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Table 3 Predictive biomarkers for treatment efficacy of PD-1/PD-L1 targeting agents

From: Development and clinical applications of cancer immunotherapy against PD-1 signaling pathway

DrugSampleMarkerResultReference
PembrolizumabNSCLCPD-L1*↑: longer OS & PFSHerbst et al., 2016; Reck et al., 2016; Mok et al., 2019
PembrolizumabMetastatic colon cancerMMR*Deficiency: ORR = 40%, DCR = 90%Le et al., 2016
Pembrolizumab11 cancer typesMMR*Deficiency: ORR = 53%, DCR = 77%Le et al., 2017
NivolumabNSCLCTMB↑: longer PFSCarbone et al., 2017
Nivolumab+IpilimumabNSCLCTMB↑: longer OS & PFSHellmann et al., 2018
NivolumabmelanomaALC≥ 1000u/L: ↑ prognosisNakamura et al. 2016
NivolumabmelanomaANC< 4000u/L: ↑ prognosisNakamura et al. 2016
IpilimumabmelanomaNLR + LDHNLR > 2.2 & ↑LDH: ↓ RRBagley et al., 2017
PD-1 targeted therapyNSCLCKi67↑: positive outcomesKamphorst et al., 2017
PembrolizumabmelanomaTCR repertoire↓ diversity: positive clonal responsesTumeh, et al., 2014
NivolumabMetastatic melanomaPD-L1 + GZMA + HLA-A↑: better clinical outcomesHiroyuki et al., 2016
NivolumabMetastatic melanomaTCR repertoire↓ diversity: ↑ responsesHiroyuki et al., 2016; Sabrina et al., 2018
PD-1 targeted therapymelanomaRuminococcaceae family↑ alpha diversity & relative abundance: ↑ responsesGopalakrishnan et al., 2018
PD-1 targeted therapyLung & kidney cancersAkkermansia muciniphila↑ relative abundance: ↑ responsesRouty et al., 2018
  1. * PD-L1 and MMR are clinically applicable biomarkers
  2. NSCLC Non-small-cell lung carcinoma, OS Overall survival, PFS Progression free survival, ORR Overall response rate, DCR Disease control rate, TMB Tumor mutation burden, ALC Absolute lymphocyte count, NLR Neutrophil-to-lymphocyte ratio, LDH Lactate dehydrogenase, RR Response rate, TCR T-cell receptor