Gan Christian, Manuelpillai Nicholas, Foley Peter, McCormack Christopher, Goh Michelle S
Skin Health Institute, Carlton, Victoria, Australia.
Department of Dermatology, St Vincent's Hospital Melbourne, Fitzroy, Victoria, Australia.
Australas J Dermatol. 2025 Jun 10;66(5):e297-301. doi: 10.1111/ajd.14544.
This retrospective analysis of Hedgehog inhibitor treatment in 32 patients with Gorlin syndrome, locally advanced and metastatic basal cell carcinoma (BCC) at three tertiary referral centres in Victoria, Australia from April 2017 until 30 June 2024 demonstrated an 84% overall objective response rate (partial and complete response combined). However, 90% of patients experienced adverse effects impacting quality of life. Secondary acquired drug resistance occurred in 77% (10/13) of locally advanced and metastatic BCC patients after a median duration of 13 months. Further work is needed to optimise the neoadjuvant use of Hedgehog inhibitors with radiotherapy or surgery given poor long-term Hedgehog inhibitor tolerability and to develop strategies to counteract the issue of acquired resistance.
这项对2017年4月至2024年6月30日期间在澳大利亚维多利亚州三个三级转诊中心接受刺猬抑制剂治疗的32例戈林综合征、局部晚期和转移性基底细胞癌(BCC)患者的回顾性分析显示,总体客观缓解率(部分缓解和完全缓解合并)为84%。然而,90%的患者出现了影响生活质量的不良反应。局部晚期和转移性BCC患者中,77%(10/13)在中位持续时间13个月后出现继发性获得性耐药。鉴于刺猬抑制剂的长期耐受性较差,需要进一步开展工作,以优化刺猬抑制剂与放疗或手术的新辅助使用,并制定应对获得性耐药问题的策略。