Division of Oncology, University of Southern California, Norris Comprehensive Cancer Center, Los Angeles, California, USA
Division of Medical Oncology, University of Miami, Sylvester Comprehensive Cancer Center, Miami, Florida, USA.
J Immunother Cancer. 2022 May;10(5). doi: 10.1136/jitc-2022-004839.
Basal cell carcinoma (BCC) is the most common malignancy worldwide, yet the management of patients with advanced or metastatic disease is challenging, with limited treatment options. Recently, programmed death receptor 1 (PD-1) inhibition has demonstrated activity in BCC after prior Hedgehog inhibitor treatment.
We conducted a multicenter, retrospective analysis of BCC patients treated with PD-1 inhibitor therapy. We examined the efficacy and safety of PD-1 therapy, as well as clinical and pathological variables in association with outcomes. Progression-free survival (PFS), overall survival (OS) and duration of response (DOR) were calculated using Kaplan-Meier methodology. Toxicity was graded per Common Terminology Criteria for Adverse Events V.5.0.
A total of 29 patients with BCC who were treated with PD-1 inhibition were included for analysis, including 20 (69.0%) with locally advanced and 9 (31.0%) with metastatic disease. The objective response rate was 31.0%, with five partial responses (17.2%), and four complete responses (13.8%). Nine patients had stable disease (31.0%), with a disease control rate of 62.1%. The median DOR was not reached. Median PFS was 12.2 months (95% CI 0.0 to 27.4). Median OS was 32.4 months (95% CI 18.1 to 46.7). Two patients (6.9%) developed grade 3 or higher toxicity, while four patients (13.8%) discontinued PD-1 inhibition because of toxicity. Higher platelets (p=0.022) and any grade toxicity (p=0.024) were significantly associated with disease control rate.
The clinical efficacy of PD-1 inhibition among patients with advanced or metastatic BCC in this real-world cohort were comparable to published trial data. Further investigation of PD-1 inhibition is needed to define its optimal role for patients with this disease.
基底细胞癌（BCC）是全球最常见的恶性肿瘤，但晚期或转移性疾病患者的治疗具有挑战性，治疗选择有限。最近，程序性死亡受体 1（PD-1）抑制剂在先前使用 Hedgehog 抑制剂治疗的 BCC 中显示出活性。
我们对接受 PD-1 抑制剂治疗的 BCC 患者进行了多中心、回顾性分析。我们检查了 PD-1 治疗的疗效和安全性，以及与结局相关的临床和病理变量。使用 Kaplan-Meier 方法计算无进展生存期（PFS）、总生存期（OS）和缓解持续时间（DOR）。毒性按常见不良事件术语标准 V.5.0 分级。
共纳入 29 例接受 PD-1 抑制治疗的 BCC 患者进行分析，其中局部晚期 20 例（69.0%），转移性疾病 9 例（31.0%）。客观缓解率为 31.0%，部分缓解 5 例（17.2%），完全缓解 4 例（13.8%）。9 例患者疾病稳定（31.0%），疾病控制率为 62.1%。中位 DOR 未达到。中位 PFS 为 12.2 个月（95%CI 0.0 至 27.4）。中位 OS 为 32.4 个月（95%CI 18.1 至 46.7）。2 例患者（6.9%）发生 3 级或更高毒性，4 例患者（13.8%）因毒性而停止 PD-1 抑制。较高的血小板（p=0.022）和任何等级的毒性（p=0.024）与疾病控制率显著相关。
在这项真实世界队列中，晚期或转移性 BCC 患者接受 PD-1 抑制的临床疗效与已发表的试验数据相当。需要进一步研究 PD-1 抑制，以确定其在该疾病患者中的最佳作用。
