Li · Heart (British Cardiac Society) 2022 · systematic review and meta-analysis of randomized controlled trials · n=32 trials (65,861 participants)

Safety of proprotein convertase subtilisin/kexin 9 inhibitors: a systematic review and meta-analysis.

Cited 29 times in the scientific literature.

Level 1 - systematic review of randomized trials

Systematic review and meta-analysis of randomized controlled trials

PubMed 35508401 · doi:10.1136/heartjnl-2021-320556 · record verified 2026-08-27

What was done

A systematic review and random-effects pairwise meta-analysis of randomized controlled trials comparing PCSK9 inhibitors with placebo, standard care, or active lipid-lowering comparators in patients requiring lipid-lowering therapy. Eligible studies had a follow-up duration of at least 24 weeks. Certainty of evidence for each adverse outcome was evaluated using the GRADE approach.

What was found

Across 32 trials including 65,861 participants (median follow-up 40 weeks, range 24 to 146 weeks), PCSK9 inhibitors caused an absolute incidence of injection-site reactions leading to discontinuation of 15 events per 1,000 persons over 5 years (95% CI 11 to 20; high certainty). With high-certainty evidence, PCSK9 inhibitors did not increase risks of new-onset diabetes mellitus, neurocognitive events, cataracts, or gastrointestinal hemorrhage. With moderate certainty, they probably did not increase risks of myalgia/muscular pain leading to discontinuation or all-cause adverse events leading to discontinuation. Evidence for influenza-like symptoms leading to discontinuation was very limited and imprecise (risk ratio 1.5; 95% CI 0.06 to 36.58). Subgroup analyses by trial duration showed no credible differences.

Why it matters

This meta-analysis provides robust evidence that PCSK9 inhibitors have a favorable overall safety profile, reassuring clinicians and patients that concerns regarding diabetes risk, cognitive decline, or cataracts are not supported by randomized trial data.

Limits

The median follow-up across included trials was only 40 weeks, limiting conclusions about very long-term safety. Some specific harm endpoints, such as influenza-like symptoms, had very few reported events and wide confidence intervals.

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