Highlights
- •We assessed FH patients using PCSK9 inhibitors in a clinical setting.
- •LDL-c decrease of PCSK9 inhibitors was comparable to clinical trials.
- •However, our patients showed more side effects compared with clinical trials.
- •This emphasizes the need to monitor the long-term side effects of PCSK9 inhibitors.
Background
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.
Objective
The purpose of the study is to describe efficacy and side effects of PCSK9 inhibitors
in FH patients in clinical practice.
Methods
Registry of all consecutive FH patients who started with a PCSK9 inhibitor at a lipid
clinic of a university hospital.
Results
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 reported by 32 patients (39%). Flu-like
symptoms (n = 12) and injection site reactions (n = 11) were most frequent. Seven
patients (8%) discontinued treatment, 5 because of side effects and 2 because of nonresponse.
Conclusion
Our initial experience of PCSK9 inhibition in FH patients in a clinical setting showed
comparable reduction in LDL-c levels but more side effects compared with clinical
trials.
Keywords
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Article info
Publication history
Published online: March 07, 2017
Accepted:
February 26,
2017
Received:
December 28,
2016
Identification
Copyright
© 2017 National Lipid Association. All rights reserved.
