Kaatz Martin, Mohr Peter, Livingstone Elisabeth, Weichenthal Michael, Kreuter Alexander, Pföhler Claudia, Leiter Ulrike, Ulrich Jens, Utikal Jochen Sven, Gutzmer Ralf, Herbst Rudolf, Schadendorf Dirk
Department of Dermatology, University Hospital Essen, Hufelandstr. 55, DE-45122 Essen, Germany.
Acta Derm Venereol. 2022 Apr 13;102:adv00695. doi: 10.2340/actadv.v102.293.
Most patients with advanced basal cell carcinomas (BCCs) may not benefit sufficiently from standard treatment comprising surgery and radiation. Vismodegib, an oral selective hedgehog pathway inhibitor, is approved for treatment of patients with locally advanced BCC inappropriate for surgery or radiotherapy, or for patients with symptomatic metastatic BCC. In order to enhance understanding of the effectiveness, safety and utilization of vismodegib in clinical practice in Germany, a non-interventional study, JONAS, was conducted. A total of 53 patients with locally advanced BCC who initiated treatment with vismodegib between 2016 and 2018 were included in the study, which was embedded in the German ADOReg skin cancer registry. Duration of response, the primary endpoint, was 12.4 months, progression-free survival 32.2 months and overall response rate 77.4%. Most adverse events were mild to moderate. Overall, results confirmed previous findings, demonstrating favourable responses and manageable safety of vismodegib in patients with locally advanced BCC in clinical practice.
大多数晚期基底细胞癌（BCC）患者可能无法从包括手术和放疗在内的标准治疗中充分获益。维莫德吉是一种口服选择性刺猬信号通路抑制剂，被批准用于治疗不适合手术或放疗的局部晚期BCC患者，或有症状的转移性BCC患者。为了增强对维莫德吉在德国临床实践中的有效性、安全性和使用情况的了解，开展了一项非干预性研究JONAS。共有53例在2016年至2018年间开始使用维莫德吉治疗的局部晚期BCC患者被纳入该研究，该研究纳入了德国ADOReg皮肤癌登记处。主要终点缓解持续时间为12.4个月，无进展生存期为32.2个月，总缓解率为77.4%。大多数不良事件为轻度至中度。总体而言，结果证实了先前的发现，表明维莫德吉在临床实践中对局部晚期BCC患者有良好的反应且安全性可控。
