旨在回顾口服 vismodegib（Erivedge；Genentech）在治疗局部晚期眼眶和眶周基底细胞癌 (BCC) 中的有效性和安全性。最后于 2023 年 9 月在 PubMed 数据库中进行了一项文献检索，以评估英语原创研究口服 vismodegib 对眼眶和眶周 BCC 的影响。确定了 60 篇文章，其中 16 篇符合纳入标准。大多数研究显示出高缓解率，在个别研究中，高达 100% 的患者对药物有反应，高达 88% 的患者出现初始完全消退。 Vismodegib 治疗导致肿瘤体积显着减小，从而使大多数患者的眼球得以保留。然而，12% 的患者仅有部分缓解。即使在最初完全缓解后，复发也频繁发生。因此，高达 79.4% 的患者需要手术干预，高达 23% 的患者仍需要切除术。使用这些药物可以缩小肿瘤体积，从而可以延迟或避免某些（但不是全部）患者需要进行切除术。重要的是，对 vismodegib 治疗后切除的组织进行分子分析显示，所有患者均存在持续性肿瘤，并且突变频繁积累，可能会对进一步的刺猬抑制剂治疗产生耐药性。尽管大多数不良事件被评为 I 级或 II 级，但副作用很常见，研究中高达 100% 的患者至少经历过 1 次事件。肌肉痉挛、脱发、体重减轻、疲劳和味觉障碍是最常见的不良事件，一些患者因此停止了治疗。此外，1 名患者死于可能由治疗引起的败血症。尽管缺乏 I 级和 II 级证据，但大多数研究表明使用口服 vismodegib 治疗眼眶和眶周 BCC 肿瘤体积有益处。然而，患者应警惕治疗的不良副作用以及可能导致长期耐药的突变肿瘤细胞的持续存在。使用 vismodegib 作为短期新辅助治疗可能会有效缩小肿瘤体积，从而降低手术发病率，同时降低副作用的频率和严重程度。作者对本文讨论的任何材料没有专有或商业利益。版权© 2024 美国眼科学会。由爱思唯尔公司出版。保留所有权利。

To review the efficacy and safety of oral vismodegib (Erivedge; Genentech) in the management of locally advanced orbital and periorbital basal cell carcinoma (BCC).A literature search was conducted last in September 2023 in the PubMed database for English language original research that evaluated the effect of oral vismodegib on orbital and periorbital BCC. Sixty articles were identified and 16 met the inclusion criteria.Most studies demonstrated high response rates, with up to 100% of patients responding to the medication in individual studies and initial complete regression occurring in up to 88% of patients. Vismodegib treatment resulted in significant reductions in tumor volume, resulting in globe preservation for most patients. However, in 12% of patients, the response was partial. Recurrences also occurred with substantial frequency, even after an initial complete response. As such, up to 79.4% of patients required surgical intervention, and up to 23% of patients still required exenteration. Use of these agents resulted in reductions in tumor volume that may delay or prevent the need for exenteration in some, but not all, patients. Importantly, molecular analysis of tissue excised after vismodegib therapy revealed persistent tumor in all patients, with frequent accumulation of mutations that may confer resistance to further hedgehog inhibitor therapy. Although most adverse events were rated as level I or II, side effects were common, with up to 100% of patients in studies experiencing at least 1 event. Muscle cramps, alopecia, weight loss, fatigue, and dysgeusia were the most common adverse events, and several patients discontinued therapy because of them. Furthermore, 1 patient died of sepsis that may have resulted from the therapy.Although level I and II evidence are lacking, most studies indicate a benefit from the use of oral vismodegib to treat orbital and periorbital BCC tumor volume. However, patients should be cautioned about the adverse side effects of treatment and the persistence of tumor cells with mutations that may cause long-term resistance. Use of vismodegib as short-term neoadjuvant therapy may be effective in shrinking tumor volume to reduce surgical morbidity while reducing the frequency and severity of side effects.The author(s) have no proprietary or commercial interest in any materials discussed in this article.Copyright © 2024 American Academy of Ophthalmology. Published by Elsevier Inc. All rights reserved.