- High vs low tumor mutation burden (TMB) is associated with significant improvement in PFS but not OS with programmed cell death protein 1 (PD-1)/programmed cell death ligand 1 (PD-L1) inhibitors vs chemotherapy.
- Patients with high TMB show significant PFS advantage with PD-1/PD-L1 inhibitors vs those with low TMB.
Why this matters
- TMB may be a preferable biomarker to select patients for PD-1/PD-L1 inhibitors.
- Meta-analysis of 8 studies including 2661 patients with cancer treated with PD-1/PD-L1 inhibitors or chemotherapy.
- Funding: National Natural Science Foundation of China; Natural Science Foundation of Guangdong Province, China.
- In the patients treated with PD-1/PD-L1 inhibitors vs chemotherapy, those with:
- high TMB had significant PFS advantage (pooled HR, 0.66; 95% CI, 0.50-0.88; P=.004)
- low TMB had no significant difference in PFS (pooled HR, 1.38; 95% CI, 0.82-2.31; P=.229).
- no significant difference in OS was observed in patients with high (pooled HR, 0.73; 95% CI, 0.50-1.08; P=.114) or low TMB (pooled HR, 1.00; 95% CI, 0.80-1.24; P=.970).
- High vs low TMB was associated with significant PFS benefit in patients treated with PD-1/PD-L1 inhibitors (HR, 0.47; 95% CI, 0.35-0.63; P=.000).
- Most of the included studies evaluated non-small cell lung cancer or bladder cancer, findings may not be generalizable to other cancers.