Table 1.
Summary of retrospective and prospective studies of ICI for treatment of BM due to NSCLC.
| Study Design | Treatment | Patients with BM* | Patients on steroids | Patients with local therapy within 2 months | Patients with PD-L1 ⩾1% a | ORR (%) | iORR (%) | PFS (months, 95% CI) | iPFS (months, 95% CI) | OS (months, 95% CI) | Ref |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Retrospective | Nivolumab | 100% (N = 5) | 0% | 0% | – | 40 | 40 | – | – | – | Li et al. 55 |
| Retrospective | Nivolumab | 40% (N = 19) | – | 79% (N = 15) | – | 11 | 0 † | 2. 0 |
– | NR | Abdulhaleem et al. 56 |
| Retrospective | Nivolumab | 100% (N = 43) | – | 16% (N = 7) | – | 14 | 11 | 2.8 (1.8–5.3) | 3.9 (2.8–11.1) | NR | Scoccianti et al. 57 |
| Retrospective | Any anti-PD-(L)1 | 25% (N = 255) | 27.4% (N = 69) | – | 62% (N = 51) | 21 | 27 | 1.7 (1.5–2.1) | – | 8.6 (6.8–12.0) | Vogelbaum et al. 15 |
| Prospective ‡ | Pembrolizumab | 100% (N = 42) | – | – | 88.1% (N = 37) | 19 | 30 | 1.9 (1.8–3.7) | 2.3 (1.9–NR) | 9.9 (7.5–29.8) | Borghaei et al. 53 , Hellmann et al. 54 |
| Retrospective | Pembrolizumab ± chemotherapy | 22% (N = 131) | 22% (N = 29) | 48% (N = 63) | – | – | 31 ⁑ | 9.2 | – | 18.3 | Sun et al. 58 |
| Prospective⁂ | Atezolizumab + Chemotherapy | 100% (N = 40) | 55% (N = 22) | 0% | 50% (N = 20) | 40 | 40 | 8.9 (6.7–13.8) | 6.9 (4.7–11.9) | 13.6 (9.7–NR) | Powel et al. 59 , Paz-Ares et al. 60 |
N represents the total number of patients with BM.
Denominator includes only patients with known PD-L1 status.
CNS response only evaluable for 12 of the 19 patients with BM.
Response rates and survival are listed only for patients in cohort 1, which included all 37 PD-L1-positive patients.
Denominator includes only patients treated with pembrolizumab alone (N = 13).
Global ORR and PFS not reported. ORR and PFS describe extracranial ORR and PFS.
OS, PFS, iPFS, ORR, and iORR apply only to patients with BM unless otherwise specified.
BM, brain metastases; iORR, intracranial ORR; iPFS, intracranial PFS; NR, not reached; ORR, objective response rate; OS, overall survival; PD-L1, programmed death 1 ligand-1; PFS, progression-free survival.