Precision-Guided Treatment Improves Outcomes for High-Risk Pediatric Cancer

Improvement in two-year progression-free survival seen with PGT versus standard of care or targeted agents not guided by molecular findings
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Medically Reviewed By:
Mark Arredondo, M.D.

FRIDAY, June 28, 2024 (HealthDay News) -- 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.

Abstract/Full Text

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