Pharmacological treatment options for severe hypertriglyceridemia and familial chylomicronemia syndrome.

Pharmacological treatment options for severe hypertriglyceridemia and familial chylomicronemia syndrome. Expert Rev Clin Pharmacol. 2018 May 29;: Authors: Chaudry R, Viljoen A, Wierzbicki AS Abstract INTRODUCTION: A spectrum of disorders, ranging from rare severe cases of homozygous null lipoprotein lipase deficiency (LPLD) -familial chylomicronemia syndrome (FCS) to heterozygous missense LPLD or polygenic causes, result in hypertriglyceridemia and pancreatitis. The effects of mutations are exacerbated by environmental factors such as diet, pregnancy and insulin resistance. Areas covered: In this review, authors discuss chronic treatment of FCS by ultra-low fat diets allied with the use of fibrates, omega-3 fatty acids, niacin, statins and insulin-sensitising therapies depending on the extent of residual LPL activity; novel therapies in development target triglyceride-rich lipoprotein particle clearance. Previously, a gene therapy approach to LPL- alipogene tiparvovec showed that direct targeting of LPL function reduced pancreatitis events. An antisense oligonucleotide to apo-C3, volanesorsen has been shown to decrease triglycerides by 70-80% and possibly to reduce rates of pancreatitis admissions. Studies are underway to validate its long-term efficacy and safety. Other approaches investigating the role of LPL modulating proteins such as angiopoietin-like petide-3 (ANGPTL3) are under consideration. Expert Opinion: Current therapeuti...
Source: Expert Review of Clinical Pharmacology - Category: Drugs & Pharmacology Tags: Expert Rev Clin Pharmacol Source Type: research