Retreatment with Vismodegib after Progression in Advanced Basal Cell Carcinoma: First-Time Report of a Single-Institution Experience

Retreatment with vismodegib in advanced basal cell carcinoma (BCC) patients who previously discontinued the drug due to disease progression (PD) has not been reported yet. The objective of our report is to determine whether vismodegib is still active when used in BCC patients who progressed during a first vismodegib course (FVC). We conducted a retrospective study on six advanced BCC patients enrolled in a clinical trial (STEVIE, NCT01367665) who discontinued vismodegib due to PD and were then retreated with the same drug. All patients underwent intercurrent therapies between the FVC and the second vismodegib course (SVC). Disease control (complete response, CR; partial response, PR; and stable disease) was achieved in 100% and 80% of cases in FVC and SVC, respectively. The overall response rate was 80% for FVC (50% of CR) and 50% for SVC (only PR). Median treatment duration of FVC and SVC was 19.5 months (range: 13-35) and 8 months (range: 3-14+), respectively. G3-G4 AEs were reported only during SVC (two cases), leading to permanent discontinuation in one case. The median interval between FVC and SVC was 21.5 months (range: 13-30). After a median follow-up of 54 months (range: 46-63) only one patient with metastatic disease had rapid progression, discontinued vismodegib, and died. All other patients are still alive and two are currently on therapy. We concluded that vismodegib rechallenge is feasible and potentially active in advanced BCC patients who previously discontinued the drug due to disease progression.