What is the role of checkpoint inhibitor therapy in the treatment of glioblastoma multiforme (GBM)?

Updated: Jul 28, 2021
  • Author: Jeffrey N Bruce, MD; Chief Editor: Herbert H Engelhard, III, MD, PhD, FACS, FAANS  more...
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In preclinical studies, inhibitors of programmed cell death-1 (PD-1)/programmed cell death ligand-1 (PD-L1) have shown some potential for treatment of glioblastoma. In clinical studies, however, anti-PD-1/PD-L1 monotherapy has shown few satisfactory results. Efficacy may be better in certain patient subgroups (eg, those with higher tumor mutation burden, higher microsatellite instability, mismatch repair system deficiency,  germline POLE mutation). Neoadjuvant checkpoint inhibitor therapy has shown promise. [122]

CheckMate 143, a phase 3 randomized clinical trial, compared overall survival (OS) in 369 patients with recurrent glioblastoma treated with either bevacizumab or the  (PD-L1) inhibitor nivolumab. The 12-month OS was 42% in both groups. The objective response rate was higher with bevacizumab than with nivolumab (23.1% versus 7.8%, respectively). The rates of grade 3/4 treatment-related adverse events were similar in the two groups. [123]

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