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Treatment effect heterogeneity of radiotherapy in localized Ewing sarcoma: A secondary analysis of the EURO-E.W.I.N.G. 99 and Ewing 2008 trial - PubMed

3 hours ago
  • #radiotherapy
  • #Ewing sarcoma
  • #individualized treatment effects
  • Standard treatment for localized Ewing sarcoma includes chemotherapy and local therapy, but previous studies show no average benefit of combined local therapy (radiotherapy + surgery) versus surgery alone.
  • This secondary analysis of two trials used causal survival forests to predict individualized treatment effects (ITEs) for 5-year event-free survival comparing combined therapy to surgery alone.
  • 19.4% of patients derived clinically meaningful benefit (≥6-month increase in 5y EFS) from combined therapy, showing substantial heterogeneity in ITEs (range: -0.7 to 1.0 years).
  • Patients predicted to benefit least had increased hazards by 53% when receiving combined therapy, while those predicted to benefit most had reduced hazards by 40%.
  • Surgical margin status was not associated with treatment benefit in patients undergoing combined therapy, challenging current decision-making practices.
  • Simulation analysis indicated that applying the personalized treatment rule could improve event-free survival.