皮肤基底细胞癌（BCC）是美国最常见的皮肤癌。在危及生命的晚期BCC中，Sonic Hedgehog抑制剂（SSHis）仍然是局部晚期BCC和转移性BCC的主要治疗选择。在这份更新的系统性回顾和Meta分析中，我们旨在通过包括关键性临床试验和新增的研究，更好地表征SSHis的功效和安全性。我们检索了包括临床试验、前瞻性病例系列和回溯性病历回顾等涉及人类主体的文章。总反应率（ORR）和完全反应率（CRR）是主要结果。为了进行安全评估，分析以下不利影响（如肌肉痉挛、味觉障碍、脱发、体重减轻、疲劳、恶心、肌肉痛、呕吐、皮肤鳞状细胞癌、肌酸激酶水平升高、腹泻、食欲减退和闭经）的普遍性。我们使用R统计软件进行分析。我们使用线性模型进行汇总分析，并使用95%的置信区间（CI）和p值，来进行主要分析以及Fisher精确检验来计算分子间的差异。共有22项研究（N = 2384名患者）被纳入Meta分析：19项研究评估了功效和安全性，2项研究仅评估了安全性，1项研究仅评估了功效。总体而言，所有患者的总ORR为64.9％（95％CI48.2-81.6％），这意味着接受SSHis的大多数患者至少会出现部分反应（z = 7.60，p <0.0001）。Vismodegib的ORR为68.5％，Sonidegib的ORR为50.1％。Vismodegib和Sonidegib的最常见不良反应分别是肌肉痉挛（70.5％和61.0％），味觉障碍（58.4％和48.6％）和脱发（59.9％和51.1％）。从Vismodegib中，患者可能会出现体重减轻（35.1％，p <0.0001）。相反，服用Sonidegib的患者比服用Vismodegib的患者更容易出现恶心、腹泻、肌肉酸激酶水平升高和食欲减退。SSHis是治疗晚期BCC疾病有效的方法。考虑到高的停药率，有必要管理患者的期望以确保其遵守和长期有效。了解最新的SSHis的功效和安全信息是至关重要的。 ©2023。 作者（部）已独家许可Springer Nature Switzerland AG。

Basal cell carcinoma (BCC) of the skin is the most common form of skin cancer in the United States. In life-threatening, advanced BCC, sonic hedgehog inhibitors (SSHis) remain a pre-eminent treatment option for locally advanced BCC and metastatic BCC.In this updated systematic review and meta-analysis, we aimed to better characterize the efficacy and safety of SSHis by including final updates from pivotal clinical trials and additional new recent studies.An electronic database search was performed for articles including clinical trials, prospective case series, and retrospective medical record reviews on human subjects. Overall response rates (ORRs) and complete response rates (CRRs) were the primary outcomes. For safety assessment, the prevalence of the following adverse effects was analyzed: muscle spasms, dysgeusia, alopecia, weight loss, fatigue, nausea, myalgias, vomiting, skin squamous cell carcinoma, increased creatine kinase, diarrhea, decreased appetite, and amenorrhea. Analyses were performed using R statistical software. Data were pooled using linear models with fixed effects meta-analysis for primary analyses, along with 95% confidence intervals (CIs) and p-values. Intermolecular differences were calculated using Fisher's exact test.A total of 22 studies (N = 2384 patients) were included in the meta-analysis: 19 studies assessing both efficacy and safety, 2 studies assessing safety only, and 1 study assessing efficacy only. Overall, the pooled ORR for all patients was 64.9% (95% CI 48.2-81.6%), implicating there is at least a partial response (z = 7.60, p < 0.0001) in most patients receiving SSHis. The ORR for vismodegib was 68.5% and 50.1% for sonidegib. The most common adverse effects for vismodegib and sonidegib were muscle spasms (70.5% and 61.0%, respectively), dysgeusia (58.4% and 48.6%, respectively), and alopecia (59.9% and 51.1%, respectively). Patients were likely to experience weight loss (35.1%, p < 0.0001) from vismodegib. Alternatively, patients taking sonidegib experienced more nausea, diarrhea, increased creatine kinase levels, and decreased appetite compared with those receiving vismodegib.SSHis are an effective treatment for advanced BCC disease. Given the high discontinuation rates, management of patient expectations is warranted for compliance and achieving long-term efficacy. It is essential to stay updated with the latest discoveries on the efficacy and safety of SSHis.© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.