# Alirocumab bij homozygoot FH: ODYSSEY HoFH

*geplaatst 2020-07-14 · Cholesterol · Journal of the American College of Cardiology · doi 10.1016/j.jacc.2020.05.027 · https://hartvaat.nl/2020/07/14/alirocumab-bij-homozygoot-fh-odyssey-hofh/*

ODYSSEY HoFH trial van alirocumab bij homozygote familiaire hypercholesterolemie. PCSK9-remming bij de zwaarste FH-vorm.

## English: Efficacy and Safety of Alirocumab in Adults With Homozygous Familial Hypercholesterolemia: The ODYSSEY HoFH Trial.

The ODYSSEY HoFH trial showed that alirocumab lowers LDL cholesterol in patients with homozygous FH, though the effect is smaller than in heterozygous FH due to the limited LDL receptor activity. The results provided an additional treatment option for the most severe FH phenotype.

## Abstract (original, from the publication)

BACKGROUND: Homozygous familial hypercholesterolemia (HoFH) is characterized by extremely elevated low-density lipoprotein-cholesterol (LDL-C) levels and early onset atherosclerotic cardiovascular disease despite treatment with conventional lipid-lowering treatment. OBJECTIVES: This study was designed to assess LDL-C reduction with the proprotein convertase subtilisin/kexin type 9 inhibitor alirocumab in adult patients with HoFH. METHODS: This randomized, double-blind, placebo-controlled, parallel-group, phase 3 study evaluated efficacy and safety of alirocumab 150 mg every 2 weeks. The primary endpoint was percent reduction from baseline in LDL-C versus placebo after 12 weeks of treatment. RESULTS: Patients (N = 69) were randomized 2:1 to alirocumab or placebo. At baseline, background lipid-lowering treatment included 67 patients receiving statin (59 patients on high-intensity statin); 50 patients on ezetimibe; 10 patients on lomitapide; and 10 patients undergoing apheresis. Mean baseline LDL-C was 259.6 mg/dl in the placebo group and 295.0 mg/dl in the alirocumab group. At week 12, the least squares mean difference in LDL-C percent change from baseline was -35.6% (alirocumab [-26.9%] vs. placebo [8.6%]; p < 0.0001). Reductions (least squares mean difference) in other atherogenic lipids at week 12 were: apolipoprotein B, -29.8%; non-high-density lipoprotein cholesterol, -32.9%; total cholesterol, -26.5%; and lipoprotein(a), -28.4% (all p < 0.0001). No serious adverse events, permanent treatment discontinuations, or deaths due to treatment-emergent adverse events were reported during the double-blind treatment period. CONCLUSIONS: In the largest randomized controlled interventional trial in HoFH patients to date, alirocumab resulted in significant and clinically meaningful reductions in LDL-C at week 12. Alirocumab was generally well tolerated, with a safety profile comparable to that of placebo. (Study in Participants With Homozygous Familial Hypercholesterolemia [HoFH] [ODYSSEY HoFH] NCT03156621.).

Auteurs: Dirk J Blom, Mariko Harada-Shiba, Paolo Rubba, Daniel Gaudet, John J P Kastelein, Min-Ji Charng, Robert Pordy, Stephen Donahue, Shazia Ali, Yuping Dong, Nagwa Khilla, Poulabi Banerjee, Marie Baccara-Dinet, Robert S Rosenson

---
Bron: Journal of the American College of Cardiology, https://doi.org/10.1016/j.jacc.2020.05.027. Bijgewerkt 2026-07-03T13:27:50Z. Citeer vrij, met bronvermelding en een link naar hartvaat.nl (de url van het record). Samenvattingen zijn redactioneel werk van HartVaat; de oorspronkelijke publicaties blijven van hun uitgevers (doi). Geen medisch advies.