Sonidegib: Real-life experience with Hedgehog inhibitor for the treatment of locally advanced basal cell carcinoma

Abstract

Introduction: Sonidegib, a Hedgehog inhibitor approved for the treatment of advanced basal cell carcinoma (BCC), reported an objective response rate of 60.6% (central review) and 74.2% (investigator review) and a median duration of response of 26.1 months in the pivotal trial BOLT. The therapy showed an excellent tolerability profile. The most frequent adverse events are muscle spasms, dysgeusia, alopecia, nausea, increased creatine kinase, weight loss and fatigue. Case Report: This article reports the case of a neglected patient in which sonidegib rapidly led to an important tumor mass reduction and improvement of quality of life without of adverse effects. Conclusion: The peculiar efficacy and tolerability of sonidegib, in addition to representing a current gold standard for the treatment of the advanced disease, allow us to hypothesize its use on a larger scale in a modern multidisciplinary strategy (delete with surgery and radiotherapy).