In patients with basal-cell nevus syndrome, treatment with the hedgehog pathway inhibitor, vismodegib, reduced basal-cell carcinoma tumor burden, though adverse events frequently led to treatment interruption, according to a study published in The Lancet Oncology.1

Hedgehog pathway signaling plays a key role in the development of basal-cell carcinoma. Researchers therefore evaluated the efficacy and safety of vismodegib in patients with basal-cell nevus syndrome.

For the multicenter, double-blind, phase 2 trial ( Identifier: NCT00957229), researchers enrolled patients aged 35 to 75 years with basal-cell nevus syndrome and at least 10 basal-cell carcinomas eligible for resection. Participants were randomly assigned 2:1 to receive vismodegib or placebo. A total of 37 patients were subsequently enrolled in an open-label phase to continue vismodegib for up to 36 months.

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After a median follow-up of 36 months, results showed that the 26 patients treated with vismodegib had an average reduced rate of new surgically eligible basal-cell carcinomas compared with those given placebo. Patients who received vismodegib had an average of 2 new surgically eligible basal-cell carcinomas per patient per year, vs 34 new carcinomas per patient that received placebo per year.

Only 17% of patients tolerated vismodegib continuously for the full 36 months of treatment. Patients who interrupted treatment had an average of 1.7 new surgically eligible basal-cell carcinomas per patient per year, vs 0.6 for those who received treatment continuously.

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Six patients treated with vismodegib reported weight loss of 20% or more; 2 patients had grade 3 to 4 muscle cramps.

Though vismodegib appeared to be efficacious in this setting, further studies are warranted to reduce toxicity associated with vismodegib.                           


  1. Tang JY, Ally MS, Chanana AM, et al. Inhibition of the hedgehog pathway in patients with basal-cell nevus syndrome: final results from the multicentre, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Oncol. 2016 Nov 9. doi: 10.1016/S1470-2045(16)30566-6 [Epub ahead of print]