Background: The significance of tumor burden for survival is unknown for patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). The purpose of our study was to evaluate the prognostic impact of programmed death ligand-1 (PD-L1) and tumor burden score (TBS) in patients with R/M HNSCC. Patients and Methods: R/M HNSCC patients who were treated with cisplatin, 5-fluorouracil plus cetuximab (EPF) or pembrolizumab (PPF) as first-line treatment were included in our study. PD-L1 and TBS were estimated and correlated with treatment responses. Kaplan–Meier curves were plotted for outcomes estimation. Results: A total of 252 R/M HNSCC patients were included, with 126 high tumor burden (HTB) and 126 low tumor burden (LTB) patients. Median progression-free survival (PFS) was 7.1 months in LTB and 3.9 months in HTB (p< 0.001) and median overall survival (OS) was 14.2 months in LTB and 9.2 months in HTB (p= 0.001). Patients with LTB had better PFS and OS than those with HTB independent of PD-L1 status. Subgroup analysis showed HTB patients treated with EPF had better survival than those treated with PPF, regardless of PD-L1 expression. For LTB PD-L1 positive patients, there was a longer survival with PPF than EPF, while for LTB PD-L1 negative patients, survival was similar between PPF and EPF. Multivariate analysis exhibited that tumor burden was significantly correlated with OS. Conclusions: Tumor burden is significantly correlated with survival in patients with R/M HNSCC. PD-L1 and TBS should be taken into consideration to determine first-line treatment.
背景：对于复发或转移性头颈部鳞状细胞癌（R/M HNSCC）患者，肿瘤负荷对生存的影响尚不明确。本研究旨在评估程序性死亡配体-1（PD-L1）与肿瘤负荷评分（TBS）对R/M HNSCC患者的预后影响。患者与方法：研究纳入接受顺铂、5-氟尿嘧啶联合西妥昔单抗（EPF）或帕博利珠单抗（PPF）作为一线治疗的R/M HNSCC患者。评估PD-L1与TBS指标，并分析其与治疗反应的相关性。通过绘制Kaplan-Meier曲线进行结局评估。结果：共纳入252例R/M HNSCC患者，其中高肿瘤负荷（HTB）与低肿瘤负荷（LTB）患者各126例。LTB组中位无进展生存期（PFS）为7.1个月，HTB组为3.9个月（p<0.001）；LTB组中位总生存期（OS）为14.2个月，HTB组为9.2个月（p=0.001）。无论PD-L1状态如何，LTB患者的PFS与OS均优于HTB患者。亚组分析显示，无论PD-L1表达水平如何，接受EPF治疗的HTB患者生存期均优于接受PPF治疗者。对于PD-L1阳性的LTB患者，PPF治疗生存期长于EPF；而PD-L1阴性的LTB患者中，两种治疗方案生存期相近。多变量分析表明肿瘤负荷与OS显著相关。结论：肿瘤负荷与R/M HNSCC患者生存期显著相关。确定一线治疗方案时应综合考虑PD-L1与TBS指标。
肿瘤(癌症)患者之家