Background

Sonic hedgehog inhibitors (SHHis) provide an additional treatment option for basal cell carcinomas (BCCs), especially for metastatic or locally advanced BCC. However, studies have been heterogeneous and lacked direct comparisons between molecules.

Objective

To determine the efficacy and safety of the class of molecules SHHi for treating BCC and to compare them individually.

Methods

We performed a PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses)–compliant systematic review of studies followed by a meta-analysis.

Results

Eighteen articles were included in our meta-analysis; 16 articles were combined for efficacy and 16 for safety. In locally advanced BCC, overall response rates (ORRs) were similar for vismodegib and sonidegib (69% vs 57%, respectively) but not complete response rates (31% vs 3%, respectively). In metastatic disease, the ORR of vismodegib was 2.7-fold higher than the ORR of sonidegib (39% vs 15%, respectively). For side effects affecting a majority of patients, prevalences for muscle spasms (67.1%), dysgeusia (54.1%), and alopecia (57.7%) were in similar proportions for sonidegib and vismodegib. Patients receiving sonidegib experienced more upper gastrointestinal distress than patients receiving vismodegib.

Conclusion

SHHis induce a partial response to locally advanced BCC disease. Side effects are common, similar across molecules, associated with high discontinuation rates, and warrant discussion beforehand.

中文翻译：

---

声波刺猬抑制剂在基底细胞癌中的疗效、安全性和比较：系统评价和荟萃分析

背景

声波刺猬抑制剂 (SHHis) 为基底细胞癌 (BCC) 提供了额外的治疗选择，特别是对于转移性或局部晚期 BCC。然而，研究是异质的，缺乏分子之间的直接比较。

客观的

确定 SHHi 类分子治疗 BCC 的功效和安全性并单独比较它们。

方法

我们对研究进行了符合 PRISMA（系统评价和 Meta 分析的首选报告项目）的系统评价，然后进行了荟萃分析。

结果

我们的荟萃分析纳入了 18 篇文章；16篇文章被合并为有效性和16篇安全性。在局部晚期 BCC 中，vismodegib 和 Sonidegib 的总体缓解率 (ORR) 相似（分别为 69% 和 57%），但不完全缓解率（分别为 31% 和 3%）。在转移性疾病中，vismodegib 的 ORR 比 Sonidegib 的 ORR 高 2.7 倍（分别为 39% 和 15%）。对于影响大多数患者的副作用，sonidegib 和vismodegib 的肌肉痉挛（67.1%）、味觉障碍（54.1%）和脱发（57.7%）的发生率相似。接受sonidegib的患者比接受vismodegib的患者经历更多的上消化道不适。

结论

SHHis 诱导对局部晚期 BCC 疾病的部分反应。副作用很常见，分子间相似，与高停药率相关，需要事先讨论。