Vismodegib适用于治疗晚期或转移性基底细胞癌(BCC)。迄今为止,对vismodegib反应的预测因素描述不佳。
主要目的是确定对vismodegib有反应的患者的概况和反应持续时间。次要目标是评估治疗持续时间与复发风险之间是否存在相关性。并定义与复发相关的因素。
我们纳入了61例局部晚期BCC(laBCC)或多发性BCC患者,用vismodegib治疗(每天150毫克),2011年7月至2015年11月,在法国南特大学医院肿瘤科。使用实体瘤版本1.1中的反应评估标准评估肿瘤反应。
39例患者患有晚期BCC(64%),22例患者患有多发性BCC(36%)。包括10例Gorlin综合征患者。无因子预测对vismodegib的反应。总人口的中位无进展生存期(PFS)为69.5个月。在多变量分析中,多发性BCC是与复发风险增加相关的唯一因素(HR:13.80[CI95%,1.93-98.64,p<0.01])。治疗持续时间降低了复发的风险(HR0.95[CI95%,0.90-0.99,p=0.0467])。在随访期间复发的20例患者中,15人(75%)用vismodegib重新治疗,反应率为66%。
虽然我们无法确定对vismodegib反应的预测因素,我们首次证明治疗持续时间延长与复发风险降低相关.
Vismodegib is indicated for the treatment of advanced or metastatic basal cell carcinoma (BCC). The predictive factors of response to vismodegib have so far been poorly described.
The primary objective was to determine the profile of patients responding to vismodegib and the duration of response. Secondary objectives were to assess whether there is a correlation between the duration of treatment and the risk of relapse, and to define factors associated with relapse.
We included 61 patients with locally advanced BCC (laBCC) or multiple BCC, treated with vismodegib (150 mg per day), from July 2011 to November 2015, in the Oncodermatology Department of Nantes University Hospital in France. Tumour response was assessed using Response Evaluation Criteria in Solid Tumours version 1.1.
Thirty-nine patients had advanced BCC (64%) and 22 patients multiple BCC (36%), including 10 patients with Gorlin syndrome. No factor predicted response to vismodegib. The median progression-free survival (PFS) was 69.5 months for the total population. In multivariate analysis, multiple BCC was the only factor associated with an increased risk of relapse (HR: 13.80 [CI95%, 1.93-98.64, p < 0.01]). Treatment duration decreased the risk of relapse (HR 0.95 [CI95%, 0.90-0.99, p = 0.0467]). Among the 20 patients who experienced relapse during follow-up, 15 (75%) were re-treated with vismodegib, with a response rate of 66%.
Although we were unable to establish predictive factors for the response to vismodegib, we demonstrate for the first time that increased treatment duration correlates with a decreased risk of relapse.