Abstract：
Treatment with Hedgehog Inhibitors in Gorlin-Goltz syndrome (GGS) yields favourable objective clinical responses, yet secondary resistance and class-related toxicity restrict treatment duration. This study aims to review current data on GGS patients undergoing vismodegib therapy, focusing on treatment duration, clinical outcomes and schedule modifications. A systematic search of the PubMed database was conducted for English articles from 1993 to 2023, identifying 31 papers suitable for inclusion. A total of 351 patients, with a mean age of 52 years, were analysed. The average treatment duration was 9.3 months for patients who discontinued treatment, and 25.1 months for those who continued vismodegib at the time this study was published. Vismodegib achieved a complete response rate of 44%. Treatment interruption predominantly occurred due to side effects (69.1%) and secondary resistance (9.1%). The use of alternative regimens, although not compromising efficacy, may enhance treatment compliance. Further investigations are warranted to ascertain the optimal treatment regimen and timeline for GGS patients. Schedule modifications offer promise in ameliorating side effects and facilitating long-term treatment.
摘要：
用Hedgehog抑制剂治疗Gorlin-Goltz综合征（GGS）可产生良好的客观临床反应，然而,继发性耐药性和类别相关毒性限制了治疗持续时间.本研究旨在回顾GGS患者接受vismodegib治疗的当前数据，专注于治疗持续时间，临床结果和时间表修改。对PubMed数据库进行了系统搜索，以查找1993年至2023年的英文文章，确定了31篇适合纳入的论文。共351名患者，平均年龄为52岁，进行了分析。中断治疗的患者平均治疗时间为9.3个月，对于在本研究发表时继续使用vismodegib的人，则为25.1个月。Vismodegib的完全缓解率为44％。治疗中断主要是由于副作用（69.1％）和继发耐药性（9.1％）。使用替代方案，虽然不影响疗效，可以提高治疗依从性。需要进一步研究以确定GGS患者的最佳治疗方案和时间表。时间表的修改为改善副作用和促进长期治疗提供了希望。
