Precision-Guided Treatment Improves Outcomes for High-Risk Pediatric Cancer
By Elana Gotkine HealthDay Reporter
FRIDAY, June 28, 2024 -- Precision-guided treatment improves outcomes for children with high-risk cancer, according to a study published online June 6 in Nature Medicine.
Loretta M.S. Lau, M.B.B.S., Ph.D., from the Children's Cancer Institute at the University of New South Wales Sydney, and colleagues reported data from 384 patients with high-risk pediatric cancer with at least 18 months of follow-up on the ZERO Childhood Cancer Precision Medicine Program PRecISion Medicine for Children with Cancer trial to examine which patients will benefit most from PGT. Overall, 67 and 29 percent of children received PGT recommendations and received a recommended treatment, respectively.
The researchers found that the objective response rate was 36 percent with PGT, and two-year progression-free survival was improved with PGT versus standard of care or targeted agents not guided by other molecular findings (26 percent versus 12 and 5.2 percent, respectively). Based on tier 1 evidence, the greatest clinical benefit was seen for PGT targeting fusions or commenced before disease progression.
"This study demonstrates that children with high-risk cancers benefit from PGT identified by comprehensive molecular profiling," the authors write. "Treatment strategies should focus on the identification of drivers and early treatment of patients with highly targetable molecular drivers."
Several authors disclosed ties to the pharmaceutical industry.
Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions.

© 2025 HealthDay. All rights reserved.
Posted June 2024
Read this next
Advanced Biological Age Linked to Increased Risk for Incident Dementia
FRIDAY, May 2, 2025 -- Advanced biological age (BA) may be a risk factor for incident dementia, according to a study published online April 30 in Neurology. Yacong Bo, Ph.D...
Exposure to Smoke Pollution Ups Risk for Hospitalization for Respiratory Disease
FRIDAY, May 2, 2025 -- For older adults in the Western United States, exposure to high levels of smoke pollution is associated with an increase in hospitalizations for respiratory...
Exercise Helps Reduce Side Effects From Cancer Treatment
THURSDAY, May 1, 2025 -- Exercise mitigates adverse outcomes associated with cancer and its treatments, according to a review published online April 29 in the British Journal...
More news resources
- FDA Medwatch Drug Alerts
- Daily MedNews
- News for Health Professionals
- New Drug Approvals
- New Drug Applications
- Drug Shortages
- Clinical Trial Results
- Generic Drug Approvals
Subscribe to our newsletter
Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.