在 3 期 KEYNOTE-040 研究中，与化疗相比，pembrolizumab 可延长既往治疗过的复发性或转移性 (R/M) HNSCC 的 OS。我们根据疾病复发模式进行事后亚组分析：仅复发性、复发性和转移性（复发性转移性）以及仅转移性 HNSCC。患者患有在含铂治疗 R/M 疾病期间或之后进展的 HNSCC，或者患有先前针对局部晚期疾病的含铂确定性治疗后 3-6 个月内复发或进展。患者被随机分配 (1:1) 接受派姆单抗 200 mg Q3W 或研究者选择的护理标准 (SOC)：甲氨蝶呤、多西他赛或西妥昔单抗。结果包括 OS、PFS、ORR 和 DOR。数据截止日期为 2017 年 5 月 15 日。仅复发亚组中有 125 名患者（pembrolizumab，53；SOC，72），复发转移亚组有 204 名患者（pembrolizumab，108；SOC，96），复发转移亚组有 166 名患者。仅转移亚组（pembrolizumab，86；SOC，80）。派姆单抗与 SOC 的死亡风险比 (95% CI) 在仅复发性病例中为 0.83 (0.55-1.25)，在复发性转移性病例中为 0.78 (0.58-1.06)，在转移性转移性病例中为 0.74 (0.52-1.05)。仅子组。所有亚组的治疗组之间的 PFS 相似。在仅复发亚组中，派姆单抗的 ORR 为 22.6%，而 SOC 为 16.7%；在复发转移亚组中，分别为 10.2% 和 6.3%；在仅转移亚组中，分别为 15.1% 和 8.8%。与 SOC 相比，派姆单抗在所有亚组中的 DOR 均更长。与 SOC 相比，派姆单抗在所有亚组中提供了更长的 OS 和持久缓解，这表明既往接受过治疗的 R/M HNSCC 患者可从派姆单抗中获益，无论复发模式如何。版权所有 © 2023。由 Elsevier 出版有限公司

In the phase 3 KEYNOTE-040 study, pembrolizumab prolonged OS versus chemotherapy in previously treated recurrent or metastatic (R/M) HNSCC. We present a post hoc subgroup analysis by disease recurrence pattern: recurrent-only, recurrent and metastatic (recurrent-metastatic), and metastatic-only HNSCC.Patients had HNSCC that progressed during or after platinum-containing treatment for R/M disease or had recurrence or progression within 3-6 months of previous platinum-containing definitive therapy for locally advanced disease. Patients were randomly assigned (1:1) to pembrolizumab 200 mg Q3W or investigator's choice of standards of care (SOC): methotrexate, docetaxel, or cetuximab. Outcomes included OS, PFS, ORR, and DOR. The data cutoff was May 15, 2017.There were 125 patients (pembrolizumab, 53; SOC, 72) in the recurrent-only subgroup, 204 in the recurrent-metastatic subgroup (pembrolizumab, 108; SOC, 96), and 166 in the metastatic-only subgroup (pembrolizumab, 86; SOC, 80). The hazard ratio (95% CI) for death for pembrolizumab versus SOC was 0.83 (0.55-1.25) in the recurrent-only, 0.78 (0.58-1.06) in the recurrent-metastatic, and 0.74 (0.52-1.05) in the metastatic-only subgroups. PFS was similar between treatment arms in all subgroups. ORR was 22.6% for pembrolizumab versus 16.7% for SOC in the recurrent-only, 10.2% versus 6.3% in the recurrent-metastatic, and 15.1% versus 8.8% in the metastatic-only subgroups. DOR was numerically longer with pembrolizumab in all subgroups.Pembrolizumab provided numerically longer OS and durable responses in all subgroups compared with SOC, suggesting that patients with previously treated R/M HNSCC benefit from pembrolizumab regardless of recurrence pattern.Copyright © 2023. Published by Elsevier Ltd.