Outcome variables
The primary endpoint was to assess the efficacy and safety of PCSK9
inhibitors and ANGPTL3 inhibitors in patients with HoFH. Efficacy
parameters for PCSK9i (Alirocumab, Evolocumab) and ANGPTL3i (Evonacumab)
included changes in lipid profiles, such as total cholesterol (TC),
triglycerides (TG), low-density lipoprotein cholesterol (LDL-C),
high-density lipoprotein cholesterol (HDL-C), and lipoprotein levels,
including lipoprotein(a) (Lp(a)), apolipoprotein A (Apo-A) and
apolipoprotein B (Apo-B). The secondary endpoint focused on safety, with
parameters including the prevalence of adverse events (AEs), and inverse
variance used for subgroup analyses between groups.