In a large, randomized trial, two doses of the direct thrombin inhibitor dabigatran were compared with warfarin in patients who had atrial fibrillation and were at risk for stroke. At 2 years, the 110-mg dose of dabigatran was found to be noninferior, and the 150-mg dose superior, to warfarin with respect to the primary outcome of stroke or systemic embolism.

In this nested case–control study of two cohorts, plasma levels of sex hormone–binding globulin were found to be associated with two polymorphisms of the sex hormone–binding globulin gene, SHBG. When these polymorphisms were used as predictive variables, low circulating levels of the globulin predicted an increased risk of type 2 diabetes, suggesting that genotypes and such plasma levels might help to stratify for the risk of type 2 diabetes.

The hedgehog pathway is active during embryonic development but dormant after birth. Mutations constitutively activate hedgehog genes in basal-cell carcinoma. This phase 1 trial tested the safety and antitumor activity of an inhibitor of the hedgehog pathway (GDC-0449) in patients with locally advanced or metastatic basal-cell carcinoma whose tumors were inoperable or had not responded to previous therapy. The drug appeared to have antitumor activity in patients with basal-cell carcinoma.

The hedgehog pathway is essential for certain aspects of embryonic differentiation. Abnormalities of this pathway have been found in some cases of medulloblastoma. This report describes a patient with a very advanced metastatic medulloblastoma that had such molecular abnormalities. The patient was treated with a small-molecule inhibitor of the hedgehog pathway. The response of the tumor to the agent was dramatic but transitory.