Vol 73, No 1 (2022)
Original paper
Published online: 2022-02-14

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Latest clinical evidence about the effect of PCSK9 monoclonal antibodies in patients with familial hypercholesterolaemia: an updated meta-analysis

Qiongfang Zhang1, Lianxiang Deng1, Cong Chen1, Xu Pan1, Shan Jiang1
Pubmed: 35381104
Endokrynol Pol 2022;73(1):110-120.


Introduction: Familial hypercholesterolaemia (FH) is the most common autosomal genetic disease of cholesterol metabolism disorder. Proprotein convertase subtilisin/kexin type 9 (PCSK9) monoclonal antibody (mAb) is a new target lipid-regulating drug related to cholesterol metabolism that has been developed in recent years. The reported rate of reduction varies widely, and comprehensive assessments of efficacy and safety are lacking. Therefore, we conducted this study to investigate the clinical effect of PCSK9 mAbs in patients with familial hypercholesterolaemia to provide a theoretical reference for clinical practice.

Material and methods: We analysed the clinical data of patients, including the percentage change in LDL-C and the incidence rates of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs), from selected articles. Weighted mean differences (WMDs), risk ratios (RRs), and 95% confidence intervals (95% CIs) were calculated to compare the endpoints.

Results: The results showed that, compared with placebo, the PCSK9 mAb reduced the percentage change in LDL-C in FH patients (WMD = –45.52, 95% CI: –49.70 to –41.34, I2 = 99.6%). In addition, there was no significant difference between the experimental and placebo groups in the incidence of TEAEs (RR = 1.03, 95% CI: 0.97 to 1.10, I2 = 19.1%) and SAEs (RR = 1.02, 95% CI: 0.72 to 1.44, I2 = 0.0%).

Conclusions: Overall, PSCK9 mAbs are an effective and safe method of LDL-C reduction in patients with FH.

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