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. 2022 May 22;27(7):538–547. doi: 10.1093/oncolo/oyac064

Table 1.

Efficacy of immune checkpoint inhibitors in melanoma and non–small cell lung cancer in patients with brain metastases.

Identifier/trial Agents Phase Patient number/cohorts Outcomes
Melanoma
NCT01449279/ CA184-04569 Ipilimumab Pilot study N = 185 1-year OS rate in patients with stable BM=31%
NCT0062376670 Ipilimumab 2 N = 72
Cohort A = 51 patients (asymptomatic, not receiving steroids)
Cohort B = 21patients (symptomatic, receiving steroids)
Cohort A IRR = 24%
CohortBIRR = 10%
NCT01654692/ NIBIT-M171 Ipilimumab,
fotemustine
2 N = 20 IRR = 50%
NCT023742472 Ipilimumab, nivolumab 2 N = 79
Cohort A = 36 patients (asymptomatic and untreated ipilimumab/nivolumab)
Cohort B = 27 patients (asymptomatic and untreated, nivolumab)
Cohort C = 16 patients (symptomatic and treated, nivolumab)

Cohort A IRR= 46%
Cohort B IRR= 20%
Cohort C IRR= 6%
NCT02320058/CheckMate 20411 Nivolumab, ipilimumab 2 N = 94 IRR = 57%
NCT0208507073 Pembrolizumab 2 N = 23 IRR=26%
Non–small cell lung cancer
NCT01454102/Checkmate 012 (Arm M)74 Nivolumab, ipilimumab 1 N = 12 IRR = 16.7%
NCT01721759 CheckMate 063CheckMate 017 CheckMate 05775 Nivolumab Pooled analysis
2
3
3
N = 46 IRR =33% (stable disease)
NCT02008227/OAK study subgroup76 Atezolizumab, docetaxel I3 N = 61 Atezolizumab with greater reduction in new BM, vs docetaxel
NCT0208507077 Pembrolizumab 2 N = 39
Cohort A = 34 (PD-L1 positive)
Cohort B = 5 (PD-L1 negative)
Cohort A IRR = 29%
Cohort B IRR = 0%
Nivolumab expanded access program78 Nivolumab Pilot study N = 409 ORR = 17%
DCR =39% in CNS metastasis patients (overall response, no CNS disease)
Multiple tumor types
NCT0208507079 Pembrolizumab 2 N = 36
(18 with melanoma18 with NSCLC)
IRR melanoma = 22%
IRR NSCLC = 33%

Abbreviations: BM, brain metastases; CNS, central nervous system; DCR, disease control rate (complete response = partial response + stable disease); IRR, intracranial response rate; NSCLC, non–small cell lung cancer; ORR, overall response rate; OS, overall survival.