PCSK9 and ANGPTL3 inhibitors in homozygous familial
hypercholesterolemia: A meta-analysis of randomized clinical trials
Abstract
Background: The aim of this meta-analysis was to compare the efficacy of
PCSK9 and ANGPTL3 inhibitors in patients with Homozygous familial
hypercholesterolemia (HoFH) Methods: We systematically searched selected
electronic databases until 30th November 2024. Main endpoint was the
effect of lipid lowering therapy on lipid profile: total cholesterol
(TC), triglycerides (TG), low density lipoprotein cholesterol (LDL-C),
high density lipoprotein cholesterol (HDL-C) and lipoproteins levels.
The secondary endpoint was adverse clinical effects. Results: 12 trials
involving a total of 392 patients with HoFH, were included in the
meta-analysis. At a median follow-up of 12 months, the ANGPTL3i group
demonstrated a greater reduction in mean TC [–4.27 mmol/L (165.1
mg/dL) vs. –1.37 mmol/L (52.9 mg/dL); p for subgroup <0.001],
LDL-C [–3.51 mmol/L (135.7 mg/dL) vs. –1.81 mmol/L (69.9 mg/dL); p
for subgroup <0.001], and TG [–0.61 mmol/L (54.1 mg/dL) vs.
–0.21 mmol/L (18.6 mg/dL); p for subgroup <0.001], but a
smaller impact on HDL-C compared to those treated with PCSK9i. Lipids
were reduced more in adults compared to children in the PCSK9i group
(p<0.01) but not in the ANGPTL3i group (p=0.23). Likewise, Apo-B
reduced more with ANGPTL3i compared to PCSK9i but Apo-A and Lipoprotein
(a) remained comparable between the two groups. The treatment-related
adverse events and discontinuation rates were not different between
groups. Conclusions: PCSK9 inhibitors have lower efficacy in reducing
lipid levels in HoFH patients compared to ANGPTL3 inhibitors,
particularly in children. Their effectiveness in different functional
variations of LDL-C receptors in HoFH patients needs to be established.