Clinical Trials of PCSK9 Inhibitors

A recent meta-analysis of 24 clinical trials involving 10,159 adults with hypercholesterolemia found that PCSK9 inhibitors lowered LDL levels by an average of 47% and dramatically reduced the risk of cardiovascular disease-related death.7 Although most trials involved patients already being treated with statins without meeting target LDL cholesterol goals, some focused only on patients who did not tolerate statins. The review also demonstrated reductions in all-cause mortality rates, cardiovascular mortality rates, and myocardial infarctions. With the exception of cardiovascular mortality outcome, all other reductions were statistically significant (P = 0.084).7

There were no significant adverse events, and average discontinuation rates in patients treated with PCSK9 inhibitors were comparable to those receiving placebo or ezetimibe.7 The most common side effects included itching, swelling, pain, or bruising where injection is given, nasopharyngitis, and flu, and hypersensitivity vasculitis.


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An editorial accompanying the meta-analysis8 advised greeting the results with “cautious enthusiasm.” The authors stated that while the meta-analysis offers “important preliminary information on clinical outcomes…long-term, ongoing, pivotal trials with prespecified primary CVD end points and monitoring of a broad range of adverse events will help establish the role of these novel agents in CVD risk management.”