尽管平滑抑制剂 (SMOi) 已证明在治疗基底细胞癌 (BCC) 方面有效，但尚无关于如何利用 SMOi 治疗 Gorlin 综合征 (GS) 的指南。评估 GS 中 SMOi 的临床反应，提供实用的指导为临床医生提供指导，并确定未来研究的领域。除了在 Medline 和 Google Scholar 上全面搜索与 Gorlin 综合征 (GS) 相关的全身疗法外，还特别寻求专家意见，以提供有关使用 SMOi 治疗 GS 的详细指导.SMOi 的间歇给药和每日给药具有相似的功效。间歇给药可以提高治疗的依从性。然而，缺乏耐受性限制了SMOi的长期使用。仅针对SMOi在GS中使用的研究很少。纳入的研究通常有一个 GS 患者亚组。虽然 SMOi 的不良事件（例如 vismodegib 和 sonidegib）可能导致在 GS 治疗期间停药，但间歇给药可能会提高依从性。需要进一步研究来确定剂量策略，以最大限度地减少肿瘤负担，同时减轻副作用，以实现最大的依从性。版权所有 © 2024。由 Elsevier Inc. 出版。

Although smoothened inhibitors (SMOi) have demonstrated efficacy in the management of basal cell carcinoma (BCC), no guidelines are available on how to utilize SMOi in the treatment of Gorlin syndrome (GS).Assess the clinical response to SMOi in GS, provide practical guidance for clinicians, and identify areas for future research.Alongside a comprehensive search of Medline and Google Scholar for systemic therapies related to Gorlin Syndrome (GS), expert opinion was specifically sought to provide detailed guidance on the use of SMOi in the management of GS.Intermittent dosing of SMOi and daily dosing had similar efficacy. Intermittent dosing resulted in better compliance with therapy. However, lack of tolerability limited long-term use of SMOi.There are few studies solely on the use of SMOi in GS. Included studies often had a subgroup of GS patients.While the adverse events of SMOi, such as vismodegib and sonidegib, may result in their discontinuation during treatment of GS, intermittent dosing may improve compliance. Further research is needed to identify dosing strategies that minimize tumor burden while mitigating side effects to achieve maximum compliance.Copyright © 2024. Published by Elsevier Inc.