Treatment of periocular basal cell carcinoma (BCC) is difficult due to proximity to critical structures, including the eye and intracranial areas. Systemic hedgehog pathway inhibitor therapy (HHIT), including vismodegib and sonidegib, has emerged as a nonsurgical alternative, approved as monotherapy for locally advanced or metastatic BCC unsuitable for surgery or radiation therapy (RT). Off-label uses of HHIT have also shown promise, such as neoadjuvant use prior to surgery or RT, or in operable cases to avoid extensive resection. We present a case of sonidegib use in an operable but surgically challenging tumor. A 72-year-old Caucasian female with right periocular BCC was treated with sonidegib in lieu of radical surgery. Marked tumor regression over six months obviated the need for extensive resection. To contextualize our case, a literature search was conducted on 4 November 2024 to evaluate outcomes of periocular BCC treated with sonidegib. Manual screening identified eight articles meeting the inclusion criteria of sonidegib use with reported outcomes. In our review, neoadjuvant sonidegib achieved a 100% complete response (CR) rate, while monotherapy achieved CR in 46.8% of the cases. Deviation from daily dosing, including pulse regimens, maintained responses while reducing adverse effects. One case reported regression with concurrent sonidegib and itraconazole. Our case adds to the limited but growing evidence supporting off-label use of sonidegib in periocular BCC, including in operable tumors to preserve cosmesis or avoid extensive surgery, and in neoadjuvant, alternative dosing, and combination approaches.
Fan et al. (2026) studied this question.