Vismodegib Review Supports Use in Locally Advanced, Metastatic BCC

Vismodegib treatment benefits confirmed for locally advanced and metastatic basal cell carcinoma

medwireNews: Systematic review and pooled analysis add further support for use of the hedgehog pathway inhibitor (HPI) vismodegib in patients with locally advanced and metastatic basal cell carcinoma (la/mBCC).

“Vismodegib was identified to have a significant, consistent effect on the median duration of therapy of laBCC and mBCC”, say John Strasswimmer and co-workers from the University of Miami Miller School of Medicine in Florida, USA.

“While mBCC responses are superior to any traditional approach, the response rate for laBCC might be considered in the context of other standard treatment options including surgery and radiation therapy”, they write in JAMA Dermatology.

The team collated information from eight studies of vismodegib 150 mg/day including 704 clinically evaluable patients.

An objective response was achieved by between 28.0% and 100% of treated patients with laBCC and 30.8% and 38.0% of those with mBCC, with weighted averages of 64.7% and 33.6%, respectively.

The weighted average for a complete response in the laBCC and mBCC patient groups was 31.1% and 3.9%, respectively, and the median duration of therapy in the two groups was 37.0 weeks and 52.7 weeks, respectively.

Pooled analysis of adverse effects associated with vismodegib in 803 participants of nine trials indicated that serious adverse events occurred in between 0% and 38.5% of patients, with a weighted average of 21.4%.

Side effects led to discontinuation in a weighted average of 28.2% of patients, prompting the authors to suggest that vismodegib be considered for delivery as a “pulse treatment”.

Muscle spasms, dysguesia, weight loss, fatigue and alopecia occurred in a weighted average of 66.4%, 57.3%, 33.4%, 20.1% and 61.1% of patients, respectively. In addition, new-onset squamous cell carcinoma was reported in a weighted average of 1.3% of patients.

John Strasswimmer et al explain that comparison of vismodegib with the HPI sonidegib was not possible as there are too few trial data for pooled analysis, but write that “we suspect that response rates and adverse effect profiles are similar owing to class effect”.

The authors conclude: “Hedgehog pathway inhibitors are an effective, well-tolerated therapy for laBCC and mBCC and have great potential to improve quality of life for many patients who have limited treatment options.

“The clinical experiences of these newly [US Food and Drug Administration]-approved medications should be critically evaluated to ensure best practices for future use.”


Jacobsen AA, Aldahan AS, Hughes OB, et al. Hedgehog pathway inhibitor therapy for locally advanced and metastatic basal cell carcinoma. JAMA Dermatol 2016; Advance online publication 20 April. doi:10.1001/jamadermatol.2016.0780

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