Efficacy and safety of tafolecimab in Chinese patients with familial hypercholesterolemia: a systematic review and meta-analysis of randomized controlled trials

Background: Familial hypercholesterolemia (FH) is a prevalent inherited disorder marked by elevated low-density lipoprotein cholesterol (LDL-C) levels, predisposing individuals to premature cardiovascular disease and related morbidities. Traditional treatments often fail to achieve target LDL-C levels in many patients, necessitating novel therapies. Tafolecimab, a monoclonal antibody targeting PCSK9, shows promise in managing HeFH by enhancing LDL receptor recycling and LDL-C clearance. Objective: To evaluate the efficacy and safety of tafolecimab in Chinese patients with FH through a systematic review and meta-analysis. Methods: This meta-analysis followed PRISMA guidelines and was registered in Prospective Register of Systematic Reviews. A comprehensive search was conducted across PubMed/MEDLINE, Web of Science, Scopus, and Embase databases. Inclusion criteria focused on randomized controlled trials (RCTs) involving patients aged 18–75 with hypercholesterolemia and high cardiovascular risk. Data extraction and quality assessment were performed independently by two reviewers. Statistical analyses were conducted using random-effects models. Results: Four RCTs involving 841 Chinese patients were included. Tafolecimab significantly reduced LDL-C (mean difference [MD]: −2.05; 95% CI: −2.19 to −1.90), apolipoprotein levels (MD: −0.53; 95% CI: −0.56 to −0.50), non-HDL-C (MD: −2.19; 95% CI: −2.32 to −2.06), and lipoprotein(a) levels (MD: −0.09; 95% CI: −0.11 to −0.07). The incidence of adverse events was higher in the tafolecimab group (risk ratio: 0.68; 95% CI: 0.61, 0.75), but no significant differences were found in serious adverse events, treatment discontinuation due to adverse events, deaths, hypersensitivity, muscle-related problems, upper respiratory issues, or liver damage compared to placebo. Conclusion: Tafolecimab effectively reduces various lipid parameters, suggesting its potential in managing hypercholesterolemia and reducing cardiovascular risk. Although adverse events were more frequent, their severity did not significantly differ from placebo. The generalizability of these findings is limited to Chinese populations, highlighting the need for further research in diverse cohorts.