Immune Checkpoint Inhibitors for Nasopharyngeal Carcinoma in a Real-world Setting in Japan

In Vivo. 2023 Mar-Apr;37(2):747-755. doi: 10.21873/invivo.13137.


Background/aim: The advent of immune checkpoint inhibitor (ICI) treatment has transformed the treatment of recurrent or metastatic head and neck cancer; however, nasopharyngeal carcinoma (NPC) has not been included in major phase III trials. The clinical outcomes of ICI for NPC in real-world practice remain to be fully elucidated.

Patients and methods: We retrospectively reviewed 23 patients with recurrent or metastatic NPC treated with nivolumab or pembrolizumab at 6 institutions from April 2017 to July 2021 and investigated the correlation of clinicopathological factors and immune-related adverse events with the effects of ICI therapy and the prognosis.

Results: The objective response rate was 39.1% and the disease control rate was 78.3%. The median progression-free survival was 16.8 months and overall survival has not been reached. As with other treatment procedures, the efficacy and the prognosis tended to be better in EBER-positive cases than in EBER-negative cases. The rate of significant immune-related adverse events that necessitated discontinuation of treatment was only 4.3%.

Conclusion: ICI monotherapy (e.g., nivolumab and pembrolizumab) was effective and tolerable for NPC in a real-world setting.

Keywords: Immune checkpoint inhibitor; nasopharyngeal carcinoma; nivolumab; pembrolizumab; real-world data.

MeSH terms

  • Humans
  • Immune Checkpoint Inhibitors* / adverse effects
  • Japan
  • Nasopharyngeal Carcinoma / drug therapy
  • Nasopharyngeal Neoplasms* / drug therapy
  • Nivolumab / adverse effects
  • Retrospective Studies


  • Immune Checkpoint Inhibitors
  • Nivolumab