Background/Objectives: The combination of anti-programmed death-1 (PD-1) inhibitors and chemotherapy is the standard first-line treatment for unresectable or metastatic esophageal squamous cell carcinoma (ESCC). However, real-world data remain limited, particularly regarding prognostic biomarkers. Methods: This multi-institutional retrospective study analyzed patients with metastatic or unresectable ESCC who received first-line pembrolizumab or nivolumab plus fluoropyrimidine and platinum-based chemotherapy. Treatment regimens mirrored those in KEYNOTE-590 and CheckMate 648. Efficacy, safety, and prognostic factors were assessed. Prognostic factors were identified using multivariable Cox regression, and a point-based risk scoring system was developed. Results: Among 87 patients, the objective response rate was 48.3%, and the disease control rate was 77.0%. Median progression-free survival (PFS) was 5.6 months (95% CI, 4.5–8.7), and the median overall survival (OS) was 13.1 months (95% CI, 10.6–not reached). Grade 3–4 treatment-related adverse events occurred in 51.7% of patients. Eastern Cooperative Oncology Group (ECOG) performance status ≥ 2, elevated C-reactive protein, and lower programmed death-ligand 1 (PD-L1) combined positive score (CPS) were independently associated with worse PFS and OS. A prognostic risk score ranging from 0 to 5 based on these factors stratified patients into four prognostic groups with distinct survival outcomes. Median PFS ranged from not reached in the low-risk group to 2.1 months in the high-risk group. Stratifying PD-L1 CPS into three levels (<10, 10–49, ≥50) revealed a graded association between CPS and treatment outcomes, supporting the need for more nuanced PD-L1 evaluation beyond binary classification. Conclusions: First-line anti-PD-1 therapy combined with chemotherapy demonstrated favorable real-world outcomes in ESCC. The proposed prognostic scoring system may help personalize treatment strategies.
背景/目的：抗程序性死亡受体-1（PD-1）抑制剂联合化疗是不可切除或转移性食管鳞状细胞癌（ESCC）的标准一线治疗方案。然而，真实世界数据仍较为有限，特别是在预后生物标志物方面。方法：本项多中心回顾性研究分析了接受一线帕博利珠单抗或纳武利尤单抗联合氟尿嘧啶及铂类化疗的转移性或不可切除ESCC患者。治疗方案参照KEYNOTE-590和CheckMate 648研究设计。评估疗效、安全性及预后因素，通过多变量Cox回归识别预后因子并建立基于评分的风险分层系统。结果：在87例患者中，客观缓解率为48.3%，疾病控制率为77.0%。中位无进展生存期（PFS）为5.6个月（95% CI，4.5–8.7），中位总生存期（OS）为13.1个月（95% CI，10.6–未达到）。51.7%的患者发生3-4级治疗相关不良事件。东部肿瘤协作组（ECOG）体能状态评分≥2分、C反应蛋白升高及较低的程序性死亡配体-1（PD-L1）联合阳性分数（CPS）与较差的PFS和OS独立相关。基于这些因素构建的0-5分预后风险评分系统将患者分为四个预后组，各组生存结局差异显著：低危组中位PFS未达到，而高危组仅为2.1个月。将PD-L1 CPS分为三级（<10、10–49、≥50）的分析显示，CPS与治疗结局存在梯度关联，提示需要超越二元分类的更精细化PD-L1评估。结论：一线抗PD-1联合化疗在ESCC中显示出良好的真实世界疗效。本研究提出的预后评分系统可能有助于个体化治疗策略的制定。
肿瘤(癌症)患者之家