Efficacy and safety of vismodegib in advanced basal-cell carcinoma Full Text
New England Journal of Medicine, 06/12/2012
Sekulic A et al. – Vismodegib is associated with tumor responses in patients with locally advanced or metastatic basal-cell carcinoma.Methods
- In this multicenter, international, two-cohort, nonrandomized study, we enrolled patients with metastatic basal-cell carcinoma and those with locally advanced basal-cell carcinoma who had inoperable disease or for whom surgery was inappropriate (because of multiple recurrences and a low likelihood of surgical cure, or substantial anticipated disfigurement).
- All patients received 150 mg of oral vismodegib daily.
- The primary end point was the independently assessed objective response rate; the primary hypotheses were that the response rate would be greater than 20% for patients with locally advanced basal-cell carcinoma and greater than 10% for those with metastatic basal-cell carcinoma.
- In 33 patients with metastatic basal-cell carcinoma, the independently assessed response rate was 30% (95% confidence interval [CI], 16 to 48; P=0.001). In 63 patients with locally advanced basal-cell carcinoma, the independently assessed response rate was 43% (95% CI, 31 to 56; P<0.001), with complete responses in 13 patients (21%).
- The median duration of response was 7.6 months in both cohorts.
- Adverse events occurring in more than 30% of patients were muscle spasms, alopecia, dysgeusia (taste disturbance), weight loss, and fatigue.
- Serious adverse events were reported in 25% of patients; seven deaths due to adverse events were noted.