Abstract

BACKGROUND:

Effect of newly approved oncological drugs in the real-world are warranted. With emerging novel treatments rapidly being approved for urothelial tract cancers, we aimed to assess real-world data, regarding effect and safety, during the first year after approval of pembrolizumab in Denmark for patients with locally advanced and unresectable or metastatic urothelial tract cancer (mUTC) in the first- and second-line setting.

MATERIALS AND METHOD:

At the six oncological departments treating mUTC in Denmark, we identified all mUTC patients receiving pembrolizumab during the first year after approval, between March 1, 2018 and February 28, 2019. A retrospective data collection was conducted from January to June 2020. Patient characteristics matching that of the relevant clinical trials for pembrolizumab in first- and second-line treatment-setting, overall survival (OS), progression-free survival (PFS), toxicity and tumor response were assessed.

RESULTS:

139 patients were identified, 53 in first-line treatment, 77 in second-line, and 9 receiving 3^rd or later lines of treatment. The population was characterized by a majority of males (70%), most patients had ECOG PS 0–1 (60.4%) and primary tumor in the bladder was predominant (90.6%). The overall response rate (ORR) in first-line was 30.2%, PFS was 3,5 months (95%CI 2,3–7,9 months) and OS 9,2 months (95%CI 7,0–20.9 months). For second-line treatment the ORR was 27,3%, PFS 2,9 months (95%CI 2,5–5,3) and OS 9.1 months (95%CI 5,4–12,8 months). Toxicity was comparable to clinical trials without any new toxicities registered.

CONCLUSION:

Real-world data on response rates, OS, PFS and toxicity for patients with mUTC receiving pembrolizumab in first- and second-line, shows comparable results to clinical trials. This study further establishes immunotherapy as an effective and tolerable treatment for mUTC.

中文翻译：

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丹麦晚期尿路上皮癌患者使用帕博利珠单抗治疗的真实世界研究

摘要

背景：

新批准的肿瘤药物在现实世界中的作用是有保证的。随着新兴的新疗法迅速被批准用于治疗尿路上皮癌，我们的目标是在丹麦批准帕博利珠单抗治疗局部晚期和不可切除或转移性尿路上皮癌 (mUTC) 患者后的第一年，评估有关疗效和安全性的真实世界数据。 ) 在第一行和第二行设置中。

材料和方法：

在丹麦治疗 mUTC 的六个肿瘤科，我们确定了在批准后第一年（即 2018 年 3 月 1 日至 2019 年 2 月 28 日期间）接受 pembrolizumab 治疗的所有 mUTC 患者。从 1 月至 2020 年 6 月进行了回顾性数据收集。患者特征匹配评估了 pembrolizumab 在一线和二线治疗中的相关临床试验、总生存期 (OS)、无进展生存期 (PFS)、毒性和肿瘤反应。

结果：

139例患者进行了鉴定，53在第一线治疗，77在第二行，和9接收3^次或治疗的行以后。该人群的特征是大多数男性 (70%)，大多数患者的 ECOG PS 0-1 (60.4%) 和膀胱原发肿瘤占主导地位 (90.6%)。一线总缓解率 (ORR) 为 30.2%，PFS 为 3.5 个月（95%CI 2,3-7,9 个月）和 OS 9.2 个月（95%CI 7,0-20.9 个月） ）。对于二线治疗，ORR 为 27.3%，PFS 2.9 个月（95%CI 2,5-5,3）和 OS 9.1 个月（95%CI 5,4-12,8 个月）。毒性与临床试验相当，没有登记任何新的毒性。

结论：

接受 pembrolizumab 一线和二线治疗的 mUTC 患者的反应率、OS、PFS 和毒性的真实世界数据显示出与临床试验相当的结果。该研究进一步确立了免疫疗法作为 mUTC 有效且可耐受的治疗方法。