Endocrine Abstracts

Background: Patients with advanced NET have limited treatment options. In the phase III RADIANT-2 study of patients with advanced low- or intermediate-grade NET and a history of carcinoid syndrome, everolimus, an oral mTOR inhibitor, + octreotide LAR provided a clinically meaningful 5.1-month increase in median progression-free survival (PFS) compared to placebo + octreotide LAR (ESMO 2010 Abstract #LBA8). An updated safety analysis from this trial is presented.<p class="a...

Background: Resistance to thyroid hormone alpha (RTHα), caused by mutations in the T3-receptor alpha 1 (TRα1) isoform, includes growth retardation, intellectual disability, and abnormal thyroid function tests. The current paradigm entails that disease features arise from decreased T3 action in TRα1-expressing tissues. However, also for patients that carry mutations that completely abolish T3-stimulated activity, neurological features vary strongly, ranging from ...