In Reply Yin and colleagues make an excellent point highlighting the fact that the underlying mutation in patched homologue 1 is not altered by vismodegib. Of course, we agree that patients must continue to be monitored long-term after the drug is discontinued for possible recurrence and more prospective data with greater patient numbers are needed to better define the ideal duration of treatment.
Our study demonstrated complete clinical regression in 2 of the 7 patients with a relatively short duration of therapy. Although Tang et al1 found that 6 of 13 biopsy specimens showed residual tumor after 3 months of therapy, this study was looking exclusively at patients with basal cell nevus syndrome. Certainly it is reasonable to expect patients with basal cell nevus syndrome to be most likely to have tumor regrowth after discontinuation of the drug. Extrapolating these results to cases of nonsyndromic BCC may not be valid.
Gill HS, Moscato EE, Silkiss RZ. Indications and Limitations of Vismodegib for Basal Cell Carcinoma—Reply. JAMA Ophthalmol. 2014;132(7):906. doi:10.1001/jamaophthalmol.2014.1805