P2.B031: Efficacy and safety of monoclonal antibodies in the treatment of children with Familial Hypercholesterolemia: a Systematic Review and Meta-Analysis

Background: Heterozygous familial hypercholesterolemia (HeFH) is a genetic disorder characterized by elevated plasma concentrations of low-density lipoprotein (LDL) cholesterol. Many pediatric patients with HeFH are unable to achieve recommended low-density lipoprotein cholesterol (LDL-C) concentrations. The evaluation of different types of human monoclonal antibodies for use in pediatric patients is needed.

Methods: Pubmed, Embase, and Cochrane Central were systematically searched for randomized controlled trials (RCTs) comparing mAbs with placebo in children with familial hypercholesterolemia. Heterogeneity was examined with I2 statistics. A random-effects model was used for all outcomes.

Results: We included 2 RCTs with 3 arms comprising 310 patients, of whom 205 ( 66.12%) underwent mABs treatment. There is a statistically significant difference in LDL-C levels between mAb and placebo groups (MD -49.46; 95% CI -67.21 to -31.72). The mean difference of non-HDL-C levels between groups in change from baseline was -35,62 (95% CI -40.85 to -30.41), and for apolipoprotein B was -33.47 (95% CI -38.21 to -28.75). No significant difference was found in the occurrence of any adverse events (RR 0.94; 95% CI 0.78 to 1.15).

Conclusion: These findings suggest that monoclonal antibody (mAB) therapy stands out as a safe and effective treatment option. The evidence presented underscores its superiority over placebo in reducing LDL-C levels to clinically desirable ranges. Thus, incorporating mAB therapy into clinical practice holds considerable promise for enhancing cardiovascular health and reducing associated risks.