New injection for familial chylomicronaemia syndrome

摘要

The main issue in familial chylomicronaemia is recurrent episodes of pancreatitis resulting from a deficiency of lipoprotein lipase. Currently this is treated by lowering plasma triglyceride levels, but this is often inadequate. Volanesorsen was tested in a double blind, randomised controlled trial of 66 people in various countries. It successfully reduced triglyceride levels compared with placebo at 3 months. However, drug related adverse events were more common with some worrying thrombocytopenia, and the trial was too small to draw conclusions about whether the drug prevented acute pancreatitis.