Proprotein convertase subtilisin/kexin 9 inhibition in patients with familial hypercholesterolemia: Initial clinical experience

Journal of Clinical Lipidology
Annette M H Galema-BoersJeanine E Roeters van Lennep

Abstract

Despite optimal lipid-lowering therapy, a minority of patients with familial hypercholesterolemia (FH) reach low-density lipoprotein cholesterol (LDL-c) target goals. In randomized trials, proprotein convertase subtilisin/kexin 9 (PCSK9) inhibitors led to impressive LDL-c reductions and a favorable safety profile. However, data about the efficacy and safety outside clinical trials are not available yet. The purpose of the study is to describe efficacy and side effects of PCSK9 inhibitors in FH patients in clinical practice. Registry of all consecutive FH patients who started with a PCSK9 inhibitor at a lipid clinic of a university hospital. We analyzed 83 FH patients (79 heterozygous FH [heFH]-65 with a genetically confirmed heFH and 14 with clinical heFH-and 4 homozygous FH [hoFH]), with a mean age of 55.1 ± 11.6 years. Treatment with a PCSK9 inhibitor resulted in an additional reduction of 55% ± 21% in mean LDL-c levels. Patients with heFH had more LDL-c decrease than those with hoFH (56% vs 38%). Patients using ezetimibe monotherapy because of statin intolerance (n = 24, 29%) had less LDL-c decrease compared with patients who concurrently used statin therapy (47% and 58%, P = .03). Side effects of PCSK9 inhibitors were repor...Continue Reading

Citations

Oct 11, 2017·International Journal of Clinical Practice·Monika KohliAnthony S Wierzbicki
Jul 28, 2018·Clinical Pharmacology and Therapeutics·Muhammed T GürgözeJeanine E Roeters van Lennep
Jul 8, 2019·Drugs -- Real World Outcomes·Klaus G ParhoferW Dieter Paar
Jun 10, 2020·American Journal of Cardiovascular Drugs : Drugs, Devices, and Other Interventions·Tim HollsteinElisabeth Steinhagen-Thiessen
Dec 29, 2020·Journal of the Endocrine Society·José Juan Ceballos-MacíasDante José Lopez-Mezquita

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