Objective: This case series explores the use of vismodegib to treat locally advanced basal cell carcinoma (laBCC), with a focus on tolerability, efficacy, and outcomes after treatment cessation.
Methods: Data from patients who underwent vismodegib treatment for laBCC at a single institution from 3/6/2012 through 3/15/2015 was utilized in this study. For all included cases, treatment responses as recorded at the first follow-up after treatment cessation were assessed and are reported as complete clinical response (CCR), partial clinical response (PCR), stable disease, or progressive disease. In cases of CCR, clinical disease free survival (DFS) was calculated as the time from cessation of vismodegib until last available follow-up, death, or recurrence. Data pertaining to side effects and adverse events was also recorded, and results are presented using descriptive statistics.
Results: A total of 24 patients and 31 tumors met inclusion criteria. CCR was observed in 17 of 31 tumors (55%), and 13 of 31 tumors (42%) demonstrated PCR. Stable disease was seen in one patient (one tumor) (3%). No cases demonstrated clinical tumor progression during treatment. The mean clinical DFS at time of data cut off for all cases of CCR was 9.3 months (range 2-21 months). In cases of PCR, the mean reduction in tumor size was 52% (range, 11%-80%). Only two patients (8%) discontinued treatment secondary to side effects.
Conclusion: Each patient and each tumor responds uniquely to vismodegib treatment, including variable tumor responses and a wide range of side effects and tolerability. This study highlights important unique observations, and our data as a whole adds to previously published studies, leading to thought provoking questions. Overall, the FDA approval of vismodegib for advanced basal cell carcinoma has markedly improved the prognosis and care of affected patients.