III 期 KEYNOTE-913 研究旨在评估派姆单抗作为晚期默克尔细胞癌 (MCC) 患者一线治疗的疗效和安全性。目的是报告 KEYNOTE-913 初步分析的结果。复发性局部晚期或转移性 MCC 每 3 周静脉注射 200 毫克派姆单抗，最多 35 次治疗（约 2 年）。主要终点是根据实体瘤疗效评估标准 1.1 版 (RECIST v1.1) 通过盲法独立中央审查 (BICR) 得出的客观缓解率 (ORR)。次要终点是根据 BICR 的 RECIST v1.1 的缓解持续时间 (DOR) 和无进展生存期 (PFS)、总生存期 (OS) 以及安全性和耐受性。 55 名患者接受了派姆单抗治疗。从首次给药到数据截止（2024 年 2 月 15 日）的中位时间为 50.3 个月（范围 38.7-59.4）。 ORR 为 49%（95% 置信区间 [CI] 35-63），其中 12 例完全缓解，15 例部分缓解。中位 DOR 为 39.8 个月（范围 4.8-52.5），24 个月 DOR 率为 69%。中位 PFS 为 9.3 个月（95% CI 3-26），24 个月 PFS 率为 39%。中位 OS 为 24.3 个月（95% CI 12.4 为未达到），24 个月 OS 率为 51%。 38 名患者 (69%) 发生任何级别的治疗相关不良事件 (AE)； 13 名患者 (24%) 经历了 3-5 级 AE。最常见的治疗相关 AE 是疲劳 (n = 12 [22%])、瘙痒 (n = 12 [22%]) 和脂肪酶增加 (n = 10 [18%])。一名患者死于治疗相关的吉兰-巴利综合征。Pembrolizumab 具有持久的抗肿瘤活性和良好的生存前景，并且对复发性局部晚期或转移性 MCC 患者具有可控的安全性，支持其在该人群中的使用。Clinicaltrials.gov，NCT03783078.© 2024。作者。

The phase III KEYNOTE-913 study was conducted to evaluate the efficacy and safety of pembrolizumab as first-line therapy in patients with advanced Merkel cell carcinoma (MCC).The aim was to report results from the primary analysis of KEYNOTE-913.Patients with recurrent locally advanced or metastatic MCC received pembrolizumab 200 mg intravenously every 3 weeks for up to 35 treatments (~ 2 years). The primary end point was objective response rate (ORR) per Response Evaluation Criteria in Solid Tumors, version 1.1 (RECIST v1.1) by blinded independent central review (BICR). Secondary end points were duration of response (DOR) and progression-free survival (PFS) per RECIST v1.1 by BICR, overall survival (OS), and safety and tolerability.Fifty-five patients were treated with pembrolizumab. The median time from first dose to data cutoff (February 15, 2024) was 50.3 months (range 38.7-59.4). The ORR was 49% (95% confidence interval [CI] 35-63), with 12 complete responses and 15 partial responses. The median DOR was 39.8 months (range 4.8-52.5+), and the 24-month DOR rate was 69%. The median PFS was 9.3 months (95% CI 3-26), and the 24-month PFS rate was 39%. The median OS was 24.3 months (95% CI 12.4 to not reached), and the 24-month OS rate was 51%. Any-grade treatment-related adverse events (AEs) occurred in 38 patients (69%); 13 patients (24%) experienced grade 3-5 AEs. The most common treatment-related AEs were fatigue (n = 12 [22%]), pruritus (n = 12 [22%]), and lipase increase (n = 10 [18%]). One patient died of treatment-related Guillain-Barré syndrome.Pembrolizumab provided durable antitumor activity and promising survival and had a manageable safety profile in patients with recurrent locally advanced or metastatic MCC, supporting its use in this population.Clinicaltrials.gov, NCT03783078.© 2024. The Author(s).