Experiences with SMO Antagonist Vismodegib for the Treatment of Locally Advanced Basal Cell Carcinoma

The study evaluated the use of vismodegib, a Smo inhibitor, in treating 11 patients with locally advanced basal cell carcinoma (BCC) over 4 years. The cohort included 2 males and 9 females, with an average age of 73, and 36% had Gorlin-Goltz syndrome. Vismodegib was administered for an average of 15 months, resulting in complete remission in 2 patients without relapse post-treatment. However, 2 patients died from unrelated causes, and 2 experienced remission followed by treatment suspension due to side effects, leading to loss of drug efficacy upon re-administration. The remaining 5 patients showed significant improvement but continued treatment despite side effects like muscle cramps, dysgeusia, nausea, loss of appetite, and hair loss. Side effects were managed with a combination of medications, highlighting the importance of addressing these to prevent therapy resistance and achieve long-term remission. The study concluded that while vismodegib was effective, its serious side effects necessitated careful management.