This retrospective case series evaluated the effectiveness of the hedgehog pathway inhibitor vismodegib for advanced periocular basal cell carcinoma (BCC).

Study design

Investigators identified 21 patients who were treated with vismodegib (150 mg/day) at 2 tertiary centers in Israel over a 5-year period. Six patients had advanced periocular disease whilst 15 had orbital disease.

Outcomes

Ten patients had complete response, 10 had partial response and 1 patient remained stable. Median treatment duration was 9 months, with follow-up of 26 months.

Among complete responders, five maintained a complete response at 16 months and three who stopped treatment had a recurrence at 8 months. Almost all adverse reactions related to the drug were low-grade. However, two patients demonstrated hepatotoxicity and one patient died of sepsis, which was possibly attributable to the drug.

Limitations

Although this is the largest report to date on the use of vismodegib for treatment of locally advanced periocular disease, it is a retrospective report with a heterogeneous patient population. They did not have a uniform treatment protocol. In addition, there was quite a bit of variability in the previous treatment of the patients, including radiotherapy.

Clinical significance

This study adds further support to the treatment of advanced periocular and orbital basal cell carcinoma with vismodegib. However, many questions remain, including the length of treatment, durability and tolerability. Side effects are not insignificant, with 8 of 21 patients discontinuing the medication due to the side effects. Treatment should be directed by a medical oncologist who can monitor the patient for more severe side effects as observed in two patients.