Lipid-lowering treatment for homozygous familial hypercholesterolaemia

摘要

Homozygous familial hypercholesterolaemia is a rare and devastating disorder associated with early cardiovascular disease, aortic stenosis, and death.1 Even with the available lipid-lowering therapy, these patients remain at increased risk. Both Marina Guchel and colleagues (Jan 5, p 40)2 with lomitapide, and colleagues and I with mipomersen,3 have shown that those drugs, albeit effective in reducing LDL cholesterol, increase liver fat in such patients. However, whether iiver findings are associated with an increased risk of insulin resistance, systemic inflammation, liver fibrosis, cirrhosis, and hepatic cancer4 remains to be determined.