Abstract 046: PCSK9 Inhibitors in Familial Hypercholesterolemia: a Meta-analysis of 9 Randomized Controlled Trials

Background: The prevalence of Familial Hypercholesterolemia (FH) is estimated to be 1 in 250 US adults. The advent of PCSK9 inhibitors has led a to a paradigm shift in the management of these patients. The purpose of this study was to analyze the efficacy of PCSK9 inhibitors in patients with heterozygous or homozygous FH. Methods: Pubmed, EMBASE and clinicaltrials.gov were searched for randomized controlled trials of PCSK9 inhibitors. Trials that exclusively recruited FH patients or those that reported data separately for FH patients were included. The primary outcome of this analysis was mean difference in LDL-C in patients treated with a PCSK9 inhibitor compared with placebo. Secondary endpoints were change in other lipids and incidence of adverse events. A random effects model was used to analyze the pooled estimates. Results: A pooled analysis of 9 trials with a total of 1361 patients was performed. Overall the mean difference in LDL-C reduction with PCSK9 inhibition was -48.6% (95% CI: -51.3 to -45.9...