PD-1/PD-L1 inhibitors in recurrent or metastatic nasopharyngeal carcinoma: A systematic review and meta-analysis
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Updated: Mar 22, 2026

Intramucosal Inoculation of Squamous Cell Carcinoma Cells in Mice for Tumor Immune Profiling and Treatment Response Assessment
Published on: April 22, 2019
Weiliang Bai1, Shengqun Xu1, Lei Miao2
1Department of Otolaryngology Head and Neck Surgery, Shengjing Hospital of China Medical University, Shenyang, Liaoning, China.
Background:
Nasopharyngeal carcinoma (NPC) has a poor prognosis, largely due to immune escape. The programmed cell death protein 1 (PD-1) receptor and its ligand, PD-L1, play critical roles in this immune evasion. Consequently, blocking the PD-1/PD-L1 pathway with immune checkpoint inhibitors has become an established therapeutic strategy.
Methods:
We performed a systematic literature search of PubMed, Embase, and the Cochrane Library for studies published up to November 14, 2024, investigating PD-1/PD-L1 inhibitors in recurrent or metastatic NPC. The analyzed outcomes included progression-free survival (PFS), overall survival (OS), 1-year PFS rate, 1-year OS rate, objective response rate, disease control rate, and adverse events (AEs).
Results:
Nine studies (comprising 10 datasets with 883 NPC patients) were included, consisting of 2 randomized controlled trials (RCTs) and 7 single-arm studies. The pooled analysis demonstrated a median PFS of 4.21 months (95% confidence interval [CI]: 2.47-5.95; P = .000; I2 = 78.5%) and a median OS of 16.27 months (95% CI: 14.60-17.94; P = .000; I2 = 0%). The pooled 1-year PFS rate was 43% (95% CI: 14%-73%; P = .004; I2 = 99.1%), and the 1-year OS rate was 80% (95% CI: 70%-91%; P = .000; I2 = 82.0%). The objective response rate was 45% (95% CI: 25%-64%; P = .000; I2 = 96.8%), and the disease control rate was 65% (95% CI: 48%-82%; P = .000; I2 = 96.4%). The incidence of any-grade AEs was 90% (95% CI: 74%-99%; P = .000; I2 = 94.1%), while grade ≥3 AEs occurred in 34% of patients (95% CI: 12%-61%; P < .001; I2 = 95.2%). No significant publication bias was identified.
Conclusion:
This meta-analysis summarizes the efficacy and safety profile of PD-1/PD-L1 inhibitors in recurrent or metastatic NPC. The findings highlight the need for additional randomized controlled trials to further validate the role of these therapies and provide an updated reference for clinical practice.
Insights
This meta-analysis shows that programmed cell death protein 1 (PD-1)/PD-L1 inhibitors offer significant benefits for patients with recurrent or metastatic nasopharyngeal carcinoma (NPC). These therapies demonstrate promising survival rates and response rates, alongside manageable adverse events.
Area of Science:
- Oncology
- Immunotherapy
- Clinical Trials
Background:
- Nasopharyngeal carcinoma (NPC) presents a poor prognosis due to immune evasion.
- Programmed cell death protein 1 (PD-1) and its ligand PD-L1 are key mediators of immune escape in NPC.
- Immune checkpoint inhibitors targeting the PD-1/PD-L1 pathway are an established therapeutic strategy.
Purpose of the Study:
- To systematically evaluate the efficacy and safety of PD-1/PD-L1 inhibitors in patients with recurrent or metastatic NPC.
- To provide an updated reference for clinical practice and identify areas for future research.
Main Methods:
- A systematic literature search was conducted across PubMed, Embase, and Cochrane Library up to November 14, 2024.
- Included studies investigated PD-1/PD-L1 inhibitors in recurrent or metastatic NPC.
- Key outcomes analyzed included progression-free survival (PFS), overall survival (OS), response rates, and adverse events (AEs).
Main Results:
- Nine studies (10 datasets, 883 patients) were analyzed, including 2 RCTs and 7 single-arm studies.
- Median PFS was 4.21 months, median OS was 16.27 months, and 1-year OS rate was 80%.
- Objective response rate was 45%, disease control rate was 65%, with 90% experiencing any-grade AEs and 34% experiencing grade ≥3 AEs.
Conclusions:
- PD-1/PD-L1 inhibitors demonstrate significant efficacy in recurrent or metastatic NPC.
- The safety profile is characterized by a high incidence of any-grade AEs, with a lower rate of severe AEs.
- Further randomized controlled trials are needed to validate these findings and refine treatment strategies.

