Omland Lise Høj, Stormoen Dag Rune, Dohn Line Hammer, Carus Andreas, Als Anne Birgitte, Jensen Niels Viggo, Taarnhøj Gry Assam, Tolver Anders, Pappot Helle
Department of Oncology, Copenhagen University Hospital, Rigshospitalet, Copenhagen, Denmark.
Department of Oncology, Herlev and Gentofte University Hospital, Copenhagen, Denmark.
Bladder Cancer. 2021 Dec 13;7(4):413-425. doi: 10.3233/BLC-211523. eCollection 2021.
Investigating the effect of newly approved oncological drugs in the real-world is 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.
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.
139 patients were identified, 53 in first-line treatment, 77 in second-line, and 9 receiving third 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.
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.
有必要在现实世界中研究新批准的肿瘤药物的效果。随着针对尿路上皮癌的新型治疗方法迅速获批，我们旨在评估丹麦批准帕博利珠单抗用于一线和二线局部晚期、不可切除或转移性尿路上皮癌（mUTC）患者后的第一年中，关于其疗效和安全性的真实世界数据。
在丹麦治疗mUTC的六个肿瘤科室中，我们确定了2018年3月1日至2019年2月28日批准后的第一年中所有接受帕博利珠单抗治疗的mUTC患者。于2020年1月至6月进行回顾性数据收集。评估了与帕博利珠单抗在一线和二线治疗环境中的相关临床试验相匹配的患者特征、总生存期（OS）、无进展生存期（PFS）、毒性和肿瘤反应。
共确定了139例患者，其中53例接受一线治疗，77例接受二线治疗，9例接受三线或更晚线治疗。该人群的特征为大多数为男性（70%），大多数患者ECOG PS为0 - 1（60.4%），膀胱原发性肿瘤占主导（90.6%）。一线治疗的总缓解率（ORR）为30.2%，PFS为3.5个月（95%CI 2.3 - 7.9个月），OS为9.2个月（95%CI 7.0 - 20.9个月）。二线治疗的ORR为27.3%，PFS为2.9个月（95%CI 2.5 - 5.3），OS为9.1个月（95%CI 5.4 - 12.8个月）。毒性与临床试验相当，未记录到任何新的毒性。
mUTC患者一线和二线接受帕博利珠单抗治疗的缓解率、OS、PFS和毒性的真实世界数据与临床试验结果相当。本研究进一步确立了免疫疗法作为mUTC的一种有效且可耐受的治疗方法。
