Conclusions

• Using a large clinical genomic database with GBM subjected to comprehensive molecular profiling, we demonstrated that amplification of CDK4 and EGFR were associated with long-term and short-term responses to bevacizumab, respectively
• Investigation into the tumors not treated with bevacizumab suggests that CDK4 amplification may be a predictive marker for bevacizumab while EGFR amplification may be prognostic of poor survival
• SETD2 mutations and PIK3R1 mutations are suggested to be predictive of bevacizumab benefit and prognostic of poor survival, respectively
• This warrants further investigation in independent cohorts controlled for age and other prognostic factors. If confirmed, a genetic basis for treatment optimization may provide meaningful clinical outcomes