Table 1. Summary of characteristics of publications reporting efficacy data.
| Publication | Lung cancer type | TMB threshold values | Agent/Setting | TMB-H patients/total patients assessed for TMB (n/N) |
|---|---|---|---|---|
| Carbone 2017 [10] | NSCLC | TMB-L: < 100 mutations TMB-I: 100–242 mutations TMB-H: > 242 mutations | Nivolumab/1L Platinum-based chemotherapy | 47/158 60/154 |
| Choi 2017 [27] | NSCLC | NR | NR | NR/108 |
| Davis 2017 [28] | NSCLC | TMB-L: < 15 mut/Mb TMB-H: ≥ 15 mut/Mb | PD-1/PD-L1 inhibitors | NR/35 |
| Gettinger 2017 [29] | NSCLC | NR | PD-1/PD-L1 inhibitors | NR/45 |
| Goodman 2017 [16] | NSCLC | TMB-L: 1–5 mut/Mb TMB-I: 6–19 mut/Mb TMB-H: ≥ 20 mut/Mb | PD-1/PD-L1 inhibitors | 3/36 |
| Haratani 2017 [31] | NSCLC | NR | Nivolumab | NR/9 |
| Hellmann 2017 [34] | NSCLC | TMB-L: Below 85th percentile TMB-H: Above 85th percentile | PD-L1 inhibitors +/- anti-CTLA-4 therapy | NR/437 |
| Hellmann 2018 [33] | NSCLC | TMB-L: < 10 mut/Mb TMB-H: ≥ 10 mut/Mb | Nivolumab + ipilimumab Chemotherapy | 139/330 160/349 |
| Hellmann 2018 [32] | SCLC | TMB-L: < 143 mutations TMB-I: 143–247 mutations TMB-H: > 247 mutations | Nivolumab Nivolumab + ipilimumab | 47/133 26/78 |
| Hu 2018 [35] | NSCLC | TMB-H: ≥ 20 mut/Mb | PD-1/PD-L1 inhibitors | 9/NR |
| Kowanetz 2017 [30] | NSCLC | TMB-L: Below 50th percentile TMB-H: Above 50th percentile | Atezolizumab/1L Atezolizumab/2L | NR/102 NR/371 |
| Mahadevan 2017 [36] | NSCLC (n = 80) and SCLC (n = 5) | TMB-L: Below 50th percentile TMB-H: Above 50th percentile | PD-1 (n = 82)/ PD-L1 (n = 5) inhibitors/ other agents (n = 7) | NR/94 |
| Park 2017 [37] | NSCLC | TMB-L: 1–5 mut/Mb TMB-I: 6–19 mut/Mb TMB-H: > 20 mut/Mb | Nivolumab | NR/36 |
| Patel 2017 [38] | NSCLC | NR | Immunotherapy | NR/50 |
| Rizvi 2015 [1] | NSCLC | TMB-L: Below 50th percentile TMB-H: Above 50th percentile | Pembrolizumab (cohort 1) Pembrolizumab (cohort 2) | NR/16 NR/18 |
| Ross 2017 [45] | NSCLC | NR | Immune checkpoint inhibitors | 545/3758 |
| Roszik 2016 [39] | NSCLC | TMB-L: < 100 mutations TMB-H: ≥ 100 mutations | Pembrolizumab | 21/29 |
| Rozenblum 2017 [40] | NSCLC | NR | Pembrolizumab and nivolumab | NR/18 |
| Singal 2017 [41] | NSCLC | TMB-L: 1–5 mut/Mb TMB-I: 6–19 mut/Mb TMB-H: ≥ 20 mut/Mb | Nivolumab | NR/444 |
| Wang 2017 [42] | NSCLC | TMB-L: Below 50th percentile TMB-H: Above 50th percentile | NR | NR/98 |
| Xiao 2016 [43] | NSCLC | TMB-L: ≤ 4 mutations TMB-H: > 4 mutations | NR | 47/335 |
| Yaghmour 2016 [44] | NSCLC | TMB-L: Below 80th percentile TMB-H: Above 80th percentile | Nivolumab, pembrolizumab, or ipilimumab | 3/23 |
Abbreviations: 1L, first-line; CTLA-4, cytotoxic T-lymphocyte-associated protein 4; mut/Mb, mutations per DNA megabase; NR, not reported; NSCLC, non-small cell lung cancer; PD-1, programmed cell death-1; PD-L1, programmed death ligand 1; SCLC, small cell lung cancer; TMB, tumor mutational burden; TMB-H, high TMB; TMB-I, intermediate TMB; TMB-L, low TMB.