Abstract
INTRODUCTION: Basal cell carcinoma (BCC) is the most common non-melanoma skin cancer. Treatment typically begins with surgical intervention. However, in cases of locally advanced and metastatic BCC (laBCC and mBCC), Hedgehog signaling pathway inhibitors such as sonidegib and vismodegib are used. MATERIALS AND METHODS: This retrospective study included 28 adult patients with laBCC who were treated with 150 mg daily oral vismodegib at Mehmet Akif Ersoy State Hospital between 2018 and 2023. Patients were monitored until disease progression, intolerable side effects, or treatment discontinuation. Treatment efficacy was evaluated using objective response rate (ORR) and disease control rate (DCR). Safety was assessed based on adverse events (AEs) reported in accordance with the CTCAE v. 5.0 classification. Statistical analyses, including overall survival (OS) and progression-free survival (PFS), were performed and analyzed using SPSS version 25. RESULTS: The overall ORR was 89.3%, with 39.3% of patients achieving complete response (CR). The median PFS was 15.1 months, and the median OS was 37.5 months. Female gender and prior surgical interventions were identified as independent prognostic factors for PFS, while ECOG-PS score and the duration of vismodegib exposure were significant predictors of OS. AEs were reported in 78.6% of patients, with dysgeusia and muscle spasms being the most prevalent. Grade 3 or 4 toxicity occurred in 14.5% of patients, and 13% (n = 3) discontinued treatment due to AEs. Patients receiving treatment for ≥ 12 months experienced a higher incidence of AEs (92.9%) compared to those treated for < 12 months (64.3%). CONCLUSION: Vismodegib demonstrated high efficacy in treating patients with laBCC, with a significant proportion achieving CR. However, long-term treatment was associated with an increased incidence of AEs, highlighting the need for careful monitoring of safety in prolonged therapies.