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J Am Acad Dermatol. 2014 Nov;71(5):904-911.e1. doi: 10.1016/j.jaad.2014.05.020. Epub 2014 Jun 11.

An investigator-initiated open-label clinical trial of vismodegib as a neoadjuvant to surgery for high-risk basal cell carcinoma.

Author information

1
Department of Dermatology, Stanford University, Stanford, California.
2
Department of Dermatology, Stanford University, Stanford, California; Scripps Clinic, Dermatologic Surgery Division, La Jolla, California.
3
School of Medicine, Stanford University, Stanford, California.
4
Department of Dermatology, Stanford University, Stanford, California; Department of Pathology, Stanford University, Stanford, California.
5
Department of Dermatology, Stanford University, Stanford, California. Electronic address: tangy@stanford.edu.

Abstract

BACKGROUND:

Vismodegib is an oral hedgehog-pathway inhibitor approved for advanced basal cell carcinoma (BCC). Although most BCCs are amenable to surgery, excision of large tumors in aesthetically sensitive sites may compromise function or cosmesis.

OBJECTIVE:

We sought to evaluate the reduction in BCC surgical defect area after 3 to 6 months of neoadjuvant vismodegib.

METHODS:

This was an open-label, single-arm intervention trial with a primary outcome of change in target-tumor surgical defect area pre- and post-vismodegib (150 mg/d). Secondary outcomes were change in tumor area and tolerability.

RESULTS:

Eleven of 15 enrolled patients, aged 39 to 100 years, completed the trial. Thirteen target tumors were excised after a mean of 4±2 months of vismodegib. In all, 29% (4 of 14 patients) could not complete more than 3 months because of vismodegib-related side effects. The mean baseline target-tumor diameter was 3.2 cm, and 10 of 13 tumors occurred on the face. Overall, vismodegib reduced the surgical defect area by 27% (95% confidence interval -45.7% to -7.9%; P=.006) from baseline. Vismodegib was not effective in patients who received less than 3 months. Over a mean follow-up of 11.5 (range 4-21) months for all tumors, only 1 tumor recurred at 17 months post-Mohs micrographic surgery.

LIMITATIONS:

Short follow-up time and no placebo control are limitations.

CONCLUSION:

Neoadjuvant vismodegib appears to reduce surgical defect area when taken for 3 months or longer for nonrecurrent BCCs in functionally sensitive locations. Further studies with larger sample sizes and long-term follow-up are warranted.

KEYWORDS:

Mohs; basal cell carcinoma; neoadjuvant; surgery; surgical defect; vismodegib

PMID:
24929884
DOI:
10.1016/j.jaad.2014.05.020
[Indexed for MEDLINE]

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