Department of Ophthalmology, Rabin Medical Center, Beilinson Hospital, Petach Tikva, Israel.
Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
JAMA Ophthalmol. 2020 Jul 1;138(7):749-755. doi: 10.1001/jamaophthalmol.2020.1539.
The outcomes of vismodegib treatment in a relatively large cohort of study participants with periocular locally advanced basal cell carcinoma (POLA-BCC) may guide physicians when considering this treatment.
To report the outcomes of vismodegib treatment in patients with POLA-BCC in the Safety Events in Vismodegib (STEVIE) study.
DESIGN, SETTING, AND PARTICIPANTS: This post hoc subgroup analysis from the STEVIE single-arm, multicenter, open-label cohort study screened all 1215 participants for ocular or periocular involvement and identified 244 participants with POLA-BCC or metastatic BCC. Data for the first STEVIE trial were collected from 167 treatment locations in 36 countries from June 30, 2011, to June 14, 2017. This post hoc analysis was performed from April 1 to August 31, 2019.
Response to treatment and adverse events.
Ocular or periocular involvement was found in 244 of 1215 STEVIE participants (20.1%), who constituted the analytic sample. The median age of the study participants was 72.0 (interquartile range [IQR], 60.0-82.0]) years, and they included 143 men (58.6%). Locally advanced BCC was diagnosed in 238 of the 244 participants (97.5%) and metastatic BCC, in 6 (2.5%). The median duration of exposure to vismodegib was 40.0 (IQR, 20.0-78.0) weeks, specifically 39.7 (IQR, 19.9-76.0) weeks for POLA-BCC and 92.4 (IQR, 53.2-163.0) weeks for metastatic BCC. Sixty-nine participants (28.3%) sustained serious adverse events (alopecia, muscle spasms, dysgeusia, weight loss, decreased appetite, asthenia, ageusia, nausea, fatigue, and diarrhea). Two hundred thirty-two study participants (95.1%) sustained more than 1 adverse effect. The overall mean (SD) number of drug-related adverse effects per study participant by first adverse event, regardless of the severity, was 5.48 (3.84). Discontinuation of vismodegib treatment owing to an adverse event was recorded in 58 participants (23.8%). During the study, 22 participants (9.0%) died, 70 (28.7%) achieved complete response, and 94 (38.5%) achieved partial response.
Vismodegib was well tolerated by the study participants with POLA-BCC. The safety of vismodegib treatment according to the STEVIE trial findings is consistent with that reported in previous studies. These data may be helpful when considering vismodegib for patients with POLA-BCC.
在一个相对较大的眼部局部晚期基底细胞癌（POLA-BCC）研究参与者队列中，维莫德吉治疗的结果可能为医生在考虑这种治疗方法时提供参考。
报告 Safety Events in Vismodegib（STEVIE）研究中维莫德吉治疗 POLA-BCC 患者的结果。
设计、地点和参与者：这项来自 STEVIE 单臂、多中心、开放标签队列研究的事后亚组分析对所有 1215 名参与者进行了眼部或眼周受累筛查，并确定了 244 名患有 POLA-BCC 或转移性 BCC 的患者。第一个 STEVIE 试验的数据于 2011 年 6 月 30 日至 2017 年 6 月 14 日从 36 个国家的 167 个治疗地点收集。该事后分析于 2019 年 4 月 1 日至 8 月 31 日进行。
治疗反应和不良反应。
在 1215 名 STEVIE 参与者中有 244 名（20.1%）发现眼部或眼周受累，他们构成了分析样本。研究参与者的中位年龄为 72.0 岁（四分位距[IQR]，60.0-82.0），其中 143 名男性（58.6%）。244 名参与者中 238 名（97.5%）诊断为局部晚期 BCC，6 名（2.5%）为转移性 BCC。维莫德吉暴露的中位持续时间为 40.0 周（IQR，20.0-78.0），具体而言，POLA-BCC 为 39.7 周（IQR，19.9-76.0），转移性 BCC 为 92.4 周（IQR，53.2-163.0）。69 名参与者（28.3%）出现严重不良事件（脱发、肌肉痉挛、味觉障碍、体重减轻、食欲下降、乏力、味觉丧失、恶心、疲劳和腹泻）。232 名研究参与者（95.1%）出现超过 1 种不良反应。首次出现不良反应时，每位研究参与者平均（SD）出现药物相关不良事件的数量为 5.48（3.84）。因不良反应而停止维莫德吉治疗的有 58 名参与者（23.8%）。在研究期间，有 22 名参与者（9.0%）死亡，70 名（28.7%）达到完全缓解，94 名（38.5%）达到部分缓解。
维莫德吉在患有 POLA-BCC 的研究参与者中耐受性良好。根据 STEVIE 试验结果，维莫德吉治疗的安全性与之前研究报告的结果一致。这些数据在考虑为 POLA-BCC 患者使用维莫德吉时可能会有所帮助。
