Inhibition of PCSK9 in familial hypercholesterolaemia.

摘要

Statins are indicated for treatment of patients with heterozygous familial hypercholesterolaemia: these drugs inhibit hepatic cholesterol synthesis and indirectly raise transcription and expression of low-density lipo-protein (LDL) receptors, resulting in augmented residual LDL receptor function. Unfortunately, estimates of the effectiveness of statins in this setting are missing, since placebo-controlled trials in patients with familial hypercholesterolaemia have not been done. Observational evidence about statin treatment in a UK cohort and a Dutch follow-up study provides the best available support for a substantially improved outcome in asymptomatic patients with familial hypercholesterolaemia. In agreement with this, patients with familial hypercholesterolaemia in the ENHANCE study, who had been treated with statins intensively, had a normal mean baseline carotid inti ma-media thickness.