Two intermittent vismodegib dosing regimens for long-term treatment demonstrated good activity among patients with multiple basal cell carcinomas, according to a study published in The Lancet Oncology.1

Vismodegib, a first-in-class Hedgehog pathway inhibitor, is approved for the treatment of adults with advanced basal cell carcinoma. Patients with multiple basal cell carcinomas, including those with Gorlin (nevoid basal-cell carcinoma) syndrome, require extended vismodegib treatment. Researchers evaluated the activity and safety of 2 long-term intermittent dosing regimens in this population.

For the double-blind, phase 2 trial (MIKIE; Identifier: NCT01815840), investigators enrolled 229 adult patients with multiple basal cell carcinomas, including those with Gorlin syndrome, who had at least 6 clinically evident basal cell carcinomas.

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Participants were randomly assigned 1:1 to receive vismodegib 150 mg orally once daily either in an intermittent schedule of 12 weeks of vismodegib followed by 8 weeks of placebo (arm A) or as 24 weeks induction followed by an intermittent schedule of 8 weeks of placebo followed by 8 weeks of vismodegib (arm B). Total treatment time for both arms was 72 weeks.

At week 73, the average number of basal cell carcinoma lesions was 62.7% (95% CI, 53.0-72.3) lower than baseline in arm A and 54.0% (95% CI, 43.6-64.4) reduced from baseline in arm B.

Ninety-five percent of the 227 evaluable patients had at least 1 treatment-emergent adverse event (94% in arm A vs 97% in arm B). The most frequently reported grade 3 or worse treatment-related adverse events were muscle spasms, elevated blood creatine phosphokinase, and hypophosphatemia.

Nineteen percent of patients in arm A and 17% of patients in arm B reported serious treatment-emergent adverse events. One patient in arm A died of a pulmonary embolism, which was potentially related to vismodegib-treatment.

RELATED: Vismodegib Reduces Tumor Burden in Patients With Basal-cell Nevus Syndrome

Arm A appeared to be associated with better activity, fewer toxicities, and improved adherence compared with arm B.


  1. Dreno B, Kunstfeld R, Hauschild A, et al. Two intermittent vismodegib dosing regimens in patients with multiple basal-cell carcinomas (MIKIE): a randomised, regimen-controlled, double-blind, phase 2 trial. Lancet Oncol. 2017 Feb 7. doi: 10.1016/S1470-2045(17)30072-4 [Epub ahead of print]