
- Precision-guided therapy in H3K27-altered diffuse midline glioma.Nature communications · 2026Observational
- Advances in targeted therapies for pediatric tumors.Acta pharmacologica Sinica · 2026Article
- Integrated Germline and Somatic Molecular Profiling to Detect Cancer Predisposition Has a High Clinical Impact in Poor-Prognosis Pediatric Cancer.Clinical cancer research : an official journal of the American Association for Cancer Research · 2026Article
- Mapping Genomic Heterogeneity in Pediatric and Adolescent-Young Adult Sarcomas: Insights from the Italian SAR-GEN2016 and SAR-GEN_ITA Prospective Multicenter Trials.Cancer research communications · 2026Article
- Comprehensive Multiplatform Tyrosine Kinase Profiling Reveals Novel Actionable FGFR Aberrations across Sarcomas Affecting the Young.Molecular cancer therapeutics · 2026Article
- OnCorr: A pan-cancer mRNA-protein correlation tool for precision oncology.NPJ precision oncology · 2026Article
- Genomic Profiling of Pediatric Solid Tumors With a Dual DNA/RNA Panel: JCCG-TOP2 Study.Cancer science · 2026Observational
- The functional imperative in high-grade glioma.Experimental & molecular medicine · 2026Review
- The Knowledge Connector decision support system for multiomics-based precision oncology.Nature communications · 2026Article
Corrections and comments
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Authors and funding
44 authors.
Funding
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.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.