Physicians' Academy for Cardiovascular Education

Real-world data PCSK9 inhibitors show same LDL-c reductions, but more side effects

Proprotein convertase subtilisin / Kexin 9 inhibition in patients with Familial Hypercholesterolemia: initial clinical experience

Literature - Galema-Boers JMH, Lenzen MJ, Sijbrands EJ and Roeters van Lennep JE - J Clin Lipidol 2017; 11(3):674-681


Early initiation of lipid-lowering therapy combined with lifestyle modifications is very effective for familial hypercholesterolemia (FH) patients in preventing cardiovascular disease (CVD) [1-3]. In additions to statins and ezetimibe, PCSK9 inhibitors can now be used for high-risk patients who do not reach their LDL-c target despite maximum therapy. PCSK9 inhibitors evolocumab and alirocumab both result in an LDL-c reduction up to 60% and are well-tolerated in general [4-7].

This study was performed to evaluate the efficacy and side effects of PCSK9 inhibitors in FH patients, in real world setting rather than in clinical trials and based on initial clinical experiences. 52 Patients received evolocumab 140 mg subcutaneously every two weeks (heFH) or 420 mg subcutaneously every two weeks (hoFH), and 31 patients received alirocumab 75 mg or 150 mg subcutaneously every two weeks (heFH).

Main results


PCSK9 inhibitors in real-world setting showed similar LDL-c reductions as those observed in randomized clinical trials. However, side effects, especially flu-like symptoms, as well as injection site-reactions were reported more often compared to clinical trials. These findings emphasize the need to establish clinical registries to monitor the long-term efficacy and side effects of PCSK9 inhibitors outside randomized clinical trials.


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Find this article online at J Clin Lipidol.

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