Clinical outcomes of immune checkpoint inhibitor-containing chemoimmunotherapy versus chemotherapy alone in locoregionally recurrent nasopharyngeal carcinoma: a retrospective cohort study

To compare progression-free survival (PFS) and overall survival (OS) between patients receiving immune checkpoint inhibitor (ICI)-containing chemoimmunotherapy and chemotherapy alone as first-line systemic treatment for locoregionally recurrent nasopharyngeal carcinoma (LRNPC). We retrospectively included patients diagnosed with LRNPC between July 2018 and June 2024. Chi-square or Fisher’s exact test, Wilcoxon rank-sum test, and Kaplan-Meier method were used for statistical analyses. Propensity score overlap weighting was used as the primary adjusted analysis, and inverse probability of treatment weighting was performed as a sensitivity analysis. A total of 37 patients were included in this study, with 20 patients (54.1%) presenting with local recurrence, 9 (24.3%) with regional recurrence, and 8 (21.6%) with locoregional recurrence. With a median follow-up of 46.5 months, 23 patients (62.2%) experienced disease progression. The objective response rate was 66.7% among patients who received ICIs, compared with 61.5% among those who did not ( P = 1.000). The 3-year PFS was 56.0% and 23.1% in the chemoimmunotherapy and chemotherapy alone groups, respectively ( P = 0.012). The 3-year OS was 82.7% and 36.9% in the chemoimmunotherapy and chemotherapy alone groups, respectively ( P = 0.006). In the primary overlap-weighted analysis, ICI-containing therapy was associated with a lower risk of death (hazard ratio [HR], 0.198; 95% confidence interval [CI], 0.059–0.665; P = 0.009), whereas the association with PFS did not reach statistical significance (HR, 0.388; 95% CI, 0.137–1.101; P = 0.075). Among the 15 deaths, 14 were attributed to tumor progression, including two cases of fatal epistaxis (13.3% of all deaths). For first-line treatment of LRNPC, ICI-containing chemoimmunotherapy brings significantly longer overall survival compared with chemotherapy alone. These findings are hypothesis-generating and require further validation in prospective clinical trials.