Eight-Year Experience of Hedgehog Pathway Inhibitors at Three Tertiary Referral Centres in the Australian State of...

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.