In NEJM this week, researchers in the US and Europe report on the efficacy and safety of vismodegib, a small-molecule hedgehog pathway inhibitor, for the treatment of basal-cell carcinoma. In a multicenter, international, two-cohort, nonrandomized study, the team enrolled 33 patients with metastatic basal-cell carcinoma and 63 patients with locally advanced basal-cell carcinoma, and administered vismodegib. They report that the metastatic patient group had a 30 percent response rate, while the locally advanced carcinoma group had a 43 percent response rate, with complete responses in 21 percent of those patients. "The median duration of response was 7.6 months in both cohorts," the authors write. "Serious adverse events were reported in 25% of patients; seven deaths due to adverse events were noted."
Also in NEJM this week, an international group of researchers describes a combination chemotherapy regimen for patients with advanced adrenocortical carcinoma. The team randomly assigned 304 patients to receive mitotane, plus either streptozocin or a combination of etoposide, doxorubicin, and cisplatin. Patients in the EDP-mitotane group had a significantly higher response rate and longer progression-free survival than patients in the streptozocin-mitotane group, the researchers found. However, there was no significant difference in overall survival between the two groups.
Finally in NEJM this week, a team led by investigators at the Massachusetts General Hospital Cancer Center report that patients with BRAF-mutated melanoma had better survival when given MEK inhibitors. The team randomly assigned 322 metastatic melanoma patients with a V600E or V600K BRAF mutation to receive either oral selective MEK inhibitor trametinib or chemotherapy. Median progression-free survival was 4.8 months in the trametinib group compared to 1.5 months in the chemotherapy group, the authors write. And the overall survival rate at six months was 81 percent in the trametinib group and 67 percent in the chemotherapy group.