西米普利单抗治疗局部晚期或转移性皮肤鳞状细胞癌：来自药物获取协议的前瞻性真实世界数据。
Cemiplimab in locally advanced or metastatic cutaneous squamous cell carcinoma: prospective real-world data from the DRUG Access Protocol.
作者信息
Verkerk Karlijn, Geurts Birgit S, Zeverijn Laurien J, van der Noort Vincent, Verheul Henk M W, Haanen John B A G, van der Veldt Astrid A M, Eskens Ferry A L M, Aarts Maureen J B, van Herpen Carla M L, Jalving Mathilde, Gietema Jourik A, Devriese Lot A, Labots Mariette, Barjesteh van Waalwijk van Doorn-Khosrovani Sahar, Smit Egbert F, Bloemendal Haiko J
机构信息
Division of Molecular Oncology & Immunology, Netherlands Cancer Institute, Amsterdam, the Netherlands.
Oncode Institute, Utrecht, the Netherlands.
出版信息
Lancet Reg Health Eur. 2024 Mar 5;39:100875. doi: 10.1016/j.lanepe.2024.100875. eCollection 2024 Apr.
BACKGROUND
The DRUG Access Protocol provides patients with cancer access to registered anti-cancer drugs that are awaiting reimbursement in the Netherlands and simultaneously collects prospective real-world data (RWD). Here, we present RWD from PD-1 blocker cemiplimab in patients with locally advanced or metastatic cutaneous squamous cell carcinoma (laCSCC; mCSCC).
METHODS
Patients with laCSCC or mCSCC received cemiplimab 350 mg fixed dose every three weeks. Primary endpoints were objective clinical benefit rate (CBR), defined as objective response (OR) or stable disease (SD) at 16 weeks, physician-assessed CBR, defined as clinician's documentation of improved disease or SD based on evaluation of all available clinical parameters at 16 weeks, objective response rate (ORR), and safety, defined as grade ≥ 3 treatment related adverse events (TRAEs) occurring up to 30 days after last drug administration. Secondary endpoints included duration of response (DoR), progression-free survival (PFS), and overall survival (OS).
FINDINGS
Between February 2021 and December 2022, 151 patients started treatment. Objective and physician-assessed CBR were 54.3% (95% CI, 46.0-62.4) and 59.6% (95% CI, 51.3-67.5), respectively. ORR was 35.1% (95% CI, 27.5-43.3). After a median follow-up of 15.2 months, median DoR was not reached. Median PFS and OS were 12.2 (95% CI, 7.0-not reached) and 24.2 months (95% CI, 18.8-not reached), respectively. Sixty-eight TRAEs occurred in 29.8% of patients. Most commonly reported TRAE was a kidney transplant rejection (9.5%).
INTERPRETATION
Cemiplimab proved highly effective and safe in this real-world cohort of patients with laCSCC or mCSCC, confirming its therapeutic value in the treatment of advanced CSCC in daily clinical practice.
FUNDING
The DRUG Access Protocol is supported by all participating pharmaceutical companies: Bayer, Janssen, Lilly, Merck, Novartis, Roche, and Sanofi.
背景
药物获取协议为荷兰等待报销的癌症患者提供注册抗癌药物，并同时收集前瞻性真实世界数据（RWD）。在此，我们展示了帕博利珠单抗在局部晚期或转移性皮肤鳞状细胞癌（laCSCC；mCSCC）患者中的真实世界数据。
方法
laCSCC或mCSCC患者每三周接受350mg固定剂量的帕博利珠单抗治疗。主要终点为客观临床获益率（CBR），定义为16周时的客观缓解（OR）或疾病稳定（SD）；医生评估的CBR，定义为医生根据16周时所有可用临床参数评估记录的疾病改善或SD；客观缓解率（ORR）；以及安全性，定义为末次给药后30天内发生的≥3级治疗相关不良事件（TRAEs）。次要终点包括缓解持续时间（DoR）、无进展生存期（PFS）和总生存期（OS）。
研究结果
2021年2月至2022年12月期间，151例患者开始治疗。客观和医生评估的CBR分别为54.3%（95%CI，46.0 - 62.4）和59.6%（95%CI，51.3 - 67.5）。ORR为35.1%（95%CI，27.5 - 43.3）。中位随访15.2个月后，中位DoR未达到。中位PFS和OS分别为12.2（95%CI，7.0 - 未达到）和24.2个月（95%CI，18.8 - 未达到）。29.8%的患者发生了68起TRAEs。最常报告的TRAEs是肾移植排斥反应（9.5%）。
解读
在这一laCSCC或mCSCC患者的真实世界队列中，帕博利珠单抗被证明具有高度有效性和安全性，证实了其在日常临床实践中治疗晚期CSCC的治疗价值。
资助
药物获取协议由所有参与的制药公司支持：拜耳、杨森、礼来、默克、诺华、罗氏和赛诺菲。
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