Niedersuess-Beke Dora, Mayrhofer Karl, Krauter Johanna, Schnabel Susanne, Gampenrieder Simon Peter, Miechowiecki Jan, Kiesl David, Luger Ferdinand, Pfuner Jakob, Wiesinger Clemens, Vallet Sonia, Andalibi Haleh, Vais Dominik, Banner Andreas, Stoiber Franz, Spielgelberg Jasmin, Barth Dominik, Bauernhofer Thomas, Aufderklamm Stefan, Weibrecht Sabine, Mühlmann Josef, Mayer Michael, Hilbe Wolfgang, Boulmé Florence, Klinglmair Gerald, Heintel Daniel, Shariat Shahrokh F, Pichler Martin, Pichler Renate
Department of Internal Medicine I., Centre for Oncology and Haematology, Vienna Healthcare Group, Ottakring, Vienna, Austria.
Department of Internal Medicine I., Centre for Oncology and Haematology, Vienna Healthcare Group, Ottakring, Vienna, Austria.
Clin Genitourin Cancer. 2025 Feb;23(1):102278. doi: 10.1016/j.clgc.2024.102278. Epub 2024 Nov 22.
Enfortumab vedotin (EV) represents a novel treatment for patients with locally advanced or metastatic urothelial carcinoma (la/mUC) refractory to platinum-based chemotherapy and PD(L)-1 containing therapies. Real-world data are crucial for informing health policy decisions and validating clinical trial findings.
We conducted a multicentre, retrospective real-world analysis comprising 128 patients with la/mUC from 16 Austrian centres treated with EV from April 2022 to April 2024, presenting the second largest real-world cohort to date. Data were analysed for efficacy and safety parameters.
The median age was 69 years, the objective response rate 31% and the disease control rate 47%, with 9% of patients exhibiting a complete remission, 23% a partial remission and 16% a stable disease. After a median follow-up of 6.2 months, the median progression-free survival (mPFS) and the median overall survival (mOS) reached 4.8 and 10.75 months, respectively. Patients with good ECOG PS 0-1, metachronous metastatic disease and absence of liver metastases had significantly better OS. No difference in efficacy was observed in patients who received a reduced dose EV after experiencing adverse events. The safety profile was acceptable, showing grade ≥3 TRAEs in 25.8% of patients.
In our real-world population, the administration of EV was feasible and effective, with no new safety signals. Lower efficacy data compared to previous trials might be explained by the use in later therapy lines and in patients with poorer ECOG PS. Our data corroborate the efficacy and safety of EV monotherapy in later lines.
恩杂鲁胺（EV）是一种针对铂类化疗和含PD（L）-1疗法难治的局部晚期或转移性尿路上皮癌（la/mUC）患者的新型治疗方法。真实世界数据对于为卫生政策决策提供信息和验证临床试验结果至关重要。
我们进行了一项多中心回顾性真实世界分析，纳入了2022年4月至2024年4月期间在奥地利16个中心接受EV治疗的128例la/mUC患者，这是迄今为止第二大真实世界队列。分析数据的疗效和安全性参数。
中位年龄为69岁，客观缓解率为31%，疾病控制率为47%，9%的患者完全缓解，23%部分缓解，16%疾病稳定。中位随访6.2个月后，中位无进展生存期（mPFS）和中位总生存期（mOS）分别达到4.8个月和10.75个月。ECOG PS 0-1良好、异时性转移疾病且无肝转移的患者总生存期显著更好。在经历不良事件后接受减量EV治疗的患者中，未观察到疗效差异。安全性可接受，25.8%的患者出现≥3级治疗相关不良事件（TRAE）。
在我们的真实世界人群中，EV的给药是可行且有效的，没有新的安全信号。与先前试验相比，疗效数据较低可能是由于在后续治疗线中使用以及ECOG PS较差的患者中使用。我们的数据证实了EV单药治疗在后续治疗线中的疗效和安全性。
