Predictive value of EGFR amplification and EGFRvIII mutation in EGFR-targeted therapy for recurrent glioblastoma: a systematic review.

TL;DR

EGFR amplification and EGFRvIII mutation lack reliable predictive value for EGFR-targeted therapy response in recurrent glioblastoma, based on systematic review of 12 heterogeneous studies showing inconsistent survival benefits regardless of molecular status.

Key findings

• EGFR amplification and EGFRvIII mutation are common alterations in glioblastoma but showed no consistent survival benefit in EGFR-targeted therapy across included studies
• Median overall survival ranged 5.7–10.3 months and progression-free survival 1.7–6.0 months with no clear stratification by EGFR status
• Evidence base limited by heterogeneous single-arm study designs with variable assessment methods (FISH, qPCR, RT-PCR, NGS)
• These biomarkers are not recommended to guide off-trial treatment decisions in recurrent glioblastoma

Why this matters at the bedside

Clarifies that EGFR amplification and EGFRvIII status should not be used as standalone predictive markers to select patients for EGFR-targeted therapy in recurrent GBM, potentially redirecting clinical decision-making away from biomarker-driven selection in this population.